Blood Safety Transcripts
DEPARTMENT OF HEALTH AND HUMAN SERVICES
ADVISORY COMMITTEE ON BLOOD SAFETY AND AVAILABILITY
Monday, September 19, 2005
Bethesda North Marriott Hotel
and Conference Center
5701 Marinelli Road
North Bethesda, Maryland 208852
- Mark Brecher, M.D., Chair
- Judy Angelbeck, Ph.D.
- Celso Bianco, M.D.
- Arthur W. Bracey, M.D.
- Paul F. Haas, Ph.D.
- Jeanne Linden, M.D., M.P.H.
- Karen Shoos Lipton, J.D.
- Gargi Pahuja, M.P.H.,J.D.
- Susan D. Roseff, M.D.
- S. Gerald Sandler, M.D.
- Merlyn H. Sayers, M.D., Ph.D.
- Mark W. Skinner, J.D.
- Pearl Toy, M.D.
- Wing Yen Wong, M.D.
- NON-VOTING EX OFFICIO MEMBERS:
- Food and Drug Administration:
- Jay S. Epstein, M.D.
- Department of Defense:
- CDR Michael Libby
- Health and Human Services, CMS:
- James S. Bowman, III, M.D.
C O N T E N T S
- Call to Order, Roll Call, Conflict of Interest,
- Minutes, Introduction of New Committee Members
- Jerry Holmberg, Ph.D. 5
- Chairman's Comments
- Mark Brecher, M.D. 8
- Review of May 2005 Advisory Committee
- Recommendations 9
- Varicella Zoster Immune Globulin (VZIG) Status,
- Dorothy Scott, M.D., FDA 14
- Update of IGIV Supply and Reimbursement:
- Update from DHHS
- Jerry Holmberg, Ph.D. 26
- IGIV Summit
- Julie Birkhofer. PPTA 37
- Immune Deficiency Foundation
- Marsha Boyle, IDF 57
- Public Comments:
- Medical Needs of Katrina Affected Area,
- Hemophilia Federation of America
- Jan Hamilton 70
- ASD Healthcare
- Tamie Joeckel 91
- Immune Deficiency Foundation 101
- Michelle Vogel
- Advanced Medical Technology Association
- Theresa Lee 105
- Committee Discussion 108
- Strategic Plan for Improving Blood Safety in
- the 21st Century:
- Report of Subcommittee Activity
- Jeanne Linden, M.D., M.P.H. 112
- Review of January and February 2005 meetings
- Jerry A. Holmberg, M.D. 115
- Structured Process for Policy
- and Decision-Making
- Jay S. Epstein, M.D. 129
- Integration of the Blood System within the
- Public Health Infrastructure
- Judy Angelbeck, Ph.D. 142
- Surveillance for Adverse Events Related to
- Blood Donation and Transfusion
- Jerry Holmberg, Ph.D. 148
- Coordination of Risk Communication
- Karen Shoos Lipton, J.D. 153
- Error Prevention in Blood Collection Centers,
- Transfusion Services and Clinical Transfusion
- Jeanne Linden, M.D., M.P.H. 158
- Donor Recruitment and Retention
- Celso Bianco, M.D. 164
- Clinical Practice Standards for Transfusion
- Art Bracey, M.D. 177
- Research Agenda 181
- Disaster Planning
- Susan Roseff, M.D. 187
- Public Comment 195
- Committee Discussion 214
1 P R O C E E D I N G S
2 Call to Order, Roll Call, Conflict of Interest
3 Minutes, Introduction of New Committee Members
4 DR. HOLMBERG: Good morning. Welcome to
5 the 27th meeting of the Advisory Committee for
6 Blood Safety and Availability. In just a few
7 minutes we will have roll call. As you have seen
8 the agenda for this meeting, we have purposely
9 dedicated a lot of time for deliberation, for
10 discussion. We have had many speakers over the
11 last couple of times and I think it is time that we
12 sit down and just really deliberate on some of
13 those discussions.
14 First of all, I want to introduce
15 everyone--probably she doesn't need any
16 introduction--but Dr. Pearl Toy is with us today.
17 She is a new member of the committee. She could
18 not be at the spring meeting, and we are pleased to
19 have you with us. Very good.
20 Now if I can go through the roll call,
21 Judy Angelbeck?
22 DR. ANGELBECK: Here.
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1 DR. HOLMBERG: Celso Bianco?
2 DR. BIANCO: Here.
3 DR. HOLMBERG: Art Bracey?
4 DR. BRACEY: Here.
5 DR. HOLMBERG: Mark Brecher?
6 DR. BRECHER: Here.
7 DR. HOLMBERG: Paul Haas?
8 DR. HAAS: Here.
9 DR. HOLMBERG: Andrew Heaton is absent.
10 Jeanne Linden?
11 DR. LINDEN: Here.
12 DR. HOLMBERG: Karen Shoos Lipton?
13 MS. LIPTON: Here.
14 DR. HOLMBERG: Gargi Pahuja?
15 DR. PAHUJA: Here.
16 DR. HOLMBERG: Susan Roseff?
17 DR. ROSEFF: Here.
18 DR. HOLMBERG: Gerry Sandler is going to
19 be here, from what I understand. He is just
20 delayed a little bit. Merlyn Sayers?
21 DR. SAYERS: Here.
22 DR. HOLMBERG: Mark Skinner?
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1 DR. SKINNER: Here.
2 DR. HOLMBERG: Pearl Toy?
3 DR. TOY: Here.
4 DR. HOLMBERG: John Walsh is absent. Wing
5 Yen Wong?
6 DR. WONG: Here.
7 DR. HOLMBERG: James Bowman?
8 DR. BOWMAN: Here.
9 DR. HOLMBERG: Jay Epstein?
10 DR. EPSTEIN: Here.
11 DR. HOLMBERG: Harvey Klein is absent.
12 Matt Kuehnert is a Public Health Service officer
13 who is deployed to the hurricane-affected area and
14 he will not be with us today. Mike Libby?
15 CDR LIBBY: Here.
16 DR. HOLMBERG: Just a word about conflict
17 of interest. On an annual basis we do a review of
18 the conflict of interest from each one of the
19 committee members for the special government
20 employees. However, I would recommend and advise
21 that any person that speaks at the microphone, if
22 there is a potential conflict of interest, I would
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1 appreciate you declaring that and also stating your
3 The minutes of the last meeting have been
4 posted on the web site. I have already introduced
5 the new committee member, Dr. Pearl Toy. Also to
6 let you know, I know that we have had a lot of
7 discussion about the membership and the change in
8 membership effective at the end of this meeting.
9 Once again, I do want to remind the people that
10 will be rotating off the committee that if the
11 bureaucracy does not move as fast as we would like
12 it to move, we do have, according to our charter,
13 the opportunity to ask you to return for the next
14 time until we can get a replacement for your
15 position. Once, again, our meeting will be in
16 January, our next meeting after this, and we will
17 reconfirm those dates at the end of the meeting
18 tomorrow. But if, for some reason, you get a phone
19 call from us, we may ask you to come back. I will
20 turn the meeting over to Dr. Brecher.
21 Chairman's Comments
22 DR. BRECHER: I would like to welcome
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1 everybody to the meeting. I am just going to
2 quickly review the recommendations from the last
3 meeting. When we last met, May 16-17, we
4 considered three topics. The first was strategic
5 actions for emerging infectious disease to reduce
6 the risk of transfusion-transmitted disease and its
7 impact on availability. The second was an update
8 on current status of bacterial detection methods as
9 a release platelet concentrate procedure. The
10 third was an update on current issues, including
11 access and availability to IGIV products.
12 Taking them one at a time, in terms of the
13 strategic actions, the committee decided that
14 numerous questions surrounding that needed to be
15 resolved prior to making a specific recommendation
16 and the issue was tabled until this meeting. So,
17 we will hear a lot more about this.
18 In terms of bacterial detection, the
19 discussion on the FDA position to require bacterial
20 testing as release criteria--we thought that there
21 was no recommendation needed and the manufacturers
22 of various platelet collection systems presented
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1 their approach to FDA-required testing and
2 postmarket surveillance. Actually, that is moving
3 along nicely I think right now. Actually, the New
4 York Blood Center will be the first to go live with
5 seven-day platelets next week.
6 An update on current issues, including
7 access and availability to IGIV products, was the
8 third topic. The committee found that, one, since
9 our prior recommendation of January, 2005 there was
10 a worsening crisis in availability of access to
11 IGIV products that is affecting and placing
12 patients' lives at risk, e.g., patients with
14 Two, changes in reimbursement of IGIV
15 products under MMA since January, 2005 have
16 resulted in shortfalls in reimbursement of IGIV
17 products and their administration.
18 Three, immediate interventions are needed
19 to protect patients' lives and health, the
20 committee, therefore, urged the Secretary to, one,
21 declare a public health emergency so as to enable
22 CMS to apply alternative mechanisms for
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1 determination of the reimbursement schedule for
2 IGIV products and, two, otherwise to assist CMS to
3 identify effectively short- and long-term solutions
4 to the problem of unavailability of and access to
5 IGIV products in those settings.
6 The Acting Assistant Secretary for Health,
7 Dr. Beato, responded to those recommendations on
8 August 8. Clearly, you cannot read that letter but
9 she thanked us for the letter. She was encouraged
10 by the progress reports on standardization of
11 protocols for detection of bacterial contamination
12 and the extension of platelet product dating. She
13 said this is an excellent example of the private
14 sector and the Department working together to
15 increase product safety and efficacy. The
16 committee's continued evaluation of strategies for
17 vigilant detection and management of emerging or
18 reemerging infectious diseases is a necessary first
19 step toward the goal of reducing the risk of
20 transfusion-transmitted diseases. The work has
21 potential impacts on blood and blood products, as
22 well as other vital products such as bone marrow,
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1 progenitor cells, tissues and organs. Please
2 continue your discussions and deliberations on this
3 important issue.
4 In terms of IGIV, she wrote that we--being
5 HHS--have investigated the current status of IGIV
6 highlighted in your comments. After extensive
7 discussions, we have concluded that at this time
8 there are sufficient supplies available to
9 patients. However, there do appear to be ongoing
10 marketplace adjustments related to how
11 manufacturers and distributors are managing their
12 respective inventories and we will continue to
13 monitor the situation. Our examination of the
14 allocation process indicates that physicians and
15 providers might best serve the patients by
16 communicating supply needs directly to
17 manufacturers and distributors. Review of the
18 current utilization of IGIV also indicates that
19 there is increased use of this product for
20 off-label use that may also be increasing pressure
21 on supplies. Therefore, we believe that physicians
22 should ensure that priority be given to IGIV
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1 treatment for FDA-labeled uses in those diseases or
2 clinical conditions that have been shown to benefit
3 from IGIV based on evidence of safety and efficacy.
4 While HHS has no control over the prices
5 manufacturers or supply distributors may charge,
6 the Centers for Medicare and Medicaid Services,
7 CMS, will continue to monitor the average sales
8 price on a timely basis, as mandated by Congress,
9 to ensure that the reimbursement reflects 106
10 percent of manufacturers' average sales price.
11 She then wrote that she was encouraged by
12 the price reports on standardization of protocols
13 for detection of bacterial contamination--we
14 already went through that one. Then, she wished to
15 express her appreciation to the committee.
16 A few days after that letter, on the web
17 site of this committee a status of immune globulin
18 intravenous IGIV products was posted, and we are
19 going to hear more about this from Dr. Holmberg in
20 a little bit. Basically, the position that was
21 presented in the letter was reiterated and there
22 was a section at the bottom that spoke to where to
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1 report acute problems to the FDA.
2 So, we are now going to move on to the
3 rest of our agenda. We will fist hear about
4 varicella zoster immune globulin, VZIG, from Dr.
5 Dorothy Scott, from the FDA.
6 Varicella Zoster Immune Globulin (VZIG)
7 DR. SCOTT: Good morning. I am just going
8 to give you a brief update on the availability of
9 varicella zoster immune globulin. I think this is
10 a new topic for this committee and we do have a
11 potential problem with shortage of this product.
12 Just a very brief background on VZIG--
13 DR. HAAS: Dr. Scott, excuse me for a
14 second. That mike is not at all clear. We are not
15 hearing well.
16 DR. SCOTT: Is that better? Can you hear
17 me better? Not really? How is this? Better?
18 Well, starting back again, I will give you
19 a brief update on this product, varicella zoster
20 immune globulin. It was licensed in 1981. It is
21 an intramuscular preparation that is made from
22 selected high anti-varicella zoster virus plasma
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1 units from normal donors. The indications for this
2 are prevention and modification of severe varicella
3 disease. This includes pneumonia, hepatitis,
4 encephalitis and mortality. The people who are
5 predisposed to this, and for whom this product is
6 indicated, are immune compromised children and
7 adults, premature infants, infants less than one
8 year of age because they are at greater risk of
9 severe disease, and selected non-immune pregnant
10 women and healthy adults that have never had
11 varicella, again, because they are at greater risk
12 of severe complications. It should be administered
13 within 96 hours of exposure to varicella. I didn't
14 mention that varicella is really chicken pox. It
15 also causes shingles.
16 We have only had one manufacturer of this
17 product, Massachusetts Public Health Biological
18 Laboratories. They are scheduled to close their
19 plasma fractionation facility and they are not
20 making anymore VZIG. They have a number of other
21 products. We are also working with them on these
22 other products to provide supply through other
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2 The VZIG supply that we have, based on
3 usage in the past several years, is anticipated to
4 last until 2006. The approximate number of vials
5 per year that are used are 10,000 of the smaller
6 vial, so larger size for adults which is 625 units.
7 It is a weight-based dosing scheme so 10,000 vials
8 treat, at a minimum, 2000 adults or 10,000 of the
9 smallest patients, and that would be 10 kg or less.
10 What have we done so far? We have
11 encouraged new INDs and BLA submissions for VZIG.
12 There are several companies not licensed in the
13 U.S. that make this product already. We defined a
14 path to licensure, or at least discussed it at the
15 Blood Products Advisory Committee meeting on July
16 21 of 2005. I will go into that in just a moment.
17 We are monitoring the supply. Fortunately, there
18 is only one distributor so that is easy to do, and
19 they are familiar with shortages of other products.
20 We are in communication with CDC to look at other
21 options and to help them make decisions about VZIG
22 and IGIV usage in substitution and we have a public
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1 communication effort.
2 Very briefly, these are the Blood Products
3 Advisory Committee meeting questions. We asked
4 them to discuss what laboratory and clinical data
5 would be sufficient to demonstrate efficacy of a
6 new product. The subset questions are which target
7 populations would be most informative to study? I
8 think I have shown you that there are a number of
9 indications for this in different patient
10 populations. What surrogate markers might be
11 appropriate for assessment of efficacy? We also
12 asked for other considerations about how to do a
13 clinical trial for licensure. In addition, we
14 asked them to comment on whether the available data
15 support use of IGIV or acyclovir as a substitute
16 for VZIG for prophylaxis against severe infection.
17 This is the outcome of their discussion.
18 The target populations are only present in low
19 numbers because there are not a lot of susceptible
20 people anymore due to childhood vaccination against
21 varicella with the vaccine. It is also difficult,
22 therefore, to study this in a short time frame due
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1 to the variety of clinical situations but small
2 numbers of any particular kind of subject.
3 They discussed the use of surrogate
4 markers for licensure, and the committee agreed
5 that a PK equivalence in normal subjects compared
6 with the licensed product, combined with a
7 laboratory demonstration of equivalence compared to
8 the licensed product, would be sufficient for
9 licensure under a surrogate marker strategy. And,
10 this comes with a Phase 4 commitment to further
11 study for its efficacy and validation of the
12 surrogate marker. A surrogate marker, for example,
13 would be anti-varicella zoster titers in people who
14 received this product.
15 The other question was could IGIV
16 substitute. Obviously, people are being vaccinated
17 and there are still plenty of donors that have been
18 naturally infectsed So, what are the titers
19 against varicella in IGIV? We were able to help
20 CDC look at this, and it looks as if they are
21 somewhere around 4-8-fold lower than what is seen
22 in the licensed product. But from lot-to-lot there
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1 is no particular titer tested for any of the immune
2 globulin products. That makes sense because they
3 don't carry this indication. However, there is
4 variation between manufacturers and among lots
5 within the same manufacturer so it would be
6 difficult to give IGIV as a substitute unless you
7 knew the titer and could give the right dose.
8 In addition, titers of IGIV in general may
9 diminish as vaccinated donors replace naturally
10 infected donors. The titers in general in
11 vaccinated people are lower than they are in people
12 who are naturally infected.
13 The other question was could acyclovir
14 just be a substitute for prophylaxis of severe
15 disease? There is not sufficient efficacy evidence
16 for this particular indication with acyclovir. It
17 may be helpful, but it appears to be more helpful
18 in later stages of the disease, whereas VZIG is
19 expected to prevent the viremia in these patients.
20 These were the speakers we had from
21 Massachusetts come to speak about the VZIG
22 manufacture or potency testing and the current
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1 supply status. Dr. LaRussa came and talked about
2 the disease correlates of protection and the
3 different options of post-exposure prophylaxis and
4 antivirals in immune globulin. CDC also provided a
5 speaker, Mona Marin, who talked about the
6 recommendations for post-exposure prophylaxis of
7 severe varicella. In addition, we had a special
8 member of the committee, Jane Seaward, also from
10 So, what is the current situation? We do
11 have ongoing supply monitoring. We are in
12 communication with the distributor, FFF Enterprises
13 and Massachusetts. We believe we have enough
14 supply to last at least through January. We are
15 requesting that only people who need this product
16 order it. It can be shipped right away and arrive
17 within 24 hours. In other words, of those 10,000
18 vials that were used last year, it seems that
19 people believe that a lot of that sat around in
20 pharmacy inventories and was never used. So, it is
21 important to get this product to people who need it
22 and not to have it sitting around outdating in
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1 somebody's inventory.
2 FFF Enterprises has agreed to do this,
3 that is, to inquire whether or not the product is
4 needed for a specific patient in order to ship.
5 This was their decision but it seems like a wise
6 choice from the standpoint of preserving supply as
7 long as possible.
8 We have agreed to review INDs and BLA
9 submissions. I would note that this product would
10 be eligible for orphan drug classification. There
11 is a very small number of people that need this in
12 the U.S. relative to regular IGIV. They would be
13 eligible to request cost recovery for an IND
14 product and we will consider treatment protocols.
15 In other words, we want to get a product to people
16 before January, a new product, and one of the ways
17 to do that, even if the license is not yet
18 approved, is to have a treatment protocol under an
20 We also have a web site posting planned.
21 We expect it will be up this week, and this will
22 tell everybody about the licensed uses; request
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1 them to only use it for specific patients and not
2 to order for inventory; and give the information on
3 how to obtain VZIG.
4 Clinicians and pharmacies should only
5 order for identified patients. This product can be
6 ordered from FFF Enterprises at this number, and it
7 can be delivered quickly. FFF Enterprises is also
8 keeping track of which hospitals they have sent
9 inventory to in the past, which gives us the
10 potential for hospital-hospital transfer of VZIG if
11 needed. In other words, there is some product out
12 there. It has already been shipped and there is
13 probably a way to move it around. They have agreed
14 to track this.
15 So, thank you for your attention and I
16 will take any questions.
17 DR. BRECHER: Art?
18 DR. BRACEY: Yes, I had a question in
19 terms of the amount of product that may be outdated
20 and, therefore, gone to waste. It strikes me that
21 in terms of the need for resource sharing I think
22 one option, of course, is the option that you
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1 presented, but the regional blood centers are
2 pretty good resources for sharing inventories and I
3 wonder if you, all, had given that some thought in
4 terms of making these regional blood centers
5 depositories of product.
6 DR. SCOTT: That is a very good point I
7 think and maybe we should talk about it a little
8 more afterwards because I am not sure I understand
9 what would be involved. But FFF right now is the
10 sole repository and they do have a very rapid
11 shipping plan for this and for other products.
12 They have worked on shortages before. But I think
13 we should consider all options and I would like to
14 discuss that further.
15 DR. BRECHER: Jay?
16 DR. EPSTEIN: Thank you for the update.
17 Another issue on which we have been getting inquiry
18 is whether it is reasonable for pharmacies to
19 aliquot smaller quantities from these larger vials
20 since really only the adult size vials are
21 available. Do we have any opinions about the
22 safety of that practice, and can it be frozen after
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1 it is aliquot'd?
2 DR. SCOTT: Right. Thanks, Jay. I should
3 have mentioned that there are only 625 unit vials
4 left, which is the dose for an adult. The doses
5 for children come in 125 and you give 1-4 of those
6 to a child depending on its weight. We think that
7 it is reasonable to consider aliquot-ing the
8 correct dosage amount if you receive this product
9 for a child. The other question was about freezing
10 of the material.
11 DR. EPSTEIN: Well, if you aliquot it,
12 then there is always the risk of breaking
14 DR. SCOTT: That is right.
15 DR. EPSTEIN: Which is the question of
16 whether you should freeze the aliquots.
17 DR. SCOTT: I think it is a good question,
18 but we tend to hesitate when it comes to
19 manipulating a product that way and it is supposed
20 to be used within a certain period of
22 DR. BRECHER: Is there any way to extend
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1 the outdate? Is it stored liquid or is it frozen
3 DR. SCOTT: It is not frozen. It is 2-8
4 storage and, actually, I don't think the outdate
5 will be a problem because we expect to run out of
6 this before the outdate. But is there a way to
7 extend the outdates in general? Absolutely there
8 is. We just need a submission and the data on
9 potency and other aspects of the product. It is
10 not difficult to do at all.
11 DR. BRECHER: Celso?
12 DR. BIANCO: Thank you for the update. Is
13 there hope to have companies approach FDA that
14 could replace the Massachusetts Lab?
15 DR. SCOTT: We have two companies that
16 have approached FDA and expressed interest, and we
17 are working hard with these companies so that we
18 can have product provided before we run out of it.
19 DR. BRECHER: If there are no further
20 comments or questions, thank you, Dr. Scott. We
21 are now going to move to an update on IGIV supply
22 and reimbursement. First we will hear from DHHS,
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1 Dr. Holmberg.
2 Update on IGIV Supply and Reimbursement
3 Update from DHHS
4 DR. HOLMBERG: Well, part of my update was
5 to go through some of the recommendations but this
6 has already been done by Dr. Brecher. You have the
7 committee recommendations from the last time, and
8 from the recommendations that were put forward I
9 have to say that the Secretary and the various
10 agencies such as CMS were very concerned about the
11 recommendations and how do we move forward with
12 these recommendations.
13 What we did shortly after the
14 recommendations were received, we did have
15 discussion with the distributors. We talked not
16 only at the distributors but we also talked to the
17 manufacturers. We have had discussions with the
18 Plasma Protein Therapeutic Association, CMS, Immune
19 Deficiency Foundation, various providers and the
20 pharmacist groups and, of course, patients.
21 The providers indicated difficulty in
22 obtaining specific brands of IGIV for some
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1 patients. This is not only for the privately
2 insured but also the Medicare. A lot of the
3 concerns that came from the providers was the fact
4 that rates that were set by Medicare were quickly
5 accepted by the other insurers and that this was
6 having a great impact on the location of where the
7 product was being infused.
8 The shift in treatment location, of
9 course, followed. We saw that very quickly after
10 January 1, and the pharmacists were the first--I
11 should say the healthcare providers--to really feel
12 the effects of this. Once the physicians moved the
13 patients over to the hospital outpatient setting,
14 the hospitals that did not have an allocation or
15 had a lower allocation than in previous years were
16 starting to really scramble to try to get their
17 product. Hospitals have reported difficulty in
18 obtaining physician IGIV product of choice for the
19 patient and we have followed up on many, many of
20 those calls and comments. There is an upward trend
21 in the price, most notably in the secondary market.
22 Some of the findings that we uncovered
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1 were that there was an increase in off-label use of
2 IGIV. This was as a result of our discussion with
3 the industry. We came to the realization that
4 there was a consolidation of the market; that there
5 are now five manufacturers. The American Red Cross
6 is shortly going to be removing itself from the
7 business. Change in business practices was that
8 companies had decided that they did not need to
9 keep a large inventory on the shelf and that they
10 could meet the needs with a shorter inventory.
11 This shorter inventory then had direct impact on
12 the distributors' quantity. So, there was an
13 overall reduction in inventory, smaller numbers to
14 the distributors.
15 As I already mentioned, the MMA, effective
16 January, 2005, changed the Medicare Part B to 106
17 percent of the manufacturer's average sales price.
18 I stress that that is the manufacturer's average
19 sales price plus 6 percent. That does not take
20 into consideration what the distributor adds on.
21 So, my understanding in investigating this is that
22 the 6 percent is for the physician storage and
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1 maintenance of the product. We also have seen that
2 the Medicare payment rate is updated quarterly and
3 that there was an increased nine percent for
4 lyophilized IGIV in July of 2005.
5 What we also uncovered was that there were
6 sufficient supplies of IGIV for patients who needed
7 the treatment. From our discussions with the
8 manufacturers we also came to the conclusion that
9 it was under the manufacturers' allocation process
10 that sometimes there were shortages at the
11 hospitals and that the physician would do best in
12 communicating that supply need directly to the
13 manufacturer. If there was an emergency need, the
14 manufacturers were very willing to establish an
15 emergency supply.
16 I know that PPTA is going to be talking in
17 a few minutes. I will let them talk a little bit
18 more about that, but with my colleagues in the Food
19 and Drug Administration, Dr. Weinstein and Dr.
20 Nippon, we did contact the manufacturers. We
21 talked to many of the executives at the
22 manufacturers for the fractionators and discussed
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1 some of the concerns out there that we were hearing
2 and seeing, and one of the things that we stressed
3 upon them was a need for an emergency inventory
4 supply being available for patients that truly
5 needed it.
6 We also found with the pharmacy groups
7 that to ensure that IGIV treatment was prioritized
8 correctly many pharmacies have established a
9 prescription review, and they prioritize towards
10 the FDA-labeled use in those diseases or clinical
11 conditions that have been shown to benefit from
12 IGIV based on evidence of safety and efficacy.
13 One of the things that I can mention here
14 is that there is only a handful of labeled
15 indications for use and, yet, the CMS does
16 permit--I think it is 30 different clinical
17 entities for reimbursement of IGIV.
18 Some of our action plan that we did was,
19 as Dr. Brecher mentioned, shortly after the letter
20 that he received from Dr. Beato, we did post on our
21 web site a report of our view of the status of
22 IGIV. When people ask me to really talk about
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1 this, I think that I use the phrase that maybe
2 somebody brought up at one of the last meetings,
3 "the perfect storm." I think that that was the
4 phrase that was coined at the advisory committee,
5 but it was a perfect storm in the fact that we had
6 a difference in supply; we had an increased demand,
7 and we also had a change in the reimbursement
9 The web posting states that if there is a
10 report of a denial of treatment or delay of
11 treatment or forced reduction in dosage, we want to
12 hear about it. We have put in there the FDA web
13 site and also the 800 number. Dr. Nippon is
14 responsible for monitoring that and she keeps me
15 posted on a regular basis as far as what the status
16 is of the calls that have come through. CMS also
17 has an 800 Medicare number that they have a script
18 written for that they can start collecting data on,
19 and they have been collecting for several months
20 the information on any denial.
21 On top of that, I have to say that any
22 time somebody calls in with a complaint to my
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1 office, I personally have followed up on it. It is
2 very interesting going back and talking to the
3 pharmacists, and also people at CMS have talked
4 directly to CEOs of different medical facilities
5 and have gotten care to the patients that are
6 needing it. So, there is merit in making sure that
7 the government is aware of any denial of service,
8 especially for Medicare patients.
9 As I mentioned before, I will leave it for
10 PPTA to discuss but the supply channel and the
11 emergency reserves have been identified with PPTA.
12 Also, each one of the manufacturers has established
13 a 1-800 number, a toll-free number, for the
14 physician that is having difficulty in obtaining
15 the product to talk to the medical director of the
16 fractionation company.
17 Another aspect, and this is more of a
18 long-term approach, is that we are seriously
19 looking at an evidence-based study to try to
20 determine what are the clinical uses of IGIV and
21 what are the data out there to support the clinical
22 use. So, that is an ongoing study that I am in
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1 discussion about with CMS and the agency for Health
2 Research and Quality.
3 CMS has been challenged by Dr. Beato to
4 continue to monitor the cost. As I have mentioned,
5 it is monitored on a quarterly basis. Something
6 else that we have initiated internally is IG
7 assistance, Inspector General assistance, in
8 looking at the IGIV problem. This has been
9 reiterated by support by Congress. I am aware of
10 at least two congressmen, and I believe I
11 incorporated those letters in your package. I have
12 requested that Secretary Leavitt enlist the help of
13 the Inspector General. This has been one of our
14 long-term or our investigational approaches also.
15 So, that is a quick update on the status.
16 As I can tell you, this is the letter that Dr.
17 Brecher has already mentioned. This was our web
18 posting of the situation, the status of the IGIV.
19 So, if anybody has not been to our web site, I
20 would encourage you to go to that. We have not
21 posted the 1-800 numbers on the government web
22 site. I refer people to the PPTA web site to get
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1 the 1-800 numbers.
2 Then also, just to give you a quick
3 update, and maybe Dr. Bowman could probably speak
4 to this a little bit better than I could but, Jim,
5 if you would like to jump in at any point, please
6 feel free to. The 2006 acute hospital inpatient
7 payment, the final ruling is out. The date of
8 publication was August 12. The 2006 HOPPS proposed
9 rule was out July 25 and the comments were to be
10 back last week, on September 16. Then also, the
11 2006 HOPPS correction went out on August 26 and,
12 again, the comments to those corrections were to be
13 back in the middle of September.
14 The 2006 physician fee schedule proposed
15 went out on August 8 and comments are due back on
16 September 30, as well as the corrections that were
17 published on September 1.
18 There are also some locations where you
19 might want to get some more information. For the
20 audience, they may want to take this information
21 down, the web site for CMS for the providers and
22 also the federal registry notice. You can go to
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1 the GPO access.gov/federalregistry. If you ever
2 want to find a federal registry, that is a good
3 place to look for it. Then also, payment for Part
4 B drugs, there is a web site listed there also. I
5 believe that is in your handouts. Are there any
6 questions for me or for Dr. Bowman?
7 DR. BRECHER: Sue?
8 DR. ROSEFF: I have a question, Jerry.
9 When I read the letter that was in our packet that
10 you just talked about, the physicians are supposed
11 to directly feed back to the manufacturers. That
12 is recommended. Is there a mechanism to make that
13 easy and to track the physicians giving input to
14 the manufacturers?
15 DR. HOLMBERG: Well, from the government
16 side, you know, what they report back to the
17 manufacturer is really out of our domain. But the
18 800 numbers have been provided and they can call
19 back and talk directly to the medical directors
20 there. However, if there are problems, especially
21 with a Medicare patient, then we strongly encourage
22 that that gets funneled through 1-800 Medicare and
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1 that way we can keep track of it and we can
2 follow-up on it. The other mechanism, as I
3 mentioned, is the FDA and this would be both for
4 Medicare and privately insured people if they are
5 experiencing some delay in getting product. But
6 direct input from the manufacturers, I don't get
7 that unless the manufacturers offer it directly to
9 DR. BRECHER: Merlyn?
10 DR. SAYERS: How much traffic did that web
11 site pick up that you posted?
12 DR. HOLMBERG: That is a good question and
13 I don't have the answer for that, but I have heard
14 a lot of people refer to it and I have referred it
15 to the press wanting to know a little bit more of
16 what is going on in the status. As I mentioned, I
17 have not posted the 1-800 numbers for the
18 manufacturers and, you know, that is probably
19 something that we need to do, to put that on our
20 web site so that there is greater dissemination of
21 those telephone numbers, but I have been directing
22 people to the PPTA.
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1 DR. BRECHER: Thank you, Jerry. Now we
2 can hear from the PPTA.
3 PPTA IGIV Summit
4 MS. BIRKHOFER: Thank you and good
5 morning. It is a pleasure to be here in Bethesda
6 again before the advisory committee to talk about
7 the reimbursement issues. The topic today is
8 intravenous immune globulin access. Dr. Holmberg
9 did an excellent job providing a summary of where
10 we are currently. I was asked to talk about a
11 summit meeting that PPTA convened on September 7.
12 Even though I am not an attorney, I just
13 want to start with a disclaimer. The summit
14 meeting was not intended to be a defined group that
15 PPTA, you know, is sanctioning as the IVIG group.
16 This was done rapidly, in about a ten-day period,
17 where PPTA went out and took a cross-sector of the
18 IVIG community and invited leaders from those
19 organizations. So, I just want to be really clear
20 that the summit group participants that were a
21 cross-section of the physicians, the consumers,
22 industry and distributors, was in no way meant to
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1 be perceived as the be-all and the end-all of a
2 defined group. It was simply a working group that
3 convened on an issue-specific Hospital Outpatient
4 Prospective Payment System, short-term, to address
5 the access in the hospital outpatient system. So,
6 I just want to really be clear on that.
7 Just to give you a sense of the impact of
8 the new proposed reimbursement in the hospital
9 outpatient rule, you can see there the rates as
10 they impact lyophilized, the powder and the liquid.
11 PPTA submitted comments on Friday, the 16th, and
12 this joint summit group also submitted comments.
13 As you can see, there is a short window period
14 between the 16th and November 1 but realistically
15 by mid-October CMS will begin to make decisions.
16 So, PPTA and interested parties are working to
17 impact the agency to have them focus on the need to
18 assure the adequacy of the rates to sustain patient
20 Currently, we have seen the impact of the
21 Medicare Modernization Act's broad, sweeping
22 legislation. When we were here in May we focused
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1 on the impact of that legislation in the physician
2 office, which is Part B. HOPPS technically is Part
3 B as well. But we see a switch in the Hospital
4 Outpatient Prospective Payment System of 83 percent
5 of ASP, which is currently the $80.68
6 reimbursement, to an ASP plus 8 percent. Again,
7 looking at lessons learned from the physician
8 office, will the ASP plus 8 percent be sufficient
9 to sustain patient access to care? That is really
10 what this discussion is all about.
11 We have looked at the definition of ASP
12 and we have tried to offer some insight into what
13 may be the cause of the limitations of ASP, and
14 there is a lag time. Currently, there is a
15 six-month lag time in physician office and a
16 nine-month lag time in the hospital outpatient. We
17 just had a meeting with CMS on September 15 and we
18 were able to clarify that they do intend to balance
19 or equalize that lag time, which should have a
20 positive impact on the calculation.
21 Additionally, as has been discussed, this
22 is a very fluid and very dynamic market. You know,
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1 prices may fluctuate. They can, and they do,
2 fluctuate within a six-month period and a CMS
3 calculated ASP may not always reflect the current
4 market dynamics. We have also respectfully asked
5 for validation or verification of the rates by a
6 third-party auditor simply because we see the
7 immediate impact these rates have on the ability of
8 Medicare beneficiaries to access therapy, and we
9 all know from previous presentations that there are
10 no generics; there are no alternatives; there are
11 no substitutes. It is not a one-size-fits-all
13 So, lessons learned: We have seen that
14 ASP plus 6 percent and likely plus 8 percent has
15 restricted the physician/patient freedom of choice,
16 and that is really what PPTA and its member
17 companies are all about. PPTA member
18 companies--Baxter, Talecris, Octapharma, Grifols,
19 ZLB Behring, those are the five companies that
20 manufacture IVIG and Bayer is also a member. They
21 are currently manufacturing a recombinant factor.
22 But those five companies are committed to making
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1 therapy. They are committed to making product
2 available. They leave the decision to the
3 physician and the patient and that is the sanctity
4 of that relationship that my member companies are
5 committed to preserving.
6 Providers currently are reporting that ASP
7 plus 6 percent is not a sustainable business model
8 and there are reported disruptions in site of
9 service. Marsha Boyle, from the IDF, will give you
10 further detail on a more current survey but there
11 is plenty of data from the IDF that show 67 percent
12 of patients receive IVIG under the physician
13 payment system in the physician office.
14 So, what has been the impact on consumers?
15 Who are we talking about? Let's really put a face
16 to Medicare beneficiaries that use IVIG. We are
17 talking about 7,000 human lives, 7,000 people that
18 need access to this life-saving therapy. There are
19 no alternatives. Again, 67 percent of those
20 receive infusions in the physician office; 32
21 percent receive infusions in the hospital
22 outpatient setting.
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1 So, when you look at consumers and what
2 the impact has been--my column should be aligned; I
3 apologize it is not--we see in 2005 a shift from
4 the physician office to the hospital setting, and
5 in 2006 we can predict a volume of
6 patients--migration if you will--from home care,
7 from physician offices, into the hospital
8 outpatient setting and that is an immediate problem
9 and the opportunity to fix it is now. Again, CMS
10 is in the rule-making period. They do have
12 So, how can they fix it? What can be
13 done? PPTA, working in unison with the IVIG
14 community--and these proposals are not anything
15 that PPTA has come up with on their own. There is
16 a group of people that all deserve credit for these
17 recommendations. We recommended classifying IVIG
18 as a biologic response modifier. That would affect
19 the physician payment side. That would get it into
20 a higher category. Right now IVIG is classified in
21 a low complexity category, similar to that of
22 saline. Those of you on the advisory committee
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1 that are physicians know that IVIG is a complex
2 therapy. Infusions need to be monitored. Expert
3 nurses deliver that infusion. It is a four- to
4 eight-hour process. There is the chance that
5 during an infusion there could be reactions. This
6 is not a low complexity drug. It is high
7 complexity and should be classified as a BRM. We
8 are working on that.
9 There are political hurdles. Everything
10 is political when it comes to this issue. The AMA
11 is involved. The AMA has issues with physician
12 payment reform if they classify IVIG as a BRM and
13 reduce the rate for something else. Congress has
14 told CMS to look at it. CMS says we can't decide
15 if it is a BRM unless we hear from the AMA. So, it
16 is this real classic game of political ping-pong.
17 At the same time, the imperative need is to assure
18 consumer, patient access. So, this back and forth
19 needs to stop and IVIG should be classified as a
20 biologic response modifier.
21 In addition, we are recommending that the
22 HCPC codes be de-bundled; that you have
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1 product-specific reimbursement based on the NDCs,
2 the National Drug Codes. Some groups have said,
3 you know, classify IVIG as a blood product. Again,
4 to you experts in blood- and plasma-related issues,
5 it is probably very apparent to you that IVIG is a
6 blood product. However, there is a disconnect.
7 Although the FDA recognizes and regulates IVIG as a
8 blood product, CMS does not because they say IVIG
9 is so highly manufactured that the end product is
10 not a blood product. I think they are thinking
11 along the lines of platelets, red cells, more of
12 the pure--although albumin is a blood product.
13 Again, it is a little bit of a disconnect but that
14 is what makes this reimbursement issue fascinating
15 and complex.
16 Additionally, we have suggested that a
17 demonstration project be conducted--similar to what
18 was done for chemotherapy, done for dialysis,
19 renal--that would result in additional payments to
20 providers that participated in conducting the
22 CMS did take action. You know, they are
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1 trying to solve the problem. It is a complex
2 problem. If any of us had the solution that was
3 easy maybe we wouldn't all be here talking about
4 IVIG on a quarterly basis. But CMS divided codes,
5 liquid versus lyophilized. It is not a complete
6 fix. That is why the industry and the IVIG
7 community, recognizing the distinct, unique nature
8 of each brand of IVIG think the better solution
9 would be to de-bundle entirely and to again have
10 the NDC-based reimbursement.
11 Of course, all of these recommendations we
12 have raised with CMS in comments; we have raised
13 with CMS at meetings. I know Dr. Holmberg has had
14 several discussions with CMS. They tell me now
15 they call him Jerry and they see Jerry all the
17 The 2006 HOPPS impact on access--again, I
18 don't have a crystal ball. I can only look at the
19 experiences from the physician office and predict
20 it will be negative. The window of time to act is
21 now. Medicare is seen as a model, also Medicaid.
22 You know, let's not forget CMS has jurisdiction
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1 over Medicaid. And, we know that Congress is
2 looking at a ten billion dollar package of savings,
3 reductions in Medicaid, and we know that Medicaid
4 will likely move to an ASP model. So, the
5 reverberations negatively on patient access to care
6 could be catastrophic.
7 So, we want to draw upon conclusions from
8 the physician office. We ask ourselves the
9 question, you know, can or will ASP plus 8 percent
10 be sufficient to sustain access to care in the
11 hospital outpatient settings, which is clearly not
12 the optimal setting for someone who is immune
13 compromised and it is also the setting of last
14 resort. As I showed you in that chart earlier, the
15 hospital outpatient setting will soon be
16 over-saturated and the question is and then what?
17 So, collectively PPTA convened a summit on
18 September 7 to come up, as I said, with a
19 short-term solution, issue specific, and to
20 immediately focus on the Hospital Outpatient
21 Prospective Payment System. Some major outcomes of
22 are that--aside from the fact that 30, 40 people
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1 were able to sit in a room and come to consensus
2 and act in a unified voice, which was I think
3 unprecedented--there was a recommendation that
4 there should be an add-on for IVIG. There should
5 be a dampening provision applied that some
6 calculations with regard to ASP should be modified
7 to include the prompt pay discount; and that IVIG
8 should be classified as a biologic response
10 Additionally, there is precedent for this
11 group recommending that there be an increased
12 reimbursement or an add-on for IVIG. MedPAC, the
13 Medicare Payment Advisory Commission, recommended
14 25-30 percent of ASP. CMS, their own APC
15 committee, recommended that the 2 percent add-on
16 would not be sufficient and that industry data on
17 additional reimbursements on the pharmacy overhead
18 should be considered.
19 So, the 2006 HOPPS situation does present
20 an urgency and opportunity. Dr. Holmberg mentioned
21 PPTA's companies' commitment to access and the fact
22 that the companies have made manufacturer toll-free
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1 numbers available. Manufacturers are reporting a
2 robust emergency supply. But, again, the
3 reimbursement situation is really defining the
4 ability for Medicare beneficiaries dependent upon
5 life-saving IVIG to access care. If there are any
6 questions I would be happy to address them.
7 DR. ANGELBECK: Could you just expand a
8 little bit for me? Your statement about providers
9 reporting ASP plus 6 percent is not a sustainable
10 business model, and even potentially at the plus 8
11 percent level it is questionable, is that providers
12 throughout the whole system? Does that include
13 physicians? Does that include companies? Can you
14 just define that a little bit more for me, please?
15 MS. BIRKHOFER: When I use the term
16 providers I am really meaning physicians and maybe
17 home care companies to a certain extent. But in
18 the Medicare settings I do know that in the
19 physician office that is causing a migration to the
20 hospital setting. The ASP plus 6 is not sufficient
21 to cover the cost of the drug.
22 DR. ANGELBECK: What about the
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1 manufacturers? Do you think that they are
2 beginning to look at this and wondering if it is a
3 sustainable business model for them for this
5 MS. BIRKHOFER: The companies are
6 committed to manufacturing life-saving therapies
7 and, you know, we have had some consolidations,
8 some shifts, some changes in the market. I would
9 like to think that there has been an equilibrium or
10 a balance brought to the market but, you know, I
11 certainly can't predict what the future will be.
12 But I can say with certainty, based on our supply
13 data, that the companies are manufacturing to
15 DR. BRECHER: Mark?
16 DR. SKINNER: I guess two things, I am
17 curious about the system where physicians are urged
18 to contact the manufacturers to report shortage of
19 use, how you see that system working and if PPTA
20 has any kind of aggregate information from its
21 members from the reports that doctors are making to
22 your member companies.
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1 MS. BIRKHOFER: PPTA does not interject
2 themselves into the relationship between the
3 manufacturer and the customer. These numbers were
4 put out there very publicly, and because it is
5 customer information the companies have numbers
6 available, not just for IVIG but for each and every
7 therapy that they manufacture. The situation
8 currently with IVIG is not any different than other
9 therapies, the factor, the alpha-1, and the need to
10 have access to care. So, we don't see a role for
11 PPTA as an association, for any variety of reasons,
12 interjecting into that customer/manufacturer
14 DR. BRECHER: Jerry?
15 DR. HOLMBERG: Julie, I saw on your slide
16 that there was one comment about the NDC-based
17 reimbursement. Can you explain that a little bit
19 MS. BIRKHOFER: Sure. Medicare and
20 Medicaid, the federal payers, have systems in
21 place, coding systems. They have HCPC codes,
22 Healthcare Common Procedure Codes; they have
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1 Ambulatory Payment Classification codes, APCs.
2 Each drug, each brand, each dosage size has a
3 specific National Drug Code, an NDC. It is down to
4 the incremental level of vial sizes. That is why
5 we think to assure access and the adequacy of
6 reimbursement to have an NDA-based reimbursement,
7 rather than everything under one HCPC code where it
8 is susceptible to volume-weighted averages, and
9 that can impact access by brand. We know that
10 consumers need access to the brand that works best
11 for them. We would like to get it down to the very
12 specific NDC-based reimbursement. So, it is really
13 a coding issue.
14 DR. BRECHER: Art?
15 DR. BRACEY: Could you clarify one thing
16 for me? Has the industry looked at the actual cost
17 of producing the product? In other words, we know
18 what the sales prices are and the wholesale prices
19 but what does it cost actually to make the product?
20 MS. BIRKHOFER: Well, I can tell you that
21 for plasma-derived therapies such as IVIG it is a
22 very capital-intensive investment. It is very
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1 costly from the raw material that is used, the
2 source plasma, through the manufacturing and the
3 fractionation process there are a series of steps.
4 These facilities are huge structures that require
5 filtration HEPA filters; the infrastructure of
6 employees, the range of employees that you need to
7 have from highly skilled down to people that keep
8 things absolutely clean so that you can be in a
9 clearance 1, air clearance 2 zone.
10 So, I can tell you that these therapies
11 are very different than traditional chemical
12 synthetic therapies and they are very costly to
13 manufacture, again, from the starting material
14 through the process. The regulatory environment
15 constantly impacts the cost and, again, there is a
16 good reason for that just to assure the safety and
17 quality of therapy. So, the companies totally
18 align themselves with the process of the regulatory
19 hurdles and thresholds and there are costs involved
20 with that.
21 Specifically, again from an association
22 perspective, I can't speak to price but I can tell
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1 you that it is a costly therapy. Depending on the
2 weight of the person and the amount of IVIG they
3 need, it can be approximately a $5,000 infusion
4 every three weeks. And, we don't hide behind the
5 fact that it is costly or expensive. It saves
6 lives. It is necessary. And, again, the entire
7 process--there are reasons for these costs. It is
8 very, very different from manufacturing pills and
10 DR. BRECHER: Jerry?
11 DR. HOLMBERG: Julie, I have two
12 questions. Let me give you the first question and
13 then I will come back and ask you the second
14 question. Back at the May meeting of the Advisory
15 Committee for Blood Safety and Availability there
16 was a web posting from the FDA on the use of
17 albumin. Has that influenced the demand of albumin
18 and improved any of the use of the product or the
19 quantities, and also the manufacturers' production
20 of this to offset the cost of some of the other
22 MS. BIRKHOFER: Yes, the information
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1 posted on the FDA site was helpful. I have not
2 seen an immediate impact but it has been
3 incremental, as would be expected. As you note,
4 the integrated product portfolio within the plasma
5 therapy products, the alpha-1, the albumin, the
6 IVIG, the plasma-derived blood clotting factor--how
7 much you can manufacture of one depends, you know,
8 on the economics of how much you can sell of the
9 other because there are storage costs, handling
10 costs. You know, you can't manufacture IVIG and
11 what do you do with the paste? What do you do with
12 the proteins that you have taken from the plasma
13 for the other therapies? But, clearly, the need to
14 have a strong albumin demand and market would
15 impact in a positive manner the IVIG situation.
16 So, we do appreciate what the FDA did and we are
17 hoping to see an upswing.
18 DR. HOLMBERG: My other question is a
19 question that I ask a lot of pharmacists when I
20 talk to them. They comment about their allocations
21 and most recently I heard from a pharmacist that
22 was responsible for two hospitals. One hospital
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1 had a small amount of allocation; the other
2 hospital had zero allocation and, yet, they saw an
3 influx of patients in both of the hospitals. The
4 pharmacists are very concerned. They get the
5 physician banging at their door and the
6 complaints--and the question that I have,
7 especially from the infusion services, is what is
8 happening to the allocations? If the physician is
9 no longer infusing in the infusion center or in the
10 physician's office, what is happening to
11 allocation? Is it being moved over to the hospital
12 where it is now being infused?
13 MS. BIRKHOFER: Well, I do know that some
14 distributors, and that is really where this
15 question gets to, do have mechanisms in place where
16 the product tracks with the user. Again, I think
17 that is kind of a function of the market, if you
18 will, as to how those determinations are made.
19 Allocation, as we have talked about in the past, is
20 an effort to assure that there is sufficient
21 product where it needs to be and it takes into
22 account historical order volumes. So, currently if
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1 a hospital or an entity has not, for their own
2 business practice decisions, chosen to engage in
3 contracts it is difficult at this time, given the
4 dynamics of the market, to get the therapy. But,
5 again, some distributors do have, from what I am
6 aware of, mechanisms in place where the product
7 tracks with the patient.
8 DR. BRECHER: Paul?
9 DR. HAAS: Julie, as a follow-up to
10 Jerry's first question, if there is an increased
11 demand for albumin I would assume that would help
12 spread the capital cost between albumin and IVIG.
13 Does that then have a lowering effect upon the IVIG
15 MS. BIRKHOFER: I really can't comment on
16 what impact that would have on pricing.
17 DR. BRECHER: Merlyn?
18 DR. SAYERS: Thanks. I didn't hear all of
19 your talks so if I missed this, my apologies. But
20 do you know what proportion of the overall use of
21 IVIG is for off-label indications, and to what
22 extent that segment of the market has grown?
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1 MS. BIRKHOFER: I know those figures from
2 data from the Immune Deficiency Foundation and I
3 have ranges that anywhere from 40-60 percent of the
4 IVIG is for off-label use. But, as an association,
5 we work with the consumer groups and we work with
6 the users of the labeled indications so I don't
7 really, you know, track that.
8 DR. BRECHER: Thank you, Julie. We are
9 now going to hear from Marsha Boyle, from the
10 Immune Deficiency Foundation.
11 Immune Deficiency Foundation
12 MS. BOYLE: While this is being set up I
13 just want to thank the committee so much for paying
14 attention to this issue. I am the president of the
15 Immune Deficiency Foundation. I am a co-founder.
16 And, I have an adult son who is married and
17 healthy, working very hard, a productive member of
18 society because he was diagnosed early. He gets
19 his IVIG and his immunologist dictates how much he
20 should get; where he should get it; and how often
21 he should get it. Not reimbursement. So, this is
22 something necessary for every patient who requires
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2 Thank you so much for acknowledging the
3 crisis that many Medicare patients are facing and
4 not being able to get IVIG. It is a life-saving
5 therapy, as you know. I know you took a rather
6 controversial position in May in recommending a
7 public health emergency. We know that no one likes
8 this terminology but, as far as I understand, it is
9 one of the only mechanisms to allow CMS to increase
10 reimbursement rates for IVIG to a purchasable rate
11 and to allow patients to receive the appropriate
12 brand at the most appropriate site of care by the
13 best trained professionals in the administration of
15 You are certainly not alone in this
16 recommendation. Over 30 members of Congress have
17 recently signed a letter to Secretary Leavitt that
18 follows your recommendation to ensure patients
19 receive access to IGIV in all sites of care. We
20 have a little packet. That letter is enclosed, if
21 you would like to look at it. So, thank you again.
22 Congressman Israel and other members of
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1 Congress have contacted CMS about patients not
2 being able to receive IVIG in their physician's
3 office. The first response was to have the
4 constituents call the 1-800 Medicare or go on-line
5 to find another physician to administer IVIG. That
6 really was not a successful response. When CMS was
7 further pressed by continued inquiries from
8 senators and congressmen, CMS wrote back to members
9 of Congress to have patients go to hospitals. That
10 also is not acceptable. The problem certainly is
11 not getting better.
12 As you have heard from Julie, PPTA did
13 host an IVIG summit to develop recommendations to
14 prevent the reimbursement crisis from occurring
15 under the hospital outpatient setting. IDF is very
16 supportive of these recommendations and is proud to
17 be part of this group. But as we work to prevent
18 access to care in the hospital patient setting from
19 being reduced for so many patients, we must not
20 forget that the other important sites of care, such
21 as physician offices, infusion suites and home care
22 settings, need to be available to our patient
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1 population immediately.
2 For many of our patients these really are
3 the most important settings for care and for the
4 ability to lead healthy and productive lives.
5 Aside from undue stress and negative health
6 outcomes from being switched, in my opinion the
7 long-term impact of physicians not being reimbursed
8 to cover the cost of treating patients is that
9 fewer specialists will be available in the future
10 to provide proper diagnosis and treatment to
11 patients whose health depends upon early diagnosis
12 and state-of-the-art care.
13 At IDF, since January 1, we have been
14 getting daily phone calls about this situation, but
15 we wanted to quantify the impact this has had on
16 the community. Therefore, we did survey our
17 community, both physicians and patients, Medicare
18 patients. I personally want to thank Jerry
19 Holmberg who has been in touch with us regularly
20 and has followed up on many of the phone calls and
21 problems that we have seen that have been quite
22 upsetting, to put it mildly.
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1 First I would like to spend a couple of
2 slides going back to a survey that we did in 1997
3 that really shows the impact of IGIV on the primary
4 immune deficiency community. This was a national
5 patient survey that was a follow-up to another
6 survey, a survey of patients who are treated with
8 As you can see, prior to diagnosis 90
9 percent had unusual or repeated infections. This
10 is not your typical situation. As far as the
11 health impact before diagnosis, something like 44
12 percent had irreversible, permanent functional
13 impairment before diagnosis and the onset of
14 therapy. As far as the health status before
15 treatment, in less than 20 percent was it good to
16 excellent after you show the impact of
17 intramuscular, which certainly was an improvement,
18 but after being on IVIG almost 75 percent indicated
19 good to excellent health. I think this is
20 self-evident but I think at times we just need to
21 be reminded of the tremendous impact of this
22 wonderful therapy for our patients.
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1 What we did, we conducted a telephone
2 survey of Medicare patients. These patients had
3 been selected from our 2002 national patient survey
4 that we knew were on IGIV and also were Medicare
5 patients. The response rate was very good, as good
6 as any survey you will find conducted by the
7 government. Really only 9 percent declined. We
8 think the results are quite indicative of the
9 impact of this reimbursement problem. Of these
10 Medicaid patients, 81 percent are now on IVIG. As
11 you can see, their current source of health
12 insurance is Medicare but some certainly do have
13 alternate sources of health insurance.
14 This is a summary of several slides, but
15 of this patient population, patients who have any
16 problems with their health because of reimbursement
17 problems is 39 percent, so almost 40 percent of
18 Medicare patients surveyed. Some of the problems
19 include less tolerated product; lower dose; less
20 frequent; changed locations, 12 percent; stopped
21 infusions, 3 percent. We receive calls on every
22 one of these.
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1 This slide was a single slide kind of at
2 the end of many of the questions, just kind of
3 asked a little differently and of these, 22 percent
4 have had to pay more; had their doses reduced;
5 interval increased; switched to less preferred
6 brand; postponed infusions. Again, we have had
7 many phone calls on postponing infusions; having to
8 pay more. In many cases in the private pay or in
9 the physician office or in the home care setting,
10 the co-pay is not taken. In the hospital it is
11 always taken and we know of patients who no longer
12 can afford to have therapy because of that
14 Change in site I think is rather dramatic.
15 As you can see, of the people who had reported
16 changing site, 51 percent had been in physician
17 offices, with 9 percent since January 1. Then, the
18 other slide is the increase in hospital outpatient.
19 So, we know where our patients are going and what
20 is happening to them.
21 Why do they change the site of infusion?
22 It is pretty self-evident. We have had quite a few
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1 verbatims but the one I like is the explanation I
2 got from my doctor which is that Medicare had
3 started not reimbursing enough to cover the
4 doctor's office cost. That sort of floored me
5 because Medicare and my insurance is paying about
6 $648 more than they were paying to the doctor's
7 office so, certainly, this is not saving money and
8 it is causing undue stress to the patients.
9 Why less frequent infusions? Now some
10 local carriers are dictating that trough levels be
11 at a certain amount--"because the hospital was
12 having problems with Medicare for this and they
13 would not treat me unless my level was below 600
14 and normal is 1,000. My doctor decided to extend
15 it to eight weeks, hoping levels would stay below
16 600 but I am having sinus infections," and it goes
17 on. Less frequent infusions--well, they are going
18 to get sick and now some carriers are, you know,
19 trying to practice medicine.
20 Why they were changed to a less tolerated
21 product, "well, because I had to change locations
22 because of the Medicare pricing. I also didn't
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1 react well to the last medication at the doctor's
2 office which was changed due to pricing." So, you
3 know, when they go into the hospital, you have
4 heard Julie talk about the allocations. If they
5 can get the product, they are getting a different
6 product and they are having reactions.
7 Some of the side effects from new
8 products, as you can see, that were reported in the
9 survey are high blood pressure; rashes; headaches,
10 85 percent; nausea; fever; shortness of breath.
11 Again, this is all because they had to change
12 product from the one that, you know, was safe for
13 them and that they were used to.
14 Negative health effects as a result of
15 problems in getting IVIG, of those who had problems
16 which was 15 percent of all Medicare patients, 40
17 percent reported having negative health effects.
18 Some of these health effects--they went on for
19 pages but trying to get it down to one slide,
20 although I don't think many people can read this,
21 but the one I highlighted is, "before I went to
22 Criticare I went to another hospital for treatment
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1 and they gave me the wrong kind and I had little
2 spots on me. I had a really bad reaction and the
3 doctor mentioned kidney failure." Other infections
4 are pneumonia, bronchial infections, stomach
5 infections--you know, it goes the gamut. Again,
6 this product is important for our patients and if
7 they have to delay getting it or not receiving it
8 their health is going to be compromised
10 Well, this is kind of scary. Who is
11 responsible for the problem in getting IVIG?
12 Forty-four percent blamed the government in one way
13 or another, and I don't think the government likes
14 to be in that position.
15 As far as confidence in future treatment
16 by experience of IGIV problems, less than half who
17 have had treatment experience are confident that
18 they will be able to get their product in the
20 Rating of the U.S. healthcare system by
21 experience with IVIG problems, again, less than
22 half the patients who have had problems think the
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1 U.S. healthcare system is doing a good job in
2 getting proper treatment to the patient.
3 Now, these results closely reflect our
4 fact survey that we did earlier in a national
5 sample of 558 physicians who reported having
6 primary immune deficient patients in our 2003
7 physician survey. As you can see, the number of
8 patients treated by these physicians who responded
9 to our facts survey was over 4,000 primary immune
10 deficient patients and about 935 other patients
11 receiving IVIG.
12 As far as asking if they had significant
13 difficulty obtaining IVIG products for patients
14 because of reimbursement, 33 percent reported
15 having difficulty and this corresponds with the 39
16 percent that we reported in our patient
17 survey--significant difficulty in obtaining IVIG
18 products by number of PID patients. I think it is
19 no surprise. It tends to go up with the number of
21 Patient impact of problems because of
22 availability, again, these are quite reflective of
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1 what was reported by the Medicare
2 patients--postponed infusions; different site of
3 care; interval increase; brands less preferred;
4 alternate therapy.
5 Adverse health events, 18 percent of all
6 doctors reported them but 43 percent of doctors had
7 patients with reimbursement problems and this,
8 again, corresponds to the patient survey with 40
9 percent of all patients having problems and 15
10 percent of all patients.
11 So, you know, with this survey we are
12 trying to give information that is not just
13 anecdotal. Our anecdotal stories are
14 heart-breaking and they are not going away. I
15 think you can see that the health of patients is
16 being needlessly compromised. Although we know it
17 certainly wasn't the government's intention, it is
18 the unacceptable outcome.
19 Patients should not have to die to get
20 attention, which has already been reported in one
21 case. We are certainly working within the system
22 to bring about change for our patients and we will
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1 continue this effort. However, we can't do it
2 alone. We need your help. We need the help of
3 this committee. We will do whatever it takes to
4 get the attention of the American public that an
5 FDA-approved product is being denied to some
6 patients who have federal insurance because of
7 reimbursement rates. This isn't acceptable and we
8 all know that private payers tend to follow
9 Medicare rates, as does Medicaid, and that
10 jeopardizes even a larger percent of our very
11 fragile population.
12 So, thank you for your concern, and we
13 hope that you will continue working on this and
14 recommend solutions to ensure that our patients and
15 all patients who require IGIV are able to obtain it
16 in all sites of care and all brands. Thank you
17 very much, and do you have any questions?
18 DR. BRECHER: Marsha, I noticed from you
19 slides that in your survey of the doctors it
20 implied that 20 percent of the patients were for
21 other indications. What is your estimate of
22 off-label use?
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1 MS. BOYLE: Again, I can't say I know.
2 Generally, for the primary immune deficient
3 patients the figure is usually around 30, 34
4 percent. Off-label, we have heard from other
5 sources that it is over 50 percent or close to 50
6 percent. I don't think anyone really knows. We
7 have a sense of our population and I actually think
8 it is larger than what the estimates have been.
9 DR. BRECHER: Other questions or comments?
10 MS. BOYLE: Thank you very much.
11 DR. BRECHER: We are now going to enter
12 one of our public comment periods. I guess we will
13 first hear about the medical needs of
14 Katrina-affected areas, Ms. Jan Hamilton, from the
15 Hemophilia Federation of America.
16 Public Comments
17 Hemophilia Federation of America
18 MS. HAMILTON: Good morning and thank you
19 for the opportunity to tell you a little bit about
20 what is really going on in Louisiana. Some of the
21 comments that I am going to make, you may wonder if
22 that really has anything to do with healthcare and
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1 I am going to tell you that it really does because
2 I want you to really think as I mention each one of
3 these things what would really happen under these
4 kind of circumstances.
5 First of all, there are things in the 21st
6 century that we take for granted--a roof over our
7 heads; food to eat; ability to earn a living;
8 access to healthcare; transportation to wherever we
9 want to go whenever we want to do it or whenever we
10 need it. Up until now no one has ever experienced
11 the wrath of a hurricane like Katrina. I have been
12 in the hurricane belt virtually all of my life. I
13 have heard the warnings. We have all heard the
14 warnings. We all know how to go out and buy
15 batteries and do all that kind of stuff, and we
16 have a tendency to feel complacent about what we
17 know we can handle and what we can't. No one has
18 ever experienced anything like what Katrina brought
19 to the Gulf Coast. I heard Sen. Mary Landrieu say
20 she had been to the tsunami area and there was a
21 difference. With the tsunami the water came and it
22 left. With Katrina it came and it stayed and it
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1 created havoc.
2 The reaction and response to the
3 hurricane--warnings were given. Evacuation--we had
4 a beautiful evacuation route planned. We had
5 widened highways. We had made contra-flow. We had
6 done all these kinds of things and some people
7 followed the advice and left early. Others had no
8 means of transportation. The City of New Orleans
9 had access to hundreds of school buses and MTA
10 buses. They didn't move them to higher ground.
11 They were under water at the time they needed to be
12 used for evacuation.
13 I have heard a lot of people say it is a
14 black/white issue. It is not a black/white issue.
15 The mayor of New Orleans is black. The fire chief
16 is black. The police chief is black. But 67
17 percent of the population is black. So, you know,
18 with that kind of percentage there are going to be
19 a lot of those people that are not able to be
20 reached. The problem is they didn't start soon
21 enough. President Bush started asking on
22 Wednesday before the storm for Governor Blanco to
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1 allow them to move in and start helping. She
2 declined until well after the storm. So, that is
3 part of the problem.
4 For the people that left on time it went
5 pretty well. For others that waited, the two-hour
6 drive as far as Lafayette turned into a 14-,
7 16-hour drive. People ran out of gas. The gas
8 stations along the way didn't have any gas because
9 there had been so many people that needed to take
10 advantage of it. They didn't take enough food or
11 water or even flashlight batteries with them so
12 that created a problem.
13 Again, when you think of the population of
14 New Orleans, and everybody says around 500,000,
15 that is just New Orleans. That is not St. Bernard
16 Parish or Plaquemine's Parish or all those other
17 parishes that were involved in the evacuation.
18 State leaders really delayed in asking for federal
19 help, causing all kinds of delays in assistance.
20 Communication didn't exist. Telephone towers were
21 wiped out. There were no cell phones. There was
22 no way to communicate. We knew and the rest of the
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1 state knew what was going on because we could watch
2 in on TV. The people in New Orleans couldn't watch
3 it on TV and many of them didn't have radios. With
4 communication gone, how do you even find patients?
5 This is a really strange story. There was
6 one hospital that continued to operate even long
7 after the hurricane had hit. Nobody knew there was
8 anybody in that building, treating patients.
9 Finally, about three days later, one of the nurses
10 went to the window and was just waving out the
11 window and finally they realized that there were
12 people in there. There were actually still
13 patients in this hospital, working on just
15 Another thing that happened, and this is
16 not funny; it is really kind of stupid and I hate
17 to say this but a lot of hospitals had generator
18 power. Guess where the generators were--in the
19 basement. It makes a lot of sense, doesn't it for
20 a city that is as far under sea level as New
21 Orleans is.
22 I am going to use an example, a model set
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1 up at the Cajun Dome in Lafayette. That is my home
2 and I do know a lot about what happened there. I
3 talked with all of the leaders, Lafayette Medical
4 Society, American Red Cross, churches, United Way,
5 Salvation Army, city parish government. All of
6 them got together and they put things into motion.
7 In the beginning it worked really well. The first
8 shelter was set up at the Cajun Dome and it was for
9 people. Then they realized that a lot of people
10 had brought their pets and, for sanitary reasons,
11 they couldn't allow the pets to stay there. So,
12 they took another facility, another arena, and set
13 it up for the pets and they got the SPCA involved,
14 all the animal care people, and everything, and
15 people were donating all kinds of cages, and
16 everything, so people could get pets over there.
17 Dog food was donated. Veterinarians were there.
18 This is very important because of the mental health
19 of these patients and they had lost everything,
20 they needed their pets with them. Some of them
21 even smuggled them inside their clothes on the
22 buses that were allowed to leave with them.
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1 Members of the medical society I am very
2 proud of. They were able in some way to get in
3 touch with the interns and residents from LSU in
4 Tulane that were evacuated to Lafayette and they
5 put them to work immediately, along with volunteers
6 from the parish medical society. They emptied all
7 of their sample closets. They got donated
8 supplies, compassionate care supplies from the
9 manufacturing companies and they set up a beautiful
10 triage clinic in the Cajun Dome. You can imagine
11 the kinds of things--infections, asthma, along with
12 the just day-to-day things that people deal with
13 like diabetes, dialyses, heart patients, cancer
14 patients, all these kinds of things. Then there
15 was a special needs center that was set up in
16 another facility that was right next door to a
17 hospital so those patients who needed even stronger
18 care could be treated there.
19 A lot of the chain pharmacies even agreed
20 to fill prescriptions. They would take on some of
21 these compassionate care products and use them to
22 fill prescriptions for people because they didn't
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1 have any money. Many of them thought they were
2 leaving home for two or three days. It has now
3 been three weeks and some of them will never go
4 back and some of them may be able to go back at
5 some time or another.
6 The university hospital system in Tulane
7 lost all their records. They didn't lose them all,
8 they just couldn't be accessed. So, you have
9 patients presenting with--yes, I take this little
10 white pill in the morning for my blood pressure,
11 and then there's this little red pill that I take
12 for this. Oh, there's this little yellow one that
13 I take for this. You have no records. You have
14 nothing to go on by what they are telling you. The
15 more educated people were able to--some of them
16 even had their bottles of medicines on them or a
17 list but, sadly, the majority of them, really they
18 didn't know. So, these physicians were starting
19 from ground zero.
20 This is the first part where I just want
21 to cry. There was friction between the Red Cross
22 and the medical volunteers because the kind of
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1 treatment they were giving didn't fit the protocol
2 of American Red Cross so they made them leave. Now
3 there were these thousands of patients who were
4 being cared for beautifully within this shelter who
5 are now--they have no cars and they now have to
6 access the emergency rooms and the walk-in clinics
7 to get care. It is really sad. For instance, in
8 our city we experienced in 15 days the growth that
9 any city is expected to do in 15 years. So, just
10 think about that, and think about the fact that
11 even to get a prescription filled in a pharmacy
12 sometimes took as much as 24-36 hours because they
13 just couldn't get enough of the drugs.
14 Our office happens to be in Lafayette. It
15 is right on I-10, the southern part of the state
16 between Mississippi and Texas, and a lot of people
17 came there. There were a lot of people that had
18 relatives there and our office is set up there.
19 So, we set up a conference call with clotting
20 factor manufacturers, along with representatives
21 from NHF, and we identified what to do with some of
22 the hemophilia patients. We identified United
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1 Blood Services in Lafayette to house and distribute
2 compassionate factor. They already have an
3 existing system, delivery system set up and they
4 carried some product anyway so it was a natural
5 for them to do it, at no charge. And, the Gulf
6 States treatment center in Houston was identified
7 for those people there. There was also a place in
8 Dallas they could go and a place in San Antonio.
9 They could go to treatment centers there. In our
10 treatment center we couldn't even find Dr.
11 Lessinger from Tulane for a while. Then she showed
12 up and guess where she showed up. In Lafayette.
13 So, we opened our doors to her and she and her
14 social worker and her staff were housed in our
15 offices. And, we seem to have become the center
16 for distributing all of these goods and services
17 that are coming in from anywhere and we truly,
18 truly, truly appreciate it.
19 In the time that we couldn't locate Dr.
20 Lessinger we contacted two groups of hematologists
21 in Lafayette who treat patients with hemophilia,
22 one group at University Medical Center and another
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1 in private practice. They agreed to do whatever
2 they could do for those patients within that area.
3 In our area the city limits are 100,000 but our
4 trade area is 500,000 so there were a lot of people
5 in the surrounding towns that were able to get care
6 that way.
7 Then on September 12 Dr. Lessinger and her
8 staff moved in. We gave them telephones, desks,
9 and so forth, and they have been set up there in
10 our offices. We have also set up a hemophilia
11 disaster relief fund for patients who have needs
12 other than medical. If you can just imagine trying
13 to start over--one day you wake up and your house
14 is two sticks and you have nothing. You don't have
15 a family picture. You have some of the pictures on
16 TV that showed the missing children and it is just
17 a little black profile. Some of them have nothing.
18 They had nothing when they left.
19 Even connecting family members separated
20 during the evacuation became a major problem. Ham
21 radio operators have been a big, big, big help but
22 they were also located in the Cajun Dome and the
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1 Red Cross asked them to leave because they wouldn't
2 allow the room that they were working out of to be
3 locked at night when they weren't there. If I was
4 a ham radio operator I wouldn't want to leave my
5 tens of thousands dollars worth of equipment there
6 either with about 10,000 people in the building.
7 During all this time, I guess it was about
8 the day after the hurricane, Rep. Bobby Jindal's
9 office called me and asked for input on the
10 healthcare needs in the face of Katrina, and they
11 helped put together the next phase of relief,
12 actually tried to cut through as much red tape as
13 possible. This, again, doesn't really have
14 anything to do with healthcare treatment and, yet,
15 it does because the results of not doing it do
16 result in healthcare, and that is the fact that
17 those buses sat there in New Orleans without any
18 drivers, the metropolitan buses and the school
19 buses that should have been moved to higher ground,
20 and the answer was that the reason they weren't
21 used is that they couldn't find any drivers. Well,
22 hello! In times of an emergency you shouldn't have
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1 to have a CDL to be able to drive a bus to get
2 people to safety and drive them as far as need be.
3 So, this began my survey of all of the
4 things that we saw as obstacles. Here are some of
5 the obstacles: Defiance of individuals not wanting
6 to leave their affected areas. This was home. It
7 is New Orleans and it is home. The same thing with
8 Biloxi. There is sort of a compassionate feeling;
9 generations had been there.
10 Lack of adequate search and rescue
11 personnel and delay in requesting federal aid. The
12 delay in requesting federal aid from the state was
13 a big, big, big mistake and that is another place
14 where we feel that the red tape should be cut. I
15 do know that at one time President Bush was
16 considering evoking the Insurgency Act and maybe
17 there should be something that could be done to not
18 have to wait for a governor to come in to help in a
19 situation like that. In the first place, just in
20 an everyday situation, you don't have enough people
21 to be able to deal with this sort of immense
22 emergency. In the second place, when a lot of them
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1 have already left you sure don't have the
2 facilities. So, you need help from somewhere.
3 There was a very slow response in our area
4 of the state by FEMA and the Red Cross to get the
5 individuals registered and get aid to the evacuees.
6 Not until a couple of days ago did the Red Cross
7 start distributing any finances to the people, and
8 it was $350 per person or up to $1,500 for a
9 five-member family.
10 The clothing and all of the other things
11 were being done by the Salvation Army and by local
12 organizations. FEMA was absolutely non-existent in
13 Lafayette. We knew that there was FEMA in Baton
14 Rouge. We could not find any FEMA in Lafayette.
15 They were in Houston. They were all over Texas but
16 they weren't in Lafayette where we had about 40,000
17 to 50,000 worth of evacuees.
18 Then my answer was, well, I will start
19 sending out e-mails to the delegation and say, you
20 know, find them. Where are they? And the next
21 day, on Sunday, I got a call from a lady in Baton
22 Rouge who was with FEMA and she said, well, we have
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1 60 contract employees in Lafayette but none of them
2 really work for FEMA. So, there was no one that
3 was calling the shots. It was just a bunch of
4 hired help and they didn't know what to do.
5 There needs to be some sort of better
6 screening process to identify the people with
7 medical problems and to keep families together.
8 There are still children who don't know where their
9 mothers are, and mothers and grandmothers who don't
10 know where their children and grandchildren are.
11 Parents of hospitalized newborn babies weren't
12 notified where their babies were air-lifted to and
13 it has taken until this past week--actually, I
14 think there is still one baby that has not been
15 united with its parents. If you can imagine going
16 through a birth during that kind of a situation and
17 then having your baby taken from you and flown out
18 some place and you are not even told where they
20 The evacuees were not given a choice of
21 which city to go to. They were just put on a bus
22 and sent somewhere. A lot of the families were
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1 separated and put on different buses.
2 All of these things lead to mental health
3 issues. They may not be actual medical issues but
4 they are mental health issues that really create a
5 major problem. I just can't even imagine, you
6 know, losing everything you have and then not
7 knowing where the rest of your family is. The
8 special needs portions of the population, whether
9 it is hemophilia, diabetes, high blood pressure,
10 multiple sclerosis, immune deficiency, alpha-1,
11 whatever it is, it has a major impact upon their
12 condition just under normal conditions. But if you
13 can imagine going through this and still having
14 that problem!
15 So, what do we do next time? Make sure
16 that the state officials invite federal help
17 immediately, before the storm hits. Mayor Nagin
18 said that he did not really want to make the
19 evacuation mandatory because some of those people
20 had been there all their lives. But nobody had
21 ever seen anything like this. The levee was built
22 for category 3 hurricanes and nobody knew what
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1 would happen. They should have been made
2 mandatory. There should be a sound plan in place
3 prior to onset and started at least two to three
4 days earlier. You know, it is better to be safe
5 than sorry.
6 Some kind of backup communication methods.
7 The TV stations had satellite communication. Why
8 couldn't that have been used by the people who were
9 in charge? Each vulnerable state, Atlantic Coast,
10 Gulf Coast, West Coast, wherever they are should
11 have in place a really good plan in order to be
12 prepared and to not face the kinds of things that
13 are being faced right now.
14 And to be sure to incorporate outside
15 help, be ready to incorporate outside help. For
16 instance, from our city there were 100 boats and
17 300 people that left at 4:30 one morning to go down
18 there to try to help evacuate the people. They got
19 down there and they weren't allowed to go because
20 they didn't have anybody to direct them where to
22 There needs to be mass transportation
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1 strategy for evacuation beyond the areas of the
2 storm's path, and I don't mean just 30 miles
3 outside but far enough away that it doesn't have
4 such a tremendous impact on the population,
5 especially for those that don't have access to
6 personal transportation, and identify in advance
7 medical centers outside of the storm's path to be
8 designated as the triage centers for the various
9 patient populations and have computer backup
10 available. Every hospital should have off-campus
11 backup somewhere safe, in a vault, doctors' offices
12 in hospitals, somewhere where that can be reached
13 when it needs to be.
14 In a recent statement released by the OMB,
15 they stated that proper response to disaster relief
16 should be unified, coordinated and effective. Boy,
17 that sums it up and that is what it has not been.
18 Some of the things that have happened--I
19 mentioned that I had e-mailed the delegation with
20 the problems and gotten responses. The first
21 response came back from FEMA. Then I got a call
22 just a few days ago from the Vice President for the
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1 Quality Assurance for the Red Cross. He said,
2 "I've gotten all these e-mails with your name on it
3 that said to call you and find out what was going
4 on," and I kind of let her have it about some of
5 the things, even the distribution of food that was
6 going to the outlying centers. It was being
7 prepared in Lafayette and taken in a U-haul truck
8 with no refrigeration, no heat control, very
9 unsanitary conditions, and that was being taken out
10 to the outlying centers. There you have another
11 health problem. What is going to happen from these
12 people eating food that hasn't been properly
13 handled from the time it was prepared? Sometimes
14 it was as much as three or four hours before that
15 food was consumed by the people in the centers.
16 There is still a lack of coordination
17 between the city officials and the federal
18 officials on what should be done and what is next.
19 Just today I heard on the news this morning that
20 there is a difference of opinion. The mayor really
21 wants to get the city back up and running. He
22 wants at least half of the population back in
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1 within a short period of time.
2 There are major parts of the city that
3 still do not have electricity or running water,
4 clean running water, potable running water. There
5 is no infrastructure. The joint commission of
6 healthcare organizations has stated that there is
7 no New Orleans hospital infrastructure right now.
8 It is gone. It doesn't exist. There are one or
9 two hospitals operating but they have minimal
10 staff. There is no 911 situation. How do you send
11 a population back in to pick up and start over
12 again when you don't have grocery stores that are
13 open? You don't have pharmacies that can give
14 drugs? It is just not there. So, it needs to go
15 much, much, much slower.
16 There is just a lot of disappointment in
17 what happened. Do you remember 9/11? Do you
18 remember when this group got together and we talked
19 about what would be the actions taken if we had
20 another terrorist attack? Katrina was not a
21 terrorist attack; it was an attack by Mother
22 Nature. But some of those same plans could have
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1 been put to use. We still have a lot of work to do
2 and I would hope that this group could be involved
3 in any emergency planning process for the future.
4 The healthcare, the access to blood and blood
5 products, the access to physicians, access to
6 hospitals is absolutely imperative in a disaster of
7 this type.
8 I know you have all been inundated where
9 you live with the accounts of what is happening in
10 that area, in the affected area. Let me tell you,
11 you are only seeing a microcosm of what is
12 happening. I also distributed to you an eyewitness
13 account of a friend of mine from White Charles who
14 went down later and was able to go in and help
15 rescue people and it shows you all the stumbling
16 blocks that even this just one person came across,
17 and they were with a group as well. It is sad. It
18 shouldn't happen. And I am hoping that if nothing
19 else comes out of it, in the future, the next time
20 North Carolina or Florida or Mississippi or
21 Louisiana get hit with anything close to this
22 immenseness, there are better plans in place to
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1 help. Any questions?
2 DR. BRECHER: Thank you, Jan. I think we
3 all appreciate what happened there and what it is
4 like to go through that. I am personally from
5 North Carolina so I know what the hurricanes are
6 like. We are going to move on to Miss Tamie
7 Joeckel, I hope I said that right, ASD Healthcare.
8 ASD Healthcare
9 MS. JOECKEL: Lack of planning, lack of
10 timely response, lack of coordination--interesting,
11 that is what happened with Katrina and I guess what
12 I am here to talk to you about, and be a little bit
13 redundant, are the issues surrounding IVIG and
14 access to care. I don't have a presentation to
15 project, I just have the speech. However, I think
16 all of you received a copy of a rather long
17 Power-Point presentation that I prepared, but I am
18 not going to bore you going through all of that.
19 Thank you for giving us the time to speak
20 to you about the issues with IVIG reimbursement. I
21 am Tamie Joeckel, from ASD Healthcare. For those
22 of you not familiar with ASD, we are a Dallas,
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1 Texas-based division of AmerisourceBergen that
2 specializes in the distribution of blood
3 derivatives, especially pharmaceuticals.
4 AmerisourceBergen is a publicly traded Fortune--we
5 are number 23 on the Fortune 100, one of the
6 largest drug distributors in the country, employing
7 over 14,000 people.
8 ASD distributes about a third of the
9 United States supply of blood derivative products.
10 We serve over 4,000 providers of this life-changing
11 therapy. Our customer base encompasses physician
12 offices, home care providers. We are the
13 Department of Defense provider of specialty
14 pharmaceuticals; hospital inpatient and hospital
15 outpatient providers. Our providers serve primary
16 immunodeficiency patients, neurology and
17 autoimmune-deficient patients.
18 We are deeply committed to ensuring that
19 the highest level of patient care is available to
20 all patients at their choice as far as site of
21 care, and we have had a lot of conversation today
22 and there has been a lot of allusions to the
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1 distributor community. Well, we are the
2 distributor community and we would be happy to work
3 with any of you to gather any level of data that
4 you need to evaluate this crisis that is happening
5 in our industry.
6 We do ask for your assistance once again
7 in helping us convey and urgent message to CMS
8 about this issue related to both patient care and
9 quality of life. We ask that CMS reevaluate the
10 impact of both the Part B and the January, 2006
11 Medicare reimbursement changes that are related to
12 IVIG. It is not just the cost of the drug; it is
13 the cost of the services reimbursement that needs
14 to be reevaluated as well.
15 Currently, Medicare reimbursement rates
16 and the required infusion services have
17 dramatically changed the landscape of our industry
18 and our patient access to care. Because the
19 reimbursement rates by Part B do not cover the
20 actual costs of the drug or services physicians and
21 home care providers have been forced to shift
22 Medicare patients to the hospital outpatient
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1 setting. I receive those calls every day. For a
2 long time I only received calls from providers. I
3 am now receiving calls--as a distributor, I receive
4 calls from patients and, obviously, it is a
5 violation of HIPPA that I even engage in those
6 conversations but, you know, the issue has
7 escalated to the level that we have the patients
8 themselves calling us, begging us to help them
9 continue to receive their care in a physician
11 We feel that the quality of care
12 accessible by Medicare patients has significantly
13 eroded, and it is going to continue on this
14 downward spiral if we don't do something about it.
15 To make matters worse, the redirection of patients
16 into the hospital outpatient setting has caused
17 supply issues. Hospitals traditionally contract
18 with manufacturers for pre-established allocations
19 of IVIG based upon their historical demand. This
20 new, unplanned drain on their supplies has caused
21 considerable issues with access to the drug.
22 While we feel that some of the supply
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1 issues will self-correct because manufacturers are
2 increasing their production of the drug, the
3 reimbursement rate deficit between what the therapy
4 costs versus what they are reimbursed remains an
5 issue. So, we feel that that redirection of
6 patients into the hospital outpatient setting, in
7 the hospital setting, is going to continue.
8 Infusing IVIG is a complex undertaking.
9 Conversations that we have with our physician
10 providers speak to the unplanned incidence of
11 life-threatening adverse events. You have to have
12 medical supervision throughout an infusion, and an
13 infusion can be, as earlier referenced, as short as
14 two to three hours but as long as eight hours,
15 depending upon the patient, depending upon the
16 drug. Reimbursement rates have to cover those
18 I know that the IDF--Marsha spoke to you
19 about some of the surveys that they did. I
20 received some information from Dr. Orange about an
21 IDF survey that they did of 1,070 patients as it
22 related to adverse events. It found that 61
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1 percent of patients have infusion rate-related
2 adverse events and 44 percent have had serious
3 adverse events. Unfortunately, the incidence of
4 these adverse events is not predictable. The IDF
5 survey also found that 34 percent of adverse events
6 occurred during the first infusion with a new
7 product, but the remainder occurred in patients who
8 previously tolerated that particular brand of IGIV.
9 I think that speaks a little bit to Julie's point
10 about possibly looking at un-bundling the
11 reimbursement and having and NDC-specific
12 reimbursement rate.
13 But today we know that reimbursement rates
14 are dictating where Medicare patients receive
15 therapy. Patient migration from a nurse- or
16 physician-supervised home therapy and physician
17 office therapy to the hospital outpatient settings
18 has the potential to increase adverse event risks
19 to patients. Prior to the implementation of the
20 Medicare Modernization Act, according to IDF, about
21 30 percent of the PID patients relied on hospital
22 outpatient facilities and, you know, anywhere from
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1 60-70 percent were actually--I think Marsha used 67
2 percent--were actually receiving their infusion in
3 a physician office. Since the implementation of
4 MMA, we know that that number is reportedly
5 increased due, at least in part, to the fact that
6 the cost of the drug and the services are not being
7 covered by reimbursement.
8 When you look through the primer--and I
9 kind of have that as an IVIG primer to talk to you
10 about some of the distribution and some of the
11 manufacturing costs--the economics of IVIG, there
12 are some physician testimonials in there that talk
13 to the point of how they, in fact, have had to stop
14 treating Medicare patients. Some of them are not
15 for-profit; some of them are for-profit physician
16 offices. But even the non-profit providers have
17 basically said they have had to use a financial
18 model to establish how many Medicare patients their
19 practice or their cost and overhead can absorb.
20 So, they kind of have an allocation of we can only
21 have X number of Medicare patients, and they have
22 to turn away and redirect the balance of those.
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1 We have to get the message that CMS has to
2 prevent the elimination or the restriction of
3 access to care, to all of these other sites of
4 care--home care, physician office inclusive. It is
5 our belief that CMS has the authority and
6 flexibility to address the existing reimbursement
7 problems that are going to continue to escalate,
8 especially if the proposed HOPPS reimbursement
9 rates are implemented.
10 We know that CMS has taken the latitude
11 and has worked with other industries to help carve
12 out their drugs to change reimbursement rates, and
13 we hope that IVIG is going to be able to obtain
14 that same latitude.
15 I had the unfortunate personal experience
16 of witnessing a patient being turned away.
17 Unfortunately, I was at the multiple sclerosis
18 research and treatment center in New York and,
19 basically, that particular practice had reached
20 their quota. This was not a PID patient. It was
21 an off-label indication that was being treated, but
22 the woman was sobbing and had basically indicated
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1 that since she had been receiving the IVIG it meant
2 the difference between her being wheelchair bound
3 versus being able to walk, albeit with the
4 assistance of a walker. But that mobility was
5 going to be lost if she did not receive that
7 I know that there has been a lot of
8 discussion about off-label indications. We have
9 been doing a little bit of a survey of our own for
10 some of the patients and would volunteer that we
11 would be happy to assist you in helping obtain some
12 of that data but, you know, at what point does
13 Medicare insurance reimbursement dictate whether a
14 treatment is medically necessary if it improves, in
15 fact, the quality of life of a patient?
16 All of these patients deserve treatment,
17 and they deserve to choose their site of care. So,
18 we ask once again that this committee help us
19 convey the sense of urgency to CMS. Thank you for
20 your past efforts and, again, we don't want it to
21 be lack of planning, lack of timely response and
22 lack of coordination that prevents us from
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1 addressing this very important issue. Thank you.
2 Are there any questions?
3 DR. BRECHER: Questions? Comments?
5 DR. SAYERS: Thanks. Can I ask you a
6 question about some of the information you have in
7 this booklet?
8 MS. JOECKEL: Yes.
9 DR. SAYERS: There really is some valuable
10 news here. One of the illustrations though speaks
11 to the expense associated with testing for
12 hepatitis D. What did you mean by excessive
13 production waste driving up the price of IVIG?
14 MS. JOECKEL: Well, again, I am not the
15 expert and this is information that we use to
16 illustrate the fact that we know that there has
17 been, for instance, with recombinant factor demand
18 versus plasma demand for these other products that
19 are made from a liter of plasma. You know, the
20 manufacturer has to recover those manufacturing
21 costs somewhere. I know there were a lot of
22 questions about why is the cost of IVIG continuing
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1 to go up, and why the ASP look-back period
2 sometimes--you know, 90 days may not be sufficient
3 because the market is dynamic. It is changing and
4 it is changing rapidly. These are businesses after
5 all. They have to cover their overhead.
6 I happen to be a CPA who runs a sales
7 organization, but I understand PNLs and I
8 understand the fact that you have direct and
9 indirect costs of manufacturing. You have to be
10 able to cover those costs. If your byproducts or
11 the finished goods that you are manufacturing--and
12 in this case a liter of plasma and there are
13 multiple finished goods that are derived from that
14 and if all of a sudden the demand for one of those
15 finished goods start diminishing you have to recoup
16 those costs somewhere.
17 DR. BRECHER: If there are no other
18 questions or comments, thank you. Are there any
19 other comments from the public?
20 Immune Deficiency Foundation
21 MS. VOGEL: Hi, I am Michelle Vogel, from
22 the Immune Deficiency Foundation. First, I want to
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1 echo Marsha Boyle by commending this committee for
2 its continued support to improve access to IVIG. I
3 would like to underscore IDF's data on the switch
4 and location for treatment for patients. Prior to
5 January 1, 51 percent of these patients were being
6 treated in physicians' offices and 17 percent were
7 in the hospitals. Now only 9 percent are in the
8 physicians' offices and 49 percent are in the
9 hospitals. These numbers are increasing every day
10 because the physicians and the home care companies
11 that had been holding onto the patients, hoping to
12 see the reimbursement rates increase are not seeing
13 those numbers and are trying to transfer them at
14 this point. But hospitals at this point are
15 over-burdened, and either they do not have enough
16 IVIG, they don't have enough staff to administer
17 it, or the facilities or personnel aren't qualified
18 to administer IVIG, which is leading to waiting
19 periods and denial of coverage or care. This
20 includes the unlabeled patients and the primary
21 immune deficient patients. We get calls every day
22 that a patient is being put on waiting lists of up
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1 to six months. They can't wait for six months to
2 get product.
3 This is under the current reimbursement
4 rate. When the rates drop in the hospitals--I
5 mean, the hospitals are being reimbursed at $80.68
6 and can't take care of these patients. When they
7 drop to match the physician's office I don't know
8 what is going to happen to these patients.
9 I know your recommendation for a public
10 health emergency was controversial, but I applaud
11 you for trying to do the right thing for patients
12 and make sure that they receive the life-saving
13 therapy and the right site of care. I think many
14 members of Congress have joined in your efforts,
15 not only with that one letter that had over 30
16 signatures but phone calls and individual letters
17 going in.
18 I can't tell you how many letters we are
19 seeing from individual patients going to CMS with
20 phone calls and getting feedback saying call 1-800
21 Medicare. Marsha said this but I have to reinforce
22 this. They are saying, well, if your doctor won't
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1 treat you, find another doctor that will. There
2 aren't any. Saying to the members of Congress we
3 will have the patient go to the hospital, they
4 can't. There is not enough product in the
5 hospitals to treat these patients or there are not
6 enough people to administer it.
7 So, this is just going to escalate on
8 January 1. I think it is important for this
9 committee today to continue to show its concern
10 over the growing problem and the catastrophic
11 outcome pending if the hospital reimbursement drops
12 to the same rates as the non-hospital provider
13 settings. I know you guys have taken a lot of heat
14 for your recommendations. But I really, really
15 think it is important for you to continue, and I am
16 not saying coming out with another public health
17 emergency but making a statement showing your
18 growing concern that access is continuing to be an
19 issue and that we are going to have a serious
20 problem come January 1 if the HOPPS rates go
21 forward that Julie Birkhofer showed you on that
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1 We have proposed some solutions and the
2 whole group has come together with those solutions.
3 If CMS doesn't accept those solutions we are in
4 trouble. These patients are in trouble.
5 Therefore, I am requesting that this committee
6 sends a letter to Secretary Leavitt regarding your
7 continued concern, as well as the need to keep the
8 hospital reimbursement for IVIG as stable as
9 possible by not dropping to the level of Medicare
10 Part B or ASP plus 8 percent. Thank you.
11 DR. BRECHER: Comments? Questions? Yes?
12 Advanced Medical Technology Association
13 MS. LEE: Hi, good morning. My name is
14 Theresa Lee, I am with the Advanced Medical
15 Technology Association, representing our blood
16 products and technology sector. My member
17 companies manufacture a wide variety of blood
18 products that screen and process blood.
19 This morning's discussion on IVIG
20 reimbursement has highlighted, at least for me, the
21 significant impact that Medicare reimbursement has
22 on patient access and the availability of blood and
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1 blood products overall. In that vein, my members
2 continue to have significant concerns about overall
3 Medicare reimbursement for blood and blood
4 products, and we have been working in coalition
5 with the American Association of Blood Banks, the
6 American Red Cross and America's Blood Centers in
7 pursuing appropriate reimbursement for blood and
8 blood products.
9 Dr. Holmberg mentioned several recently
10 published Medicare payment regulations either in
11 proposed or final form at this juncture. I would
12 like to bring just three developments to your
13 attention in those regulations.
14 First, I would note that in the inpatient
15 final regulation the Medicare program rolled blood
16 and blood products, which had previously been a
17 separate category, into sort of a catch-all
18 category of miscellaneous items. Previously, you
19 may recall, blood and blood products had been
20 attached as an index to blood derivatives, and I
21 think some of the fluctuations in the plasma
22 derivatives market caused blood reimbursement to
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1 decline in that context. Now they have attached it
2 to a separate producer price index that is
3 completely unassociated with blood and the concern
4 is that fluctuations in that index could lead to
5 further cuts. I wanted to just bring it to your
7 It also highlights the fact that we need
8 to stay on top of the issues related to blood
9 reimbursement, particularly in the inpatient
10 setting where, as I understand it, over 80-90
11 percent of all blood and blood products are used.
12 Second, I would note that in the
13 outpatient proposed rule the Medicare program has
14 proposed to cut leukoreduced red blood cells by
15 approximately 10 percent. I would note that the
16 APC advisory panel, which is an advisory panel that
17 specifically advises CMS on outpatient
18 reimbursement, has proposed that CMS freeze blood
19 and blood product payment at 2005 levels. As I
20 understand it, the American Red Cross and AABB and
21 America's Blood Centers are also behind that
22 recommendation, and I hope that this committee
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1 would support that recommendation to have payment
2 levels frozen.
3 Finally, I would like to thank CMS and
4 this committee for issuing transmittal 496 which
5 has attempted to provide additional consolidation
6 clarification in blood reimbursement guidance to
7 hospitals and billers and coders nationwide. I
8 would note that we are working in coalition with
9 ABC, AABB and the Red Cross to provide some
10 additional recommendations to refine that guidance
11 and further clarify the regulations. Thank you
12 very much for your time.
13 Committee Discussion
14 DR. BRECHER: Thank you. Any additional
15 public comments? If not, the committee will go
16 into a discussion period regarding the morning
17 presentations. Before we begin, I want to stress
18 that I think it is clear that HHS has heard the
19 message about IVIG. They are continuing to monitor
20 the situation. I don't want to speak for CMS, but
21 I think that they are also hearing the message.
22 So, comments? Questions? Proposals?
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1 I guess one question is does the committee
2 need to send another message to the Assistant
3 Secretary and the Secretary, or has the message
4 already been delivered? Jay?
5 DR. EPSTEIN: I can't answer your second
6 question. I think the committee might have to
7 discuss that a bit. I guess my take on what is
8 going on is that the problem hasn't been solved. I
9 think what we have heard is that patients are
10 continuing to suffer the kinds of disruptions in
11 care that were described to us months ago and,
12 although there has been movement at CMS to update
13 the reimbursement schedule, there are underlying
14 problems that remain to be solved.
15 I guess one question in my mind is how one
16 might react to the consensus proposal that was
17 brought forward by the PPTA. I personally do not
18 feel sufficiently expert--in fact, I am totally
19 ignorant--to understand how these might help the
20 situation, but it does strike me that if a
21 thoughtful group got together and looked from a
22 collective standpoint among stakeholders on how to
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1 make things better, that these suggestions warrant
2 some consideration.
3 DR. BRECHER: Karen?
4 MS. LIPTON: I agree with Jay. I don't
5 feel competent myself to evaluate the proposals. I
6 think we do need to send a message that the issue,
7 even though they are taking steps, isn't resolved
8 and perhaps we could specifically request that they
9 sit down and look at some of the proposals that
10 have been put forward. I think there is something
11 going on that is a lot bigger. And, I do think it
12 was very interesting, looking at the ASD, and I was
13 trying to run through it very quickly while she was
14 speaking, but it does appear to me that we are also
15 seeing a shift in manufacturing and I don't totally
16 understand how switching the recombinant is
17 affecting all of this, but I suspect that we are
18 stuck in a place perhaps where the model and the
19 return for these companies is shifting dramatically
20 and we don't understand how that is affecting both
21 the reimbursement policies and the effect on
22 patient accessibility to these products. But I
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1 think it is something that we need to pay attention
2 to, and I think in looking at the reimbursement
3 they really do need to go deeper and look at how
4 the market is shifting.
5 DR. BRECHER: Celso?
6 DR. BIANCO: Well, I want to support Jay
7 and Karen and say that we should send a message or
8 at least a reminder that this is unresolved.
9 DR. BRECHER: It sounds like that, at a
10 minimum, what we are going to do is at least say
11 that the problem is ongoing and requires further
12 attention and consideration of other solutions,
13 such as perhaps what PPTA has suggested. Do we
14 want to draft that at this time, or do we want to
15 save the draft wording until tomorrow? Tomorrow?
16 Okay. So, why don't we take a break now, a
17 15-minute break?
18 [Brief recess]
19 DR. BRECHER: We are going to resume if
20 everyone will take their seats. We are now going
21 to move on to a strategic plan for improving blood
22 safety in the 21st century. This is in some ways a
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1 continuation of topics covered in our last two
2 meetings. We will start with a subcommittee report
3 from Jeanne Linden.
4 Strategic Plan for Improving Blood Safety
5 in the 21st Century
6 Report of Subcommittee Activity
7 DR. LINDEN: If you recall from the
8 previous meeting, the subcommittee had been
9 established to look at infectious risks--
10 DR. BRECHER: If everyone in the back
11 could, please, sit down and be quiet so we can hear
12 the speaker? Thank you.
13 DR. LINDEN: The subcommittee was also
14 tasked with looking at some of the issues about
15 risk reduction in blood safety and availability
16 that had broadly been discussed by this committee
17 on several different occasions at different
18 meetings. The subcommittee looked at these issues
19 and pondered discussions of would it be most
20 productive to write sort of a report; what sorts of
21 actions could we take given the resources that we
22 have? It was thought that really what we needed
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1 was a strategic plan that would supplement the
2 existing FDA blood action plan that has been in
3 existence for several years to be more current, and
4 specifically address s some of the issues that had
5 arisen, both in the area of infectious diseases,
6 both in known pathogens and unknown agents that may
7 be emerging, as well as some of the non-infectious
8 risks which continue to be out there.
9 We took the basic issues that had come up
10 before and looked at eight different issues that
11 had been identified. One, the need for a
12 structured, open and transparent process for policy
13 and decision-making; the integration of the blood
14 system in the public health infrastructure; the
15 surveillance of adverse events related to blood
16 transfusion and blood donation, including the known
17 infectious diseases, the unknown or emerging
18 infectious diseases, as well as non-infectious
19 adverse reactions. A question for this was should
20 focus on blood only also include tissues, organs,
21 HPCs and coordination of risk communication to be
22 effective, accurate and timely; error prevention
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1 and other non-infectious risks and, in terms of
2 blood availability, donor recruitment and retention
3 issues and coordination of those. Also, clinical
4 practice standards to address the judicious use
5 and, therefore, availability issues, as well as,
6 obviously, decreasing risks if people are not
7 transfused as much. Also, the importance or a
8 research agenda to address a variety of relevant
9 issues, including measuring outcomes of any
10 strategies that are taken to address risks. Also,
11 disaster planning and what further efforts could
12 supplement the existing task force.
13 What we did was take these eight items and
14 each member of the subcommittee was tasked with
15 specifically reviewing those particular
16 subjects--some of the things we learned; some of
17 the things we might already know from other
18 sources; some of the issues and questions that have
19 come up. So, the idea of what we wanted to do
20 today is recommend that the committee consider a
21 recommendation of putting together a strategic plan
22 and considering what elements might be in that
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1 plan, and who would be involved, and what role HHS
2 could play in the committee possibly. So, that is
3 what we sort of wanted to put on the table.
4 Some of the members are going to be making
5 very brief presentations of the issues, posing some
6 questions which are not intended to be answered at
7 this committee or we would be here for weeks, but
8 really just to provoke some thought and discussion
9 for consideration in the overall scheme of what we
10 are talking about. So, our thought was, with the
11 Chair's permission, to take questions on the
12 presentation for, say, something that wasn't
13 understood without getting into discussion at this
14 time of the individual presentations. So, that was
15 our recommendation to the committee.
16 DR. BRECHER: Thank you, Jeanne. First we
17 will go to Jerry Holmberg.
18 Review of January and February 2005 Meetings
19 DR. HOLMBERG: My task on this was to go
20 back through and try to identify and review for you
21 the activities of the last couple of meetings. But
22 I do want to raise the questions that the
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1 subcommittee has put together to address this
2 issue. We will be discussing these at the very end
3 but I wanted you to start thinking about these
5 Does the committee believe that there is a
6 need for the Department to develop a strategic plan
7 for detecting and preventing
8 transfusion-transmitted complications? That
9 includes both infectious and non-infectious
10 complications in the 21st century.
11 If a strategic plan is recommended by the
12 committee, what scope of issues does the committee
13 believe that the plan should address, and what role
14 should the ACBSA and its subcommittees play in the
15 development of the strategic plan?
16 Jeanne already mentioned the HHS blood
17 plan that has been in effect for several years, and
18 one of the things that the strategic plan has
19 really helped was to really pave a path for future
20 direction. So, if we go back and even look at the
21 HHS strategic plan that was first initiated by the
22 Food and Drug Administration and then taken on by
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1 the HHS, you can see that many of those things have
2 been accomplished. I did put that in your
3 handouts, to take a look at that because, by no
4 means, I don't think what we want to do is to take
5 away from what has already been done but I think it
6 has come to a point where we need to look, for the
7 future, where do we move from here.
8 I just want to go pack to August of 2004,
9 over a year ago, and I know that we have on the
10 committee several people that have experience now
11 with the transfusion-
12 related acute lung injury and we did talk about
13 TRALI at that time; the implementation of clinical
14 education; the model for impact of deferral on
15 screening interventions and the research that may
16 come along with that. So, I bring that out and I
17 think that Dr. Bracey will talk a little more about
18 that in his presentation of clinical outcomes and
19 then maybe also in the research and this may be
20 discussed also.
21 The response from the Secretary was to
22 continue to monitor progress of the scientific
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1 community. Some of the action that has already
2 been taken is that the National Heart, Lung and
3 Blood Institute has moved TRALI to a top priority
4 of all non-infectious transfusion complications and
5 there are two institution-supported investigations
6 that are currently being pursued.
7 The other recommendation from August, 2004
8 was access to treatment for individuals with rare
9 bleeding disorders. From there, we have actually
10 looked at some of the research. We have also
11 developed a workshop. Not only did FDA develop a
12 workshop, HHS helped support it in trying to
13 determine what kind of pathways needed to be put
14 into place and then what kind of new products
15 needed to be out there. Then, of course, the
16 reimbursement issue.
17 The recommendations are being considered.
18 As mentioned numerous times last year, just before
19 Secretary Thompson left his position he did sign
20 off on his medical innovation process and each one
21 of the agencies has a part in this medical
22 innovation process. For instance, the FDA has the
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1 Critical Pathway and also, as I mentioned, we did
2 have a workshop put on by the FDA on this issue of
3 rare blood disorders.
4 In 2004 we also looked at bacterial
5 detection in plasma concentrations and seven-day
6 platelets. I don't really think we need to spend
7 much time on that. We have seen progress over the
8 last year and it was good to hear today that the
9 New York Blood Center is moving forward with this.
10 The recommendations on platelet detection
11 were that--of course, the Secretary's response was
12 that recommendations are being considered. The FDA
13 innovative regulatory pathway allowed collection of
14 post-approval information on the QC data, and they
15 modified the field study. AABB task force put
16 together two guidance documents and also put
17 together a survey, which Dr. Brecher is one of the
18 primary authors on that will be considered for
19 transfusion. I think it was very enlightening,
20 that survey, to see the impact of this and also
21 some business model changes that took place within
22 the blood field. Then, of course, we had activity
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1 with the manufacturers.
2 One of the things too in August, just to
3 reflect back, in August, 2004 we talked about the
4 minipool nucleic acid test for blood donor testing.
5 This was a topic that was discussed at BPAC. It
6 was discussed at our internal blood safety
7 committee, and then also the Acting Assistant
8 Secretary referred this to the Advisory Committee
9 for Blood Safety and Availability.
10 The recommendation or the actions that
11 took place, as I already mentioned, were discussed
12 at the various advisory committees and finally the
13 Blood Safety Committee concurred with the FDA
14 policy and made the recommendation that came out
15 that current data do not support a recommendation
16 for routine use of the Roche molecular system
17 minipool NAT to screen blood donors and plasma
18 donors. Existing donor tests appear to be adequate
19 and the new test appears to provide very limited
20 public health benefit at this time. However,
21 public health officials will reconsider possible
22 recommendations for routine donor screening for HBV
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1 by nucleic acid tests based on experience with
2 voluntary use of the test, further technology
3 developments, and any other factors that might
4 affect the public health benefits expected from
5 such testing.
6 In January of 2005 we had our meeting
7 where we talked about different issues. Topic one
8 was the bacterial detection and the progress
9 reports on seven-day platelets; the reimbursement
10 issues associated with plasma and recombinant
11 analog therapy. Then we started our discussion
12 which the subcommittee are really going to be
13 presenting today, and that is the current and
14 emerging infectious pathogens, sharpening our
15 approach to the 21st century to reduce the risk of
16 transfusion-transmitted diseases. As you can see
17 from that topic and from how Jeanne has already
18 introduced today's discussion, there has been some
19 evolution in our thinking and, hopefully, that will
20 come out today in some of the discussion. I will
21 quickly go over this. I really don't think we need
22 to spend time on the bacterial issue again.
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1 Reimbursement issues--I just want to say
2 that although at the last meeting the Secretary did
3 not respond to the recommendation, it was picked up
4 on and incorporated into the response that Dr.
5 Brecher got this summer and also some of the
6 comments that were referred to this morning by Ms.
7 Lee as far as the transmittal 496. A lot of that
8 was rolled into some of the endeavors that we were
9 working on.
10 Let me just quickly go through some of the
11 discussion that we had back in January on the
12 current and emerging infectious pathogens. We
13 looked at the IOM report on microbial threats to
14 health. This has been a very good guiding document
15 for a lot of us. I think that what we have seen
16 even over the last nine months has been that some
17 of the comments made in the IOM report as far as
18 the transmission of diseases have really magnified
19 or come to light, and that is that one of the
20 things that the IOM report talks about is natural
22 Since then we have had the tsunami and we
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1 have also had hurricane Katrina. We have to
2 constantly be thinking about how can some of these
3 natural disasters affect the way we do business.
4 Most recently, the AABB Transfusion-Transmitted
5 Disease Committee was even considering a voluntary
6 deferral for those people that were in shelters to
7 reduce the risk of hepatitis A virus. But I just
8 brought that out, that we need to keep going back
9 to the IOM report and take a look at some of those
10 recommendations. There is a lot of good
11 information in there.
12 We looked at an overview of current
13 blood-borne threats systems approach and we did a
14 case study of various disease entities and how did
15 we respond. I think everybody recognizes that for
16 the West Nile virus the stars were aligned and I
17 think that is one of the models that we really did
18 well. There have been papers written on it that
19 really explain some of the progress that was made.
20 But some of the issues, like with Chagas disease,
21 are still an unmet challenge; some of the HIV, the
22 evolving changes, HHV-8 still is unresolved; and
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1 also the vCJD is a good example of risk
2 communication. So, we had a presentation of model
3 responses, unmet challenges, evolving challenges,
4 unresolved scientific evidence and risk
6 The IOM report, just to highlight some of
7 the things in the IOM report, talked about
8 enhancing global response capacity; rebuilding
9 domestic public health capacity; improving domestic
10 surveillance through better disease reporting;
11 explore innovative systems of surveillance; develop
12 and use diagnostics; educate and train the
13 microbial threat work force; develop vaccines and
14 production capacity; appropriate use of
15 antimicrobial drugs and new antimicrobial drugs;
16 vector-borne and zoonotic disease control;
17 comprehensive ID research agenda; and
18 interdisciplinary ID centers.
19 With that, at the end of that meeting we
20 sort of had a direction that we were looking at,
21 and that is that for future discussion we wanted to
22 look at surveillance, appropriate research, product
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1 development, global information sharing,
2 transparency in policy process, and also risk
4 Just to show you some of the progress that
5 we have made, I think sometimes as a committee we
6 all--and I will use the collective "we"--we don't
7 realize the progress that we make. We make one set
8 of recommendations and move on and, at the same
9 time, government is still working in the background
10 so you don't see the impact of some of your
11 recommendations until much later. But this was a
12 request to CMS that talked about some of the
13 problems with reimbursement and plasma and
14 recombinant issues, and also some of the language
15 that was used within the MMA that needed to be
17 I know this is hard for you to see but I
18 did incorporate this in your package. On May
19 13--and, unfortunately, I didn't get this before
20 the last meeting so I didn't have it to share with
21 you, but this was a response from Dr. McClellan,
22 thanking Dr. Beato for bringing a lot of these
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1 issues to his attention and also, as you have
2 already heard from Ms. Lee this morning, the CMS
3 manual, the transmittal 496 which explains to
4 hospitals how to charge, and it incorporated some
5 of the corrections in some of the terminology. So,
6 we have made progress, and there is still more
7 progress that we need to make.
8 As I mentioned before, we identified these
9 issues as far as the different aspects of
10 surveillance, appropriate research, product
11 development, global information, transparency in
12 policy, and risk communication, and at the May
13 meeting we looked at approaches to reducing the
14 risk. We had Dr. Scwhartz, who talked about the
15 pandemic action plan, and he did a very good job.
16 At that point of his discussion the federal
17 government was still in a draft mode and I
18 understand that at the beginning of August the
19 draft action plan was submitted to the Secretary.
20 We also had discussions from various
21 public health officials, state and local. We had
22 discussions from the National Association of County
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1 and City Health Officials, Association of State and
2 Territorial Health Officials, and the Council of
3 State and Territorial Epidemiologists. All three
4 of these groups came and talked to us. I think
5 that what we gleaned out of that discussion was
6 that there was a need for active communication and
7 that one of the things that the IOM report brought
8 out was that we have a very fragile grassroots
9 public health system. I think that is a problem
10 that has been mentioned over and over again in a
11 lot of the literature, but the discussions with
12 these three groups really brought to mind that we
13 really need to have active dialogue with them.
14 We also looked at various models of
15 disease reporting and adverse event surveillance.
16 We had the hospital epidemiology surveillance
17 system from CDC and also the hemophilia treatment
18 center database. They have some very unique ways
19 of tracking all of the patients that receive
20 products within the hemophilia treatment centers.
21 We also had some discussion on orphan test
22 development. Some of the other organisms we talked
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1 about with Chagas and malaria and different
2 organisms--how could we move forward and
3 develop--instead of an orphan drug test or orphan
4 drug, is there a way that we could foster the
5 orphan test development?
6 Then, one of the things that I know Dr.
7 Beato appreciated very much was that the committee
8 did not rush into a recommendation. One of the
9 things that Dr. Beato has said numerous times is
10 where is the evidence to backup the
11 recommendations? And, has this been given adequate
12 thought? So, I really do appreciate that the
13 recommendations were tabled until the committee
14 could further discuss and concur on something to
15 put forward to the Secretary.
16 So, once again, I just come back to the
17 questions that I would like you to consider at the
18 end of our discussions over the next couple of
19 days: Does the committee believe there is a need
20 for the Department to develop a strategic plan for
21 detecting and preventing transfusion-transmitted
22 complications in the 21st century?
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1 If a strategic plan is recommended by the
2 committee, what scope of issues does the committee
3 believe that the plan should address, and what role
4 should the ACBSA and its subcommittees play in the
5 development of this strategic plan? Thank you.
6 DR. BRECHER: We have time for a couple of
7 content questions, if there are any. If not, we
8 will move on to the second speaker, Jay Epstein,
9 talking about a structured process for policy and
11 Structured Process for Policy and Decision-Making
12 DR. EPSTEIN: Thank you very much, Mark.
13 As your agenda shows and as Jeanne Linden
14 suggested, the subcommittee on EIDs considered the
15 question of whether there ought to be a
16 recommendation in favor of developing a new
17 strategic plan for blood safety and availability.
18 A set of elements was posed which are reflected in
19 a set of introductory talks, of which this is the
21 So, the first element of a candidate plan
22 is a structured process for policy and
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1 decision-making. Let me start by suggesting that
2 effective action depends on making good decisions,
3 and this leads to the idea that one ought to review
4 the decision-making process itself to figure out
5 whether it has characteristics that would lead to
6 making good decisions.
7 The rationale for this is that ensuring an
8 adequate supply of safe blood is an essential
9 national responsibility that requires support at
10 the national level. Additionally, the cost,
11 complexity and evolution of the blood system
12 necessitate an ongoing process of decision-making
13 in order to set priorities and to address newly
14 recognized and emerging risks.
15 Additionally, the structured process can
16 foster better public health outcomes by promoting
17 the integration of scientific, economic and social
18 factors into the decisions while, at the same time,
19 enhancing their general acceptance.
20 Now, we did hear a presentation at the
21 January, 2005 committee meeting on the elements of
22 a good policy process based on work from expert
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1 groups. Without, of course, the ability to go into
2 this in any detail, I am simply going to hit the
3 high points.
4 The experts in this field have suggested
5 that elements of a good policy process include an
6 outcome orientation based on a needs assessment; at
7 least within a democracy, a clear and open
8 decisional process of procedure; the development of
9 robust scientific evidence to support selected
10 policies and actions; the efficient use of both
11 human and financial resources; active engagement of
12 stakeholders as partners; and clear communication
13 of risks and benefits, including their
15 Now, within that framework there is also a
16 concept that a structured process can lead both to
17 better decisions and better acceptance and
18 awareness of those decisions. These essentially
19 are formal tools. We call them assessment tools
20 that can be used to analyze the feasibility, the
21 likely benefit, the projected cost, the risks and
22 tradeoffs, equity, sustainability and timeliness of
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1 these actions, and the use of these tools then
2 plans a role in a cyclical process of assessment,
3 action and reassessment that works more or less in
4 the following way:
5 One comes upon a situation. The first
6 step is to analyze the situation. Then one moves
7 to the construction and analysis of policy
8 alternatives, followed by a deliberate choice of
9 preferred options, presumably preferred on a
10 rational basis integrating the data that comes out
11 of these formal assessments. One must then
12 communicate the policy decision so as to encourage
13 not just understanding but also endorsement and
14 active participation. There is then the
15 implementation phase and, in a good quality process
16 that is inevitably accompanied by outcome
17 monitoring which then leads to reevaluation. So,
18 the cycle then repeats itself in essence
20 Once again, the experts in this field
21 would be quick to say that nothing in the real
22 world actually follows this schema; that you may
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1 find yourself concurrently at different phases of
2 this process. But the conceptual model is helpful
3 because it gives you a road map of what you are
4 trying to do as you are in the midst of a problem
5 solving situation.
6 So, within this framework we are proposing
7 for consideration by the committee as issues that
8 might be incorporated in the tasking of a group to
9 develop strategic plan questions of this sort, and
10 these do reflect the characteristics of what I have
11 described as at least an academician's description
12 of a good policy and decision-making process:
13 First, is our national investment in blood
14 safety and availability sufficient to meet its
15 objectives? Second, are our policy and
16 decision-making processes adequately transparent
17 and inclusive? Third, do we utilize analytical
18 tools appropriately in our decision-making?
19 Lastly, are our decisions sufficiently
21 Let me see if there is another slide--yes,
22 additional questions: Can we enhance the
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1 effectiveness of communication of our policies and
2 their rationale, and do we monitor the outcomes of
3 our decisions and actions sufficiently?
4 So then, this, hopefully, will provide the
5 committee with an introduction to what the
6 subcommittee thought about this element, should it
7 become an element of a strategic plan. I am happy
8 to answer any questions or we can just move on.
9 DR. BRECHER: Gerry?
10 DR. SANDLER: Dr. Epstein, in leadership
11 for the last couple of decades the nation hasn't
12 done very badly in terms of a strategic plan for
13 preventing this kind of a complication. Are you,
14 in front of an open mike, in a position to give us
15 your opinion as to whether such a plan would best
16 be accomplished by expanding the resources of the
17 team that you have been working with or whether,
18 for some reason, you think it would be necessary to
19 go external to your office to create such a thing?
20 I know it is a difficult question to answer but it
21 is the one that I would see as pertinent.
22 DR. EPSTEIN: Well, this is a personal
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1 opinion and I am not speaking on behalf of my
2 agency, but my opinion is that we do have
3 structures in place that would permit us to do all
4 of the things that I have described at an even
5 higher level of proficiency, and that it is more a
6 question of putting forward the principles under
7 which we seek to operate in enhancing our ability
8 to do so, in other words, removing encumbrances.
9 But I do think that our structures are adequate to
10 the task. Others may debate this, of course.
11 DR. BRECHER: Karen?
12 MS. LIPTON: Jay, thanks. This is
13 actually a very good presentation to start us off
14 in again thinking about some of these issues. As I
15 look at it, I just wanted to respond that I think
16 what we have been saying around the table is that
17 our national investment isn't sufficient. As you
18 said, we may have the structures in place but we
19 really haven't managed to garner sufficient
20 resources to do what we all think we need to do,
21 both in the government and the private sector.
22 I would answer the second question in a
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1 very positive framework. I think that we are
2 transparent and inclusive, and maybe that is
3 because we have this committee. I think that FDA
4 at the BPAC meeting has been successful in getting
5 the right people to make the decisions, and the
6 entire revamping that went on several years ago of
7 the advisory committee structure I think is
9 The one that I am not as clear about is
10 the analytical tools that we use in
11 decision-making, or at least I am not aware of all
12 of them and how evenly they are used in all of the
13 decisions. Actually, Jay, you may be able to
14 respond to that. From my perspective as a
15 committee member I am just not certain about that.
16 Are decisions sufficiently evidence-based?
17 I think they are when they can be. There are a
18 number of decisions that we sometimes have to make
19 because of maintaining public confidence in the
20 safety of the blood supply and adequacy. That is
21 how I would answer those questions. But, Jay,
22 could you comment on the analytical tools?
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1 DR. EPSTEIN: My feeling is that they
2 could be utilized more. Analytical tools are
3 difficult to use. They generally require gathering
4 and analyzing data, and that always raises an issue
5 of resources. Also, there is the balance between
6 studying problems and doing something about them.
7 And, using these kinds of tools is often also time
8 consuming and unless you planned well in advance
9 you find yourself in a situation where you need to
10 make a decision and you can't wait for that kind of
11 modeling. So, I tend to agree with you--and,
12 again, this is a personal opinion, not an agency
13 opinion--that that is an area where we could do
15 DR. BRECHER: Celso?
16 DR. BIANCO: I just want to reinforce a
17 little bit of what was said. But, Jay, I think the
18 most important question that I feel is number one
19 is are national investments in blood safety and
20 availability sufficient to meet its objectives? I
21 think that we have to define a little bit better
22 the objectives. We talk in a generic sense about
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1 safety and availability but we need to work on
2 that, and that would be part of the work for a
3 strategic plan.
4 The second thing is we have a combination
5 of approaches and groups that participate in the
6 process. There is the private sector of blood
7 collecting agencies, there is the private sector of
8 hospitals which manage the blood administration and
9 utilization, and we have regulatory agencies and
10 government. And you have this somewhat
11 schizophrenic thing in which we have the site of
12 collection being a volunteer site--sacred, white
13 hat, and always depending on the funding that is
14 obtained from the activities that follow blood
15 collection and the difficulty of placing itself
16 within the system. So, I think that we need to
17 expand a little bit that question. But I think
18 this is wonderful, what you just did.
19 DR. BRECHER: Last comment, Merlyn?
20 DR. SAYERS: That was outstanding.
21 Reference was made earlier by Karen to revamping of
22 the FDA's advisory committee, Blood Products
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1 Advisory Committee. I saw that in a slightly
2 different light. It looked to me like a reduction
3 in opportunity for inclusiveness. I was wondering
4 what your opinion was. How does one get around the
5 sense that individuals can do with that specialized
6 knowledge, and by virtue of that knowledge,
7 inevitably find themselves in a conflictive
8 position? And, is it possible to get contributions
9 from those individuals without the decisions being
10 tainted by what might be seen as conflict on the
11 part of those contributions?
12 DR. EPSTEIN: I am not sure that that is
13 really a question for me, Merlyn. You know, how we
14 charter advisory committees is a very delicate
15 matter because the committees have to be free of
16 taints and, at the same time, they have to be
17 sufficiently expert to do their business. As you
18 know from all the orientations you have had to live
19 through, there is a body of regulations that
20 attempts to deal with that inherent tension, and
21 whether there are other ways that we could do
22 business I am not sure. I think one thing that we
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1 do is have workshops where we can bring in experts
2 to speak freely as experts from their various
3 vantage points and try to separate that, as it
4 were, from the policy-making process per se so
5 that, at least at the stage of information
6 gathering and play of ideas, we don't have to worry
7 about who is speaking and why. But I think that
8 this is a very large issue and it has been the
9 subject of many, many deliberations over the years
10 by the Congress, by the agencies, by the IOM, and
11 it is just not a simple one.
12 DR. BRECHER: All right, Karen.
13 MS. LIPTON: Just one quick comment. I
14 think that, yes, the issue is the regulatory
15 structure and I think then it is incumbent upon us
16 to make sure that we participate in the process as
17 fully as we can, you know, giving the information
18 we can to the panel. I also think the workshops
19 are extremely helpful, and I know that that is
20 quite a stress on the staff. Do you feel that you
21 are adequately funded and resourced to do the
22 number of workshops that you would like to see take
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2 DR. EPSTEIN: Well, I think that we would
3 like to be able to do more workshops than we can
4 afford, put it that way. In any given year, we do
5 as many as half a dozen. Generally they are very
6 well received. There is the opportunity also for
7 the industry or other outside parties to sponsor
8 workshops to which FDA and other government
9 agencies will bring participation. I think that if
10 there were more of a shared agenda, it might
11 facilitate the process of finding sponsors,
12 co-sponsors and alternative sponsors. So, we live
13 in a world where we have significant resource
14 limitations and we attempt to leverage out efforts
15 through these co-sponsorships but, certainly, there
16 is room for more but it would require them to step
18 DR. BRECHER: Thank you, Jay. We are now
19 going to move on to integration of the blood system
20 within the public health infrastructure, Judy
22 Integration of the Blood System within
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1 the Public Health Infrastructure
2 DR. ANGELBECK: In considering this topic,
3 integration of the blood system within the public
4 health infrastructure, I certainly went back to
5 documents and talks that we had heard in the past
6 two meetings and reviewed that information an
7 considered the topic not only as one who
8 participates in the private sector of the blood
9 industry, but as a citizen who requires from time
10 to time perhaps healthcare--although I have never
11 required a blood transfusion but may at some point
12 in the future--and tried to understand how best to
13 address this topic.
14 So, what I tried to do here was to provide
15 just an overview strictly by identifying entities
16 that now participate in the current structures.
17 For the oversight of blood safety and availability
18 within the Department of Health and Human Services,
19 of course, there is the advisory committee. There
20 is the U.S. Public Health Service, the CDC, the
21 FDA, the NHLBI, and that is in cooperation with the
22 Department of Defense. Then, in the private sector
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1 is the American Association of Blood Banks,
2 America's Blood Centers, the American Red Cross,
3 the Plasma Association and there are select state
4 health agencies, again back in the government
6 On the public health structure side, as I
7 saw what I reviewed, we are looking at government
8 agencies at various levels, from the United States
9 Public Health Service, the CDC, the FDA at the
10 federal level, state health agencies, territorial
11 health agencies, tribal health agencies, county
12 health departments, city health departments and
13 local health boards. A challenge, from my
14 perspective, to this integration is that the U.S.
15 blood and plasma collection and distribution is a
16 free enterprise network of non-profits and
17 for-profits. They are not governmental agencies.
18 In addition to that, from some of the
19 presentations at the previous meetings, what have
20 we learned about how those two structures interact?
21 9/11 underscored the need for a coordinated message
22 to the public about the need for blood. The
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1 pre-event smallpox vaccination program emphasized
2 the need for advanced planning and consideration of
3 the impact of new vaccine programs on the blood
4 supply. Transfusion-associated West Nile virus
5 transmission required public health and blood
6 collection agency cooperation with the emergence of
7 a new infectious threat for the blood supply and
8 perhaps a place where all the stars were aligned
9 for what appears to have been a very successful
10 collaboration. Now, we are faced with situations
11 such as hurricane Katrina with what appears to be a
12 complete breakdown of the system, much less in our
13 future--we hope not--a pandemic.
14 So, questions to consider: At the
15 national level, state level or the community level,
16 what would integration of the blood system into the
17 public health system add to the blood safety and
19 Since the U.S. blood and plasma
20 distribution is a free enterprise network or
21 not-for-profit or for-profit, how could they be
22 integrated into a government public health
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2 In a major public health event, does blood
3 safety and availability have any real priority now
4 and would integration change that?
5 Is a collaboration rather than an
6 integration of the blood system into the public
7 health infrastructure a more realistic goal? If
8 so, then what strategies and tactics will aid in
9 building on the collaborative efforts that
10 succeeded in developing the response to
11 transfusion-associated West Nile virus?
12 Would integration of the blood system
13 within the public health infrastructure provide a
14 more coordinated approach and funding to dealing
15 with the threat of transfusion-transmitted diseases
16 and complications? That concludes my presentation.
17 DR. BRECHER: Content questions?
18 DR. BIANCO: A quick one, simple, very
19 easy to answer, what do you mean by integration?
20 How do you define integration?
21 DR. ANGELBECK: Well, that is a good
22 question and it is one that I struggled with. I
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1 think integration of an organization could mean
2 that they are more closely aligned in their
3 structures and their development. If you look at
4 countries--for example one that I am most familiar
5 with as a customer of ours is Canada which has a
6 national blood system. It has a means of risk
7 assessment. It has a means of taking that through
8 the regulatory process and then interpreting that
9 into actions or recommendations to the blood
10 collecting organizations. Here, I feel our system
11 is much more fragmented and does not allow or
12 permit, for example, that level of coordination or
13 integration. When you have the blood collecting
14 and the blood supply essentially in the private
15 enterprise and you have public health in the
16 government, be it at the federal level or the state
17 level, they can partner but they cannot necessarily
18 integrate, in my view. They can be collaborative
19 in what they do but I don't see that in my
20 definition of integration. If that helps? I am
21 open to anyone else's definition of integration.
22 DR. BRECHER: Jay?
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1 DR. EPSTEIN: Well, one point that I think
2 you have made and that we have heard discussed at
3 previous meetings is that the public health
4 infrastructure itself, at least at present, is
6 DR. ANGELBECK: Yes.
7 DR. EPSTEIN: So, one could possibly take
8 the point of view that the blood system--probably
9 mainly because of two things, regulation and the
10 force of the voluntary trade organizations--is
11 actually much less fragmented than the public
12 health system. So, I wonder what exactly it means
13 to integrate the blood system in the public health
15 That said, I couldn't agree more strongly
16 that we do need a better interactive dialogue to
17 make decisions about blood safety and availability
18 in the larger context of public health planning,
19 but how you get there in the current state of
20 affairs I think is a little bit puzzling.
21 DR. ANGELBECK: I think it would be very
22 challenging. I think you would need to go outside
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1 the box perhaps to figure out how to do that.
2 DR. BRECHER: Any other questions or
3 comments? It is interesting that, despite having
4 such a fragmented system, I think we have been more
5 successful than almost any other country in
6 protecting our blood supply. So, we shouldn't lose
7 sight of that. Thank you, Judy.
8 Jerry Holmberg is going to fill in for Mat
9 Kuehnert, who could not be at this meeting to talk
10 about surveillance for adverse events related to
11 blood donation and transfusion.
12 Surveillance for Adverse Events Related to Blood
13 Donation and Transfusion
14 DR. HOLMBERG: I am sure that Matt would
15 do a much better job than I am going to do but he
16 sent me his information by way of blackberry from
17 where he was deployed in the South so I will try to
18 give it justice.
19 Some of the things that he wanted us to
20 look at are, first of all, with surveillance there
21 appears to be a need to define what we need by
22 surveillance. As we know, in other countries there
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1 are programs in place for hemovigilance, and his
2 comment here is either as part of or distinct from
4 Some aspects of surveillance are
5 monitoring the known pathogens that are tested, and
6 that seems to be what many other countries do;
7 monitoring adverse events; outcomes in recipients;
8 and then monitoring availability and transfusion
9 practices which, again, I think Dr. Bracey will
10 refer to.
11 The thing that I think we all learned from
12 our last meeting was that there are some
13 surveillance systems that already exist at CDC,
14 FDA, Health and Human Services and also at NIH, NIH
15 with the research at NHLBI with repository of
16 samples that they have. But some of the weaknesses
17 that have been identified are a fragmented or
18 absence of integration. I don't know so much of
19 fragmented but definitely, from my point of view
20 and from what I have heard, it just seems like a
21 lot of these surveillance systems do not talk
22 together and share the information.
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1 Also, another weakness is that there is
2 passive reporting. Definitely, we have a lack of
3 denominator in trying to determine how large of an
4 issue we are looking at.
5 Few approaches to unknown pathogens, and
6 that is something that we are constantly really
7 looking at, that is, how do we look beyond the
8 horizon? There is also little emphasis on clinical
9 education of transfusion-transmitted infections.
10 What we also learned from our previous
11 meetings is that we need to consider both domestic
12 and global needs. Again, partnership in public
13 health needs to be identified and encouraged, and
14 this might go along with the collaboration or the
15 integration of the public health system. Matt also
16 laid out that the possible interventions include
17 integration and standardization of existing tools,
18 in other words, can we ride along on some of the
19 other systems that are currently out there but just
20 enhance them? Analyze analysis of data on
21 currently screened pathogens; use of repositories
22 for pathogens and disease discovery; coordination
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1 of transfusion adverse event systems; connection
2 between blood availability and adverse event
4 Again, I think that Art will talk more
5 about this but creating a link to clinicians for
6 feedback of data and, at the same time, educate on
7 transfusion-associated adverse events and
8 transfusion utilization.
9 I think that over the last couple of years
10 we have heard a lot of discussion about the
11 hemovigilance versus biovigilance, and I think the
12 general conclusion or some of the comments that
13 have been brought forward are that all transfused
14 and transplanted human-derived products need to be
15 considered in an integrated response.
16 Some of the questions and, again, these
17 are questions that I created; Matt did not create
18 these but I throw them out to you: In a perfect
19 world what would surveillance to ensure blood
20 safety include? Should blood safety surveillance
21 include HPC organs and tissues? If so, how would a
22 case for this be developed to support it?
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1 DR. BRECHER: Questions for Jerry?
2 DR. BRACEY: Well, one thing I think we
3 really should focus on is that a lot of the effort
4 has been focused on surveillance of infectious
5 diseases and non-infectious problems that we
6 encounter. I think it is a very important part of
7 our task. I am a bit concerned about the
8 involvement of the end-user, the hospitals. You
9 know, the surveillance that we talk about is
10 surveillance that has been sort of government
11 structured and required reporting. But for many of
12 the non-infectious complications and other
13 complications of transfusion there really isn't a
14 driving force that would, in essence, make the
15 hospital share that information. So, I think one
16 of the things we need to consider is a way to
17 engage that group of folks as well.
18 DR. BRECHER: Yes, we have talked about
19 this in the past, that maybe some sort of sentinel
20 hospital program that aggressively went out and
21 looked for complications as opposed to passive
22 reporting might be one solution. Thank you, Jerry.
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1 Now we are going to move on to
2 coordination of risk communication, Karen Shoos
4 Coordination of Risk Communication
5 MS. LIPTON: Thank you. We really
6 haven't, in the committee as it exists today or as
7 it is presently constituted, had any formal
8 presentations on risk communication so what I am
9 going to talk about today is really some of the
10 presentations and public comments that we have
11 heard that have raised the theme of risk
12 communication, and then move on to my own research,
13 thanks to Judy Angelbeck and to Jerry Holmberg, on
14 some of the principles of risk communication that I
15 have looked at for the committee.
16 I think we can all say that the NGO and
17 the federal agency representatives have all
18 described the difficulties that are inherent in
19 effective communication to physicians and patients
20 about emerging risks to the blood supply. The
21 subcommittee actually included risk communication
22 as one of the proposed elements in the strategic
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1 plan for blood safety and availability that is
2 going to be put forth before this committee today.
3 Current barriers to effective risk communication
4 that have been identified in presentations are,
5 first, lack of a formal and integrated process for
6 risk assessment process. That is, what are we
7 going to say the risks and benefits are? What do
8 we not know about a topic? What do we know and who
9 is responsible for bringing that assessment
11 Risk assessment is not optimally
12 harmonized or coordinated on a global level. We
13 are seeing more and more that some of the things
14 that are happening outside of the United States
15 where people are taking actions and making
16 pronouncements to the public are coming into our
17 country and it is not always clear that we are in
18 advance of that, having appropriate discussions.
19 Timeliness of risk communication is a
20 tremendously big issue for all of us. Sometimes I
21 believe that some of the associations and other
22 patient advocacy groups feel that they need to make
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1 communications that have to occur in advance of
2 federal agency action or information, and it is
3 just because they have an advocacy group or a
4 constituency that is really waiting for information
5 and getting it tomorrow is really critically
7 Then accountability for risk communication
8 is not well understood. I mean, certainly we have
9 a legal system that tells certain organizations
10 that they have an obligation to inform of risk but
11 I think that generically we don't quite understand
12 among all of us, whether it is the AABB, ABC or
13 FDA, who has the primary role in communicating
15 Application of risk communication
16 principles--again, I went back and started looking
17 at some of the scientific literature and it is true
18 that risk communication is a science-based
19 approach, and it is a science-based approach for
20 communicating effectively in what they call high
21 concern situations. There are a lot of things that
22 were said about risk communication but I thought
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1 perhaps the most important was that risk
2 communication is a two-way, interactive process
3 that respects different values and treats the
4 public as a full partner. Sometimes what that
5 means is that you need to communicate with the
6 public in some way through focus groups or
7 something else to understand what their concerns
8 are before you even develop the message.
9 Major barriers to effective risk
10 communication--well, it is conflict and lack of
11 coordination among the stakeholders; inadequate
12 risk communication planning, preparation,
13 resources, skills and practice. We heard a number
14 of presentations that commented on, well, the
15 message might have been right but it was the wrong
16 person stating the message. We have also heard
17 that sometimes even the skill of the person
18 presenting the message--are they a credible person
19 to the public or to the patient population is very,
20 very important.
21 Incomplete understanding and application
22 of models that are highly predictive of how people
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1 react to communication of risk, this is really
2 where the scientific principle comes in because
3 there is a lot of literature out there and a lot of
4 scientific modeling around specific words that
5 should be used when you talk about risk
6 communication; specific words when you talk about
7 lack of information but you still need to
8 communicate. And, we probably could do a better
9 job of integrating those into our own risk
10 communication process.
11 So, the questions for this committee to
12 consider: Are the roles for communicating risk in
13 various circumstances clearly defined? How should
14 the message be developed? Who is the target
15 audience and who should deliver the message and in
16 what media?
17 Two, are the principles of effective risk
18 communication clearly understood by the parties
19 responsible for creating and delivering the
21 Three, should there be a risk
22 communication plan relating to threats to safety of
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1 the blood supply? I called it safety of blood
2 processes for lack of a better word but that is
3 really around the issue of things like
4 glucoreduction and bacterial detection? And,
5 should there be a risk communication plan relating
6 to threats to blood availability? That concludes
7 my presentation.
8 DR. BRECHER: Content questions?
9 [No response]
10 I guess that was perfectly clear. Thank
11 you, Karen. Our last speaker before we break for
12 lunch is Jeanne Linden on error prevention in blood
13 collection centers, transfusion services and
14 clinical transfusion settings.
15 Error Prevention in Blood Collection Centers,
16 Transfusion Services and Clinical
17 Transfusion Settings
18 DR. LINDEN: This topic, although it
19 wasn't discussed recently, has been discussed
20 previously by this committee and we have had some
21 presentations focused not solely on the infectious
22 risks but significant risks, particularly in terms
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1 of mortality currently that continue to be acute
2 transfusion reactions due to errors in blood
3 administration or preparation, and so forth, and
4 also TRALI, which we have talked about previously.
5 Many of the errors, based on analysis to
6 date, appear to be preventable. Therefore, we may
7 be able to do something about those. And, there
8 tend to be underlying systems factors in many
9 cases, what are be called latent systems pathogens
10 that may be present that predispose to some of
11 these active errors, and identification of those
12 may facilitate preventing errors and just making
13 the process of transfusion safer. We tend, in this
14 committee, to look at infectious diseases and blood
15 safety in terms of the product itself but
16 transfusion is really a process. It goes from the
17 donor's vein all the way to the recipient's vein
18 and the product could be completely sterile, but if
19 it is the wrong component for the wrong person,
20 then that can be just as deadly as an infectious
22 We also have heard that many of the issues
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1 identified in the transfusion process have
2 commonalities with other industries, including some
3 with very significant adverse events such as the
4 aviation industry and the nuclear power industry.
5 Some of these other industries have done a very
6 good job in having good error reporting systems and
7 identifying factors that can be addressed. So,
8 what could we, in the blood industry, in a plan use
9 from those other industries as lessons that could
10 be incorporated?
11 One difference, however, is that the blood
12 transfusion process does involve many different
13 individuals with different types of expertise. As
14 Dr. Bracey just mentioned, here the input of the
15 clinicians has often not been incorporated as much
16 as it could be and they, on the front line, are
17 very critical to this process and, in fact, several
18 studies have shown that over half of the
19 transfusion-related errors are outside the blood
20 band, are on the clinical side and that is where it
21 may be productive to focus some of our efforts.
22 There certainly are quite a few existing
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1 surveillance systems. They are not really
2 coordinated or comprehensive. There is a lot of
3 focus on fatalities and morbidities as sentinel
4 events. As has been mentioned with infectious
5 disease surveillance, there is often not a lot of
6 denominator data available with many of these
7 systems. A couple--you know, the U.K. system has
8 some fairly good data. A lot of the rest are
9 estimates at this point and this is another place
10 where we could put further efforts.
11 Assuming that strategies to prevent errors
12 can be identified in this process, if they are to
13 do any good they need to be implemented. They must
14 be acceptable to the individuals, the stakeholders
15 who are going to be using them. Thus, their input
16 needs to be incorporated into the process. They
17 can not be too cumbersome. They should make it
18 easy to do the right thing and difficult to do the
19 wrong thing, when possible. They need to address
20 human factors issues in their design, and how can
21 that be accomplished and applied to the blood
22 transfusion setting to promote blood safety?
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1 So, some of the questions to think about
2 are how can surveillance of non-infectious risks,
3 and specifically errors that are identified,
4 increase the knowledge of these risks and
5 facilitate the identification of the underlying
6 systems factors through a root cause analysis type
7 of approach or some other approach to identify
8 these underlying problems?
9 What else can we learn by looking at some
10 of these other industries? How can we apply these
11 lessons to this particular situation? And, how can
12 we get input and involve the clinicians in the
13 process of determining what the goals would be and,
14 once those goals are determined, to raise the
15 awareness of these problems so that they feel that
16 they are involved in the process, accept the
17 strategies that have been identified, and also to
18 increase the recognition of adverse reactions when
19 do occur to facilitate early intervention which may
20 be possible?
21 Who exactly are the stakeholders that need
22 to be involved? What is the role of the Department
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1 and this committee and how could these issues, even
2 the transfusion process, the non-infectious risks
3 perhaps be incorporated into surveillance systems
4 that we are discussing for infectious
5 complications? Can those be more integrated as a
6 total human vigilance type of approach as is done,
7 for example in the United Kingdom where they look
8 at all of the serious hazards of transfusion and
9 not only the infectious ones? Thank you.
10 DR. BRECHER: Content questions for
11 Jeanne? If not, we will adjourn for lunch for an
13 [Whereupon, at 12:50 p.m., the proceedings
14 were recessed for lunch, to reconvene at 1:50 p.m.]
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1 A F T E R N O O N P R O C E E D I N G S
2 1:51 p.m.
3 DR. BRECHER: If everyone will take their
4 seats, we are going to get started again.
5 We are going to begin with Dr. Bianco
6 talking about donor recruitment and retention.
7 Donor Recruitment and Retention
8 DR. BIANCO: As I started working on those
9 questions to the committee, as part of this kind of
10 strategic thinking for our program, I went back and
11 reviewed the committee recommendations pertaining
12 to blood donors.
13 We hear a lot about blood donors, or heard
14 at least in the past, there was always the theme of
15 the blood shortage, the concern whose
16 responsibility was the blood shortage, what could
17 be done to alleviate it, and what was the role that
18 this committee could have, and government in
19 general, in terms of helping with that.
20 So, it starts in April '99 when we
21 reviewed actually the discussions on
22 hemochromatosis and was the timing when FDA also
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1 looked at those questions and ultimately, came with
2 ways by which individuals with hemochromatosis
3 could donate, and their blood could be used for
5 There was somewhat of a fantasy that this
6 was going to resolve the problems of the blood
7 supply, but certainly we all know that their
8 contribution, while it is meaningful, it was not
9 enough to really resolve it.
10 The second thing was the discussion that
11 we should, because of the shortages, and maybe to
12 better understand the blood system in the country,
13 that we should collect data. There wasn't enough
14 data, and there isn't enough data, and there aren't
15 too many models that can predict blood shortages.
16 We know, on Mondays, what is the total
17 that was collected by all the movie houses in the
18 country per movie, but we really don't know how
19 many units of blood are in our shelves except that
20 now organizations are working harder to try to
21 collect that, and the market has found a system of
22 balancing supply, and actually, we are in a period
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1 in the last couple of years after all the
2 investments of a reasonable blood supply.
3 In January 2002, we continued discussions,
4 but now they were tainted by the September 11
5 disaster, and the concern that we all had that we
6 should have mechanisms to fund the development of a
7 reserve that could make sure that in case of need,
8 we would have that blood.
9 There was a recommendation from this
10 committee for funding, not only funding, but to
11 evaluate in a recommendation to the Secretary, and
12 to really make the blood donor and the blood
13 donation a national service, and recognize it as
14 many of the other public services that are
15 performed by the population.
16 I remember someone mentioning at that
17 time, I believe it was Ron Gilcher, if we have
18 volunteer fire departments, if we have volunteer
19 ambulances, we should, in the same way, have enough
20 people dedicated to blood donation.
21 In September 2002, we continued to discuss
22 the promotion of blood donations through a number
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1 of mechanisms that could help raise the level of
2 the blood supply, but again, we did not resolve at
3 that time who was going to be in charge of that.
4 There was the hope that sometimes was interpreted
5 as whining that government would take a fundamental
6 role in funding this approach.
7 In 2004, in January 2004, we again decided
8 that it was very important to take steps to develop
9 a 5- to 7-day inventory of blood components in all
10 blood centers to stabilize the blood system.
11 Again, here, we identified CMS through
12 reimbursement as an agency that could contribute to
13 that effort, and that a national blood reserve
14 should be funded as a government-private sector
15 partnership. That has not happened.
16 So, I think that the questions that come,
17 and those discussions very much reflect over time,
18 all the issues that were raised regarding blood
19 donors, is what is the blood safety and
20 availability role of each of the responsible
22 We are, and I think that integration was
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1 the word that Judy came with, but essentially, what
2 is the role of the blood providers, should they do
3 it by themselves, should they fund entirely the
4 donor recruitment, or is there some government,
5 social responsibility in that sense?
6 I think that that question is unanswered,
7 and we have let it to go through market forces that
8 not always works. I believe that this should be
9 discussed in detail, what is the role of
10 transfusion services--and we will have Art
11 discussing some of that in a few minutes--the role
12 of government and each one of its agencies, HHS,
13 this committee, FDA, CDC, National Heart, Lung, and
14 Blood Institute, and CMS, and then Homeland
15 Security and FEMA that have been very much in the
16 news in the last few days, state and local
18 Who should participate of the process,
19 what is the responsibility of each one regarding
20 blood donations?
21 The second question is what is the ideal
22 blood supply? We had concerns or have concerns
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1 from time to time about the supply, shortages, and
2 the impact that it has in the whole healthcare
3 system, but we don't have an answer what is the
4 ideal blood supply - is it 3 days, 5 days, 7 days,
5 25 days? How many days inventory are necessary and
7 This is a short-lived product, we don't
8 want wastage, but at the same time, we don't want
9 to be in a situation where we don't have what we
10 need, and that has to be decided.
11 And then, what is the additional inventory
12 of red cells, platelets, and plasma needed to be
13 maintained to ensure availability during times of
14 the collection, and here, we can talk about
15 Christmas, summer, and emergencies. There is
16 localized epidemics, public health actions like
17 mass vaccinations for it could be smallpox, massive
18 donor deferrals, or a disaster like happened with
19 Katrina, that is there, not so much the need of
20 blood was the issue, but certainly the blood center
21 in New Orleans, the building was destroyed.
22 They are working out of their--they moved
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1 their operations to Baton Rouge, and they have a
2 contribution from Dallas, from Carter Blood Care,
3 that is actually housing some of its staff, but
4 their collections were totally disrupted, as were
5 the collections of several blood centers in
6 Mississippi and in Louisiana.
7 Finally, how do we fund that? Is it still
8 even if the donor is a volunteer that is donating
9 blood to us, and if the rest of the system has to
10 work under market forces, who should fund it, is
11 the hospital and payer that will pay for that
12 effort of having these donors, or is there a role
13 for more of society to invest in this process?
14 If there are any questions, I will be glad
15 to attempt to clarify them.
16 DR. BRECHER: Any comments or questions?
17 [No response.]
18 DR. BRECHER: Perfectly clear, Celso.
19 Thank you.
20 We are now going to move on to the
21 Clinical Practice Standards for Transfusion, Art
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1 Clinical Practice Standards for Transfusion
2 DR. BRACEY: By way of background, the
3 committee has not discussed the actual clinical
4 indications for transfusion in previous meetings,
5 so I would have a blank as my first slide.
6 But I think one of the things that we all
7 know, and many of us around the table have invested
8 a lot of time in this activity, is that many
9 transfusions today aren't necessary. If one
10 surveys the literature, you can find papers that
11 report anywhere from 20 to 50 percent inappropriate
12 transfusion incidents, and that is a problem.
13 In addition, in medicine, one of the
14 mantras is first do no harm, and really, the
15 inappropriate use of blood increases the risk of
16 the transfusion therapy irrespective of how safe
17 the unit is.
18 You know, much of our focus has been on
19 minimizing the infectious risk of blood, but we
20 must be certainly aware of the fact that as the
21 infectious risk decreases, that alters physician
22 behavior. The physicians then may begin to
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1 transfuse more liberally, and then to perhaps
2 enhance other risks associated with transfusion.
3 Clearly, as in driving SUVs and consuming
4 lots of gas, unneeded transfusions will have a
5 direct impact on blood availability. You know,
6 it's amazing. Many transfusion services, if you
7 talk to folks out in the hallways at national
8 meetings, they will see, "Well, what do you do
9 during a blood shortage?"
10 Well, you know, I just go around and tell
11 the guy that he doesn't need to give that
12 transfusion today, but we don't do this on an
13 ongoing basis. So, we have a system that really is
14 a permissive system, but not a system that is very
15 proactive in terms of controlling how blood is
17 Transfusion practice is highly variable.
18 Dr. Toy and other members of the Transfusion
19 Medicine Academic Awardee Group had a very
20 interesting study of one select group of patients,
21 and these are cardiac surgery patients, and they
22 have demonstrated that depending upon the hospital
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1 that you are in, your risk of transfusion varied
2 anywhere from 25 percent up to 100 percent, and
3 this was in 1992.
4 What is amazing to me is that if you look
5 at a follow-up study done by a group of
6 anesthesiologists, well, it's about the same. So,
7 there is a great degree of variation in terms of
8 practice, and I think it really behooves us to look
9 at why is there such wide variation.
10 Now, one big part of the problem is that
11 there are really no uniformly accepted guidelines.
12 The NIH, recognizing in the early '80s that we
13 really did have problems in terms of, you know,
14 when one needed to use blood components, set up a
15 series of consensus conferences, and there was some
16 good information that came out of there.
17 A lot of the information really basically
18 said that we need more information, but what
19 happened then is that various subspecialties or
20 societies developed guidelines, so you had all
21 these--really, the guidelines weren't divergent,
22 but they still weren't uniform.
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1 They weren't not one and the same. So,
2 for the physician, one would have to decide whether
3 to use the ASA guidelines, or whether to use ASIM
4 guideline. There is no single guideline.
5 Even worse is if you are in a hospital, if
6 you practice in a city and go from hospital A to
7 hospital B, between those two entities, there could
8 be totally divergent guidelines for transfusion, so
9 it would really be helpful to have a uniform
11 Sonny Dzik recently published a paper
12 looking at the use of FFP, and the paper's title, I
13 think really speaks the problem that we have. Its
14 title was A Paucity of Clinical Trials Exists--I
15 can't remember the exact title, but he captured the
16 scenario. There is a paucity, there is a dearth of
17 clinical trials related to transfusion decisions.
18 Now, there is help on the way, because the
19 NIH and the NHLBI has a Transfusion Medicine and
20 Hemostasis Clinical Trials Network that is in
21 progress to address some of these issues, but
22 still, in this point in time, there are very few
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1 clinical trials that we can use.
2 Beyond that, if one looks at systems--and
3 we talked before about communication systems,
4 public health talking to, et cetera--if you look at
5 operations within a hospital, the way things work,
6 our systems, to predict transfusion requirement,
7 really need to get improved.
8 If you look at certain facilities or
9 publications where they have designed a near-site
10 testing systems' ready access to data, so that one
11 could transfuse based upon data-driven decisions,
12 you always see improvement, but that is the
13 exception. Hospitals that have that sort of a
14 system are the exception rather than the rule.
15 Even further, if you look at the tools
16 that we have to diagnose a deficiency in the blood
17 in terms of its function or the need of a given
18 patient, we are also limited, very limited.
19 I mean there was the meeting of the
20 Hemoglobin Oxygen Carriers Group, and they just
21 couldn't decide, you know, what was a reasonable
22 hemoglobin. If you look at evolving issues in the
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1 field now, there are patients that are getting very
2 potent anti-platelet drugs. Most hospitals don't
3 have a way to test for the effect of those drugs.
4 So, our diagnostic systems are: (a)
5 really not geared up, and (b) they are just
7 A big problem for me, because what happens
8 in many hospitals, is that the accountability for
9 blood use resides in the Pathology Department.
10 Now, wait a minute. I don't write the orders for
11 the blood, the physician that is caring for the
12 patient writes the order, so there are problems in
13 terms of having really an accountable situation for
14 the person that is prescribing the blood
16 There have been some interesting
17 approaches to that, that other centers have had,
18 such as indexing physicians related to blood
19 utilization, but that again is the exception rather
20 than the rule.
21 Clinicians--and when I say "clinicians," I
22 am taking in the broad sense, I am talking about
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1 nurses, and I am talking about physicians--are
2 poorly trained in transfusion medicine.
3 If you look in an ICU, and you ask a nurse
4 about dose of dopamine or how to deliver dopamine,
5 they actually know more than many of the early
6 trainees. If you ask them a few questions about
7 blood or blood transfusions or how to administer
8 blood, you often get sort of a blank look.
9 So, we really have I think an important
10 role to play in terms of enhancing the education of
11 those within the field.
12 Then, one real pet peeve of mine is that
13 there are resources that the AABB has put together
14 and various other organizations, but those
15 resources aren't getting to the end user.
16 A classic example is the Circular of
17 Information. It is sort of a treasure trove of
18 facts and figures about how to use blood. Whenever
19 I show this to a surgical resident, you know, their
20 eyes light up. These things are unknown, they are
21 uncovered, so we have to figure out a way to get
22 those resources to the people that really need
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2 So, what I was thinking about, questions
3 along the lines of clinical practice, there are
4 several questions that came to mind.
5 One is--and one can demonstrate in the
6 short term when you publish a paper, that
7 educational efforts in fact do improve blood
8 transfusion--but the question that exists is how
9 durable is this and are we using the right
10 educational efforts, the ones that we are investing
11 in today.
12 The second is, you know, this is the world
13 or this is the time now of benchmarking. One thing
14 that my hospital, and I am sure all hospitals pay
15 attention to right now, is where they are
16 benchmarked, and the benchmarking is largely
17 related to certain outcome measures.
18 In fact, one of the benchmarks is
19 bleeding, for example, for cardiac surgery, but is
20 there some way to tie in transfusion to this
21 benchmarking activity, and can that in some way
22 improve performance or practice or blood
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2 I read recently a trial, the PACMAN trial,
3 which is a trial of patients using pulmonary artery
4 catheters, and there was an editorial to it, which
5 I found very interesting.
6 In the editorial, it said, well, even
7 though there are clinical trials that prove a given
8 point, what is it that will make the practitioner
9 actually pay attention to that trial and adopt the
10 finding of the trial, the point being that the
11 people that perform trials and read the literature,
12 that is one group, but there is whole other
13 universe of people out there.
14 So, the question is how do you get that
15 information, when you have the trial, how do you
16 best disseminate it to impact practice.
17 Another element is, is the blood community
18 really effective in implementing change, and by
19 that, what I mean is are we insiders or are we
20 outsiders. I was really very much impressed by a
21 statement that was made.
22 I was at an international meeting in
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1 hematology, and a well-known figure in platelet
2 function--the discussion was, you know, what sort
3 of tests one would order in advance of surgery--and
4 the point that was made is that, you know, whatever
5 this individual said, or people that were sort of
6 outside of the sphere of a given area of practice,
7 was largely ignored.
8 So, the question is how can people within
9 transfusion get out of a shell and begin to branch
10 out to the other prescribers or users of blood.
11 Last, is what really is appropriate role
12 for government in enhancing transfusion practice.
13 It is interesting because, you know, there is this,
14 well, this is the practice of medicine, so the
15 government should not interfere with the practice
16 of medicine, but on the other hand, if there are
17 practices that aren't optimal, that impact safety
18 and that impact availability, then, should the
19 government get involved.
20 So, I would end with that in terms of my
21 considerations, in terms of practice. I think
22 there is much to be done, and one thing that I
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1 didn't mention is that there are governments where
2 this is now evolving after the vCJD issue in the
3 UK. There is a huge effort there to impact
4 practice in blood utilization.
5 So, I will stop with that and open up for
7 DR. BRECHER: Content questions?
8 [No response.]
9 DR. BRECHER: Okay. Thank you, Art.
10 We are now going to move to the Research
11 Agenda. Merlyn Sayers.
12 Research Agenda
13 DR. SAYERS: If you go to your agenda, it
14 says Research Agenda, and then it says TBD, and I
15 confess to being TBD.
16 Jerry approached me to make some comments
17 about the research agenda because Harvey Klein and
18 Andrew Heaton are out of town, so I did not attend
19 any of the sessions that they had, I certainly had
20 access to their notes, but I said to Jerry that I
21 would take up this task if he recognized that this
22 would give me an opportunity to sprinkle my
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1 interpretation of what the group thought about,
2 sprinkle those thoughts with my prejudices.
3 Against that background, if you suspect
4 that you hear echoes of what Celso has said, what
5 Art Bracey has said, what Jeanne Linden has said,
6 your suspicions are well founded.
7 So, let's start out with this preface. I
8 have said here that research in blood banking and
9 transfusion medicine from the safety point of view
10 is particularly strong in certain areas. An
11 example is red cell immunohematology and
12 transfusion-transmitted diseases.
13 From the point of view of availability,
14 there certainly have been investigators, and Jane
15 Piliavin is somebody that came to mind who made
16 important contributions here, that research is much
17 less focused on an understanding of pro-social
18 behavior, on altruism, and on motivation.
19 As far as our national inventory is
20 concerned, we seem to lurch between surplus and
21 insufficiency, and at the moment, our inventories
22 are full as a result of the outpouring from the
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1 community in response to Katrina, but we do know
2 from emerging evidence that crisis responders are
3 not the individuals who are promptly converted to
4 regular donors.
5 We have been saying for something like 40
6 years now that something like 60 percent of
7 individuals are eligible, but only 5 percent do
8 donate, and so long as we persist with that lament,
9 as long as we have been doing that, we really
10 haven't been assured of a stable inventory.
11 I think that is just a reflection of our
12 ignorance as to what the key elements are in
13 understanding behavior, pro-social behavior, and
15 So, there is this disproportionate
16 emphasis then, and it was really revealed by a
17 review of the research issues that were discussed
18 at recent meetings here. I have listed some of
19 those issues - optimal treatment for rare blood
20 disorders, bacterial contamination, the risk of
21 transfusion-related acute lung injury, universal
22 leukoreduction, mad cow disease, HHV-8, babesiosis,
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1 Chagas, pathogen inactivation, and the risk of
2 contamination of the blood supply with bioterror
4 I don't want my remarks to be construed as
5 criticism of anyone who would want to eliminate
6 even the remotest risk associated with transfusion,
7 but we really do need to develop a script that
8 addresses the common, as well as the rare.
9 We have heard even today, take Chagas, for
10 example, that this is a quote, "unmet" challenge,
11 but are seven cases in the United States and Canada
12 since 1987 really of such dire consequence that we
13 could label that risk as an unmet challenge.
14 I mean that is one case every two or three
15 years. It does reflect, though, the devotion to
16 research that is intended to further reduce the
17 risk of transfusion-transmitted infection, and
18 while we are witnessing that drive to the zero risk
19 blood supply, the major contributor to fatalities
20 associated with transfusion has really not enjoyed
21 the same research intensity, and patient
22 misidentification persists and patient
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1 misidentification accounts for more acute deaths
2 than all the other transfusion-transmitted
3 infections combined.
4 So, why does that risk persist? It may be
5 that we are just not good at multi-disciplinary
6 approaches. How do we bring together hospital
7 administration, nursing, information management,
8 physicians, pharmacy, the blood bank?
9 As far as the availability is concerned,
10 if maintaining availability is going to earn equal
11 research attention, then, recruitment needs to be
12 based on an understanding of donor behavior.
13 I don't want to sound melodramatic, but
14 when the patient says, "Is my transfusion safe,"
15 the patient has to be reassured, first, that the
16 blood is going to be available should he or she
17 need it, and that, secondly, we have to respond to
18 the question about safety with, well, we have to be
19 assured that we are not going to confuse you with
20 some other equally deserving recipient.
21 In fact, this committee actually had a
22 recommendation which goes back to January of 2003,
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1 urging the Secretary to take steps to encourage and
2 facilitate implementation of measures that could
3 prevent errors in the transfusion setting.
4 So, here are a couple of questions, then,
5 to consider, just at a very plodding level.
6 Should the Department encourage research
7 into systems that would ensure something as simple
8 as the right unit of blood goes to the right
10 It might have been a little more
11 intellectually satisfying to have worded that
12 question along the lines of should research be
13 encouraged to ensure that the common risks are
14 addressed, as well as the esoteric.
15 Having dealt with the safety side of
16 things, then, the other question to consider is:
17 Should the Department encourage interdisciplinary
18 approaches to understanding altruism?
19 I am afraid that if we don't understand
20 altruism, we are going to have the pitfalls and the
21 troughs in the national blood supply, and an
22 interdisciplinary approach would achieve something
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1 that we have not really achieved well, and that is
2 bringing together the sociologists, the behavioral
3 psychologists, the motivational psychologists, and
4 those individuals that would help us understand
5 what really is behind the active volunteer
7 End of sermon. Thanks.
8 DR. BRECHER: Questions for Merlyn?
9 [No response.]
10 DR. BRECHER: Then, we are going to move
11 on to Disaster Planning. Dr. Sue Roseff.
12 Disaster Planning
13 DR. ROSEFF: I am here to discuss disaster
14 planning, and I am at a little bit of a
15 disadvantage since I just joined the committee at
16 the last meeting, and there were extensive
17 discussions about disaster planning after September
18 11th, so I am relying on a little help from my
20 I want to thank Jerry and Mark and Karen
21 for supplying me with much of the information I
22 will be discussing. I would also like to invite
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1 the members of the committee who were here or
2 anyone else involved in the discussions to feel
3 free to add anything that I have omitted or changed
4 the focus of what I am discussing.
5 After the September 11th attacks, the
6 Interorganizational Task Force on Domestic
7 Disasters and Acts of Terrorism was formed in
8 December 2001 in order to develop a response plan
9 for future national disasters.
10 One of their charges and one of the things
11 that they felt was important was to have a smooth
12 process in place for blood collection efforts, and
13 as we all know, after September 11th, we lost a
14 great deal of trust with the public and donors
15 after it was discovered that much of the blood that
16 was collected, or not much, but a certain amount of
17 it was thrown out and never used.
18 So, therefore, it was very important,
19 according to this task force, that we develop a
20 policy that would allow a central coordinating
21 effort to give a consistent message to all blood
22 donors and to the public.
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1 They also recognized a need for a national
2 inventory management program, and Southwest talked
3 about this in a little bit of detail, and again,
4 the question of should this be a 5- to 7-day
5 inventory, and also the importance of having
6 adequate inventories at all times in order to
7 respond to disasters.
8 As we know, the blood that is used at the
9 time of a disaster is not the blood that is
10 collected the next day. It is the blood from donors
11 who have donated to maintain the supply up to that
12 point. So, therefore, the question was do we need
13 to encourage this in some form to have a supply
14 that will be there in case of a disaster, not after
15 the disaster.
16 Finally, the AABB was tasked with
17 coordinating this entity, and I have listed here
18 the alphabet soup of organizations that are
19 involved in the task force.
20 After September 11th, in the winter of
21 2002, this committee met, and their task was to
22 look at lessons learned after September 11th, and
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1 ask can we strengthen the safety and availability
2 of the United States blood supply.
3 As a result of the meeting, the committee
4 then wrote a letter to then Secretary Thompson and
5 brought up the following points. First, the
6 committee endorsed the role of the AABB Task Force.
7 They also recommended the incorporation of
8 the task force recommendations and members into
9 some of the federal structure that is involved in
10 disaster response, so that there would be a more
11 coordinated effort.
12 Again, they discussed the need to build
13 blood reserves and to have a system that monitored
14 blood availability on an ongoing basis, so we could
15 detect if there were shortages that might affect
16 the need or the ability to respond to a disaster.
17 In addition, they discussed the importance
18 of an infrastructure for transportation in times
19 when a certain part of the country is affected, how
20 can we move blood around, how can we move reagents
21 around, how can we move testing around in order to
22 meet needs, the need for an integrated
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1 communication facility or group, so that again, we
2 get a consistent message out that is able to speak
3 to all the stakeholders during these times of
5 Also, redundancy. We need to have
6 redundancy in case, of course, certain parts of the
7 country are destroyed and the capability of
8 collecting, transporting, and testing blood can't
9 be done in one region, we need to obviously be able
10 to move that very rapidly, so that there isn't a
11 loss of resources at that time.
12 Also, it was recommended that if there are
13 any regulatory revisions, either permanent or
14 temporary, that these should only be addressed in
15 terms of what was needed for patient care at the
17 As part of this letter, too, the committee
18 recommended to the Secretary that blood donors be
19 considered a national resource.
20 Finally, some questions to consider for
21 discussion. Should disaster planning be part of
22 any kind of strategic plan that this committee
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1 comes up with? What is the current role of the
2 AABB Interorganizational Task Force on Domestic
3 Disasters and Acts of Terrorism?
4 One thing I would like to add is that
5 during Katrina, we did have a good, consistent
6 message about blood, the need for blood or the lack
7 of need for blood, and we didn't see the same
8 rushing to blood centers of donors as we saw after
9 9/11, so that was very effective.
10 Also, is the structure of the task force
11 and its funding adequate currently? Is there
12 currently a structure in place to move resources in
13 times of disaster, and is what is the status
14 currently of a national blood reserve?
15 DR. BRECHER: Content questions or
16 comments besides the open question of what reserve?
17 Maybe this might be a good time to get an
18 update on the Interorganizational Task Force.
19 Maybe Karen might say something about that.
20 MS. LIPTON: Yes. Well, we were operative
21 during Katrina and most of our issues I think were
22 trying to help our facilities that were affected
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1 physically in the area to deal with some of the
3 We don't have a full report because our
4 usual process is we actually afterwards go through
5 a whole process of evaluating. I will see that I
6 think one of the things that did happen is, because
7 the other problems were so immense and so
8 overwhelming, that I believe it was a little bit
9 difficult at times for us to get the attention that
10 we needed, and we didn't have massive amounts of
11 blood required, but we did have ongoing operations
12 for some of the centers that were affected.
13 So, we will promise to bring back a full
14 report at the next meeting, if that is all right
15 with you.
16 DR. BRECHER: One other quick question.
17 What if the hurricane had hit Washington? The
18 Interorganizational Task Force is basically run out
19 of AABB, is there provision for an alternate site?
20 MS. LIPTON: Well, one of the issues
21 related to that, that we have been struggling with,
22 is trying to get enough money for redundant
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1 resources within AABB. We have a server that is in
2 Virginia, but we do have to worry about, if one of
3 those servers goes down, how do we communicate with
4 everyone else.
5 We are not as much people dependent in the
6 sense that we have people all over the country, and
7 actually, in different parts of the world, who
8 could step into the position of being a
9 communication person and the point person, but I do
10 think that the systems are the things that we need
11 to worry about, and we need to worry about
13 We have not gotten any funding for this
14 activity, as you probably all know, so it is really
15 something that the blood organizations and the AABB
16 do on top of everything else that we do, but we
17 have been in dialogue with the Department, and I
18 think they understand our needs, and we will
19 continue to work on the issue.
20 DR. BRECHER: Thank you. Any other
21 comments or questions?
22 If not, we are going to move into another
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1 public comment period. So, if anyone has a public
2 comment, could they come to the microphone and
3 identify themselves.
4 The first one is Corey.
5 Public Comment
6 MR. DUBIN: Our thanks to Jerry, the
7 committee, for getting the opportunity to speak. I
8 am Corey Dubin of the Committee of Ten Thousand. I
9 think what makes us unique in the process is we
10 have been around since the beginning, previous to
11 this committee. It was the committee of Ten
12 Thousand that approaches Senators Graham and
13 Kennedy, which resulted in the IOM study.
14 We asked for a congressional
15 investigation. They gave us the IOM study. It
16 turned out to be a very good one and a very wise
17 choice on their part. We were around for the
18 founding of the committee, and we have been here
19 throughout the process.
20 Our comments today are rooted in our
21 perceptions and our board of directors' and
22 community's perception, and distinct from the NHF
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1 or other hemophilia organizations, our primary
2 constituency is those infected with HIV and HCV
3 from tainted blood.
4 We really grew out of the disaster. We
5 grew out of services not being available. We
6 started as a support group.
7 The IOM recommendation establishing this
8 committee talked about interagency coordination, it
9 talked about coordinating the federal response, and
10 those are things that we think are very important.
11 We saw that as the mission of the committee, and we
12 saw the committee's client as the Secretary of HHS,
13 Health and Human Services.
14 The question our board would raise today
15 is, if we would all agree that the client of this
16 committee is the Secretary, has there been a
17 breakdown in recent years between the committee and
18 the Secretary, has the value of this committee and
19 what this committee brings to the table been lost
20 on seniors at HHS, are seniors at HHS clear about
21 what this committee is about and what it can do.
22 We think it is a unique history of this
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1 committee, a history born of the epidemic, born of
2 everyone's frustration, and as a result of that
3 frustration, a willingness to think out of the box,
4 to do things different.
5 Our board this past week asked the
6 question do seniors at HHS understand that unique
7 history and what was accomplished between
8 government and all of the stakeholders - industry,
9 community, Red Cross, the public health structure,
10 and we continue to question that, and we believe
11 that it's most important to nurture the all
12 stakeholders' grass roots community participation
14 We think that that is the model is what is
15 in trouble right now. We are concerned that the
16 trust we had, and continue to have at this level,
17 may not be shared above, and it may just be a
18 question of understanding that history.
19 It is our hope that it is not that that
20 history is not valued in this particular historical
21 period, but that it is not understood.
22 We are also concerned that the question of
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1 keeping our eyes on the prize has also been a
2 problem, that we have drifted. Some of the ideas
3 that originally came up in the IOM, that we feel
4 are on the table and haven't been worked on,
5 no-fault compensation for those that are injured by
6 blood and blood products, and even more important,
7 a national blood policy which we went into the IOM
8 report asking for in the hearings and through the
9 process, talked to Congress and believed that this
10 was the committee where the framework, if you will,
11 could be knocked down, the hard knocks part that
12 had to be discussed had to be worked between
13 communities, had to be negotiated, could ultimately
14 be worked out with an eye towards taking it towards
16 We see this as kind of the model of how
17 the committee is structured today and how it works,
18 and we are more concerned in seeing this kind of
19 model that has a more clarity of communication
21 I come from the radio world, radio
22 journalism, and we always talk about loops, be they
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1 60-cycle hum loops, or be they communication loops
2 between reporters in different places.
3 We think the loop outside the community,
4 outside of this room and the committee, is not
5 strong like it used to be. Our board has expressed
6 a real concern about that, and a desire that I stay
7 focused on that point with the committee today in
8 our presentation.
9 These are the stakeholders as we see it,
10 and this slide is just putting them on paper,
11 really, you all know - the blood-banking industry,
12 both the voluntary and for-profit, the
13 manufacturers from the fractionators to biotech,
14 the health and medical community, and the end
15 users, consumers, advocates, organizations, such as
16 the NHF, the Committee of Ten Thousand, Hemophilia
17 Federation, the Immune Deficiency Foundation, all
18 of us representing the community.
19 This is how our community, and I suspect
20 through our work with the plasma users coalition,
21 how some of the other communities view the mission,
22 to coordinate the Federal Government's response to
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1 threats to the nation's blood supply, using the
2 interagency tools at its disposal, to evaluate
3 supply and allocation of blood, blood product
4 resources, ensuring available, safe supplies for
5 communities and individuals in need, to bring
6 relevant federal agencies together to ensure safety
7 to the greatest degree available, and ensure
8 availability through strategic planning for today
9 and the future.
10 This is our sense of what works. The IOM
11 report worked because it stressed the work between
12 communities. The establishment of the ACBSA and
13 the presence of grass-roots community
14 representatives at the table worked.
15 Those were some fairly heady days in '96,
16 '97, '98. There was a real sense of urgency and an
17 openness on all sides of the table to listen to
18 each other, to learn from each other, to help
19 educate each other to move through what was then
20 considered a crisis.
21 The inter-stakeholder dialogue and
22 discussion that resulted, the interactive learning
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1 that occurred on all sides of the table, the
2 respectful and thoughtful dialogue discussion, it
3 happened here. It also happened at FDA in the
4 Blood Products Advisory Committee, and it was a
5 very interesting period.
6 The openness of government to allow and
7 nurture this creative and unique process to go
8 forward, all parties working together to ensure
9 adequate funding for the continuation of this
10 interactive process and the inter-stakeholder
11 process, and a key point - historical continuity.
12 We don't want 1997 viewed in a vacuum, or
13 1998 viewed in a vacuum. That was a moment that
14 was important, but we saw that as the beginning of
15 a new historical reality, a new mission, a new way
16 government and communities that we impacted and
17 affected by government decisions, industry as the
18 producers, blood bankers, everybody could come
19 together and talk to each other in a way they had
20 never done before.
21 We are concerned, and our board talked
22 about this, as well, is what we loosely called, and
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1 we wrestled very much with how to communicate this
2 in a way we felt would be effective, but resist the
3 logic of power and a narrow professionalism in
4 order to keep the committee alive, and we don't
5 mean to take a swipe at professionalism, we do
6 believe in it, but we think there is a natural
7 thrust of government to move towards more
8 centralization, less community involvement, and a
9 narrowness to make sure everybody at the table has
10 a DR in front of their name, Doctor, Ph.D. after,
11 which is a good thing, but what we are concerned
12 about is the exclusion that those who don't have
13 that, who are the recipients of the decisions made
14 here, made it to Food and Drug Administration, and
15 made it upstairs in HHS, and we are very concerned
16 about that.
17 Government and community support for
18 grass-roots advocacy, we think advocacy has lost
19 some of its value, at least upstairs at HHS. We
20 don't necessarily see that in the committee because
21 we still feel an openness from you all to work with
22 us in a continued presence on the committee, people
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1 like Mark Skinner, Paul Haas, people who come from
2 our community.
3 One of the things that really worked in
4 terms of this model for positive change, that I had
5 the honor of being a part of, was the HIV
6 Prevention Program, the Cooperative Exchange
7 Program that went on between the Centers for
8 Disease Control and the states.
9 We had 56 people sitting at the table in
10 California from every community over 6 years, and
11 we wrote a prevention plan that won numerous
12 awards, and it really was the authorship of all
13 these communities.
14 In the first few meetings, everybody had
15 their own agenda including me, and we got nowhere,
16 and by the third meeting, a group of us sat down in
17 one of the hotel rooms and said this is going
18 nowhere, people are dying, what do we do, and
19 everybody's guard came down, and everybody's
20 posturing stopped, including mine, and everybody
21 got with the mission.
22 It was an incredible experience. I did it
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1 for 7 years. I ended up 2 years as chair of the
2 statewide committee. I think it's a model we
3 should look at and understand, because it's one
4 that really works.
5 Learning from the past, HIV. Obviously,
6 everyone knows this, but I am going to walk through
7 it. It is important to revisit it. It is not if
8 new and unknown pathogens will present themselves,
9 but when.
10 The issue is coordinated response and the
11 time frame. Inaction ultimately leads to serious
12 injury and potential death for the end users, as we
13 found out with HIV and we are finding out right now
14 with HCV.
15 Openness to new approaches is critical, be
16 they medical approaches, be they policy approaches,
17 principle of self-criticism as very distinct from
18 denial and obfuscation on all sides of the table.
19 Hepatitis C, where did this epidemic
20 originate? We are still not getting answers. How
21 did we get such a high caseload, roughly 4 million
22 we hear from CDC, and we still have not understood
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1 the landscape from where.
2 Long-term historical decisions and
3 assumptions were made and never revisited. I heard
4 talk of acceptable risk or risk communication
5 today. None of us communicated about the risk of
6 hepatitis C. Decisions were made probably in the
7 1960s that resulted in hepatitis C as being seen as
8 an acceptable risk.
9 I can tell you, as those of you that know
10 me know, it is not an acceptable risk. I have
11 lived with it for 35 years, and I am in pretty good
12 shape. People are dying quietly in hemophilia
13 again, in the darkness, without treatment, without
14 care, and without any discussion about it, and we
15 have a problem with that, and we will continue to
16 raise it.
17 Decisions regarding risk must include the
18 consumers. We have made progress in that area, but
19 we need to underline how important it is. CJD, we
20 have been unhappy about the response of this
21 government to CJD right along. We think the
22 British and the Europeans are ahead of the game.
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1 I heard how wonderful our system is.
2 There is no doubt we have a wonderful system.
3 There is no question we have made serious progress.
4 I don't worry about lipid envelope viruses anymore.
5 I do worry about CJD, variant CJD, and other
6 unknowns, and I do worry about the lack of what we
7 perceive of coordination between the blood side and
8 the food side, between FDA and blood, and FDA and
9 food, between FDA and USDA.
10 We are testing a small amount of our
11 cattle. I can get the specific number, it's in my
12 notes, but given the size of the herds, it is way
13 too small in number, and doesn't give us enough.
14 Grass-roots advocacy. The object of the
15 system evolves into the subject of change. We
16 became agents of change. We were the subject of a
17 problem--we were the object of a problem, an
18 epidemic HIV.
19 We transitioned ourselves to become agents
20 of change. Direct access to end users and
21 consumers allows for a clear vision and view of the
22 material conditions on the ground in various
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2 It also allows for the ability to present
3 solid anecdotal information and data regarding end
4 user communities, creative thinking, not narrowed
5 by traditional norms and boundaries is important,
6 peer advocacy programs that emerge from the
7 conditions in the ground in end users' communities,
8 and a needs assessment from those who are actually
9 in need.
10 That is what we did in California, and we
11 still put it on the table as really important. The
12 creation of interdisciplinary approaches better
13 suited to the natural conditions that traditional
14 models may not be. A well-honed psychosocial
15 program that addresses the emotional soul needs for
16 end user communities.
17 I have heard a lot about communication of
18 risk. I have heard a lot also about the IVIG
19 problem. I am not sure, and I think those of you
20 that are clinicians do know this, but I wonder if a
21 lot of you understand the impact on us when we
22 can't get IVIG or we are told we can't get factor.
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1 I am lucky. I haven't had that problem
2 except for once. When I was told by Blue Cross on
3 a Saturday that I had capped out, I had no more
4 coverage, I melted down for two days. Luckily, my
5 father was there, and he had plenty to say, but the
6 fear, the effect on my health.
7 About a week later, I had the bane of my
8 existence with hemophilia, iliopsoas bleeds. I had
9 a rip-roarer. I believe it was directly tied to
10 being told I had no insurance because there was no
11 injury, but there I was back in the hospital.
12 I think when we look at the whole client,
13 not just the physical client, these kind of
14 messages can be deadly. If you are
15 immune-suppressed, you will get sick. Odds are you
16 will pick something up. I think we can't
18 I was glad to hear I think, Dr. Linden,
19 you referred to this in risk communication, and
20 someone else did. I was very glad to hear that. I
21 think it is very important. I think there has to
22 be a continued active role regarding empowered
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1 communities, be they NHF, be they the Federation,
2 be it COG, be it IDF, the value of all these
4 Now, I want to say the most difficult
5 thing I have to say. To all of you that are
6 parents, that is my little girl, that is my
7 youngest daughter. That is her quote. I have a
8 hard time not coming to tears when I look at that,
9 because unlike my twins, who are 32, she never had
10 me without HIV hanging over us.
11 The twins never thought hemophilia would
12 kill me, they figured he will bleed, he will hurt,
13 but when we talk, they say we never thought you
14 would die until we were 13 and you told us. This
15 little girl never knew any different.
16 This is one of the little girls we are
17 servicing. She's a carrier. What about her
18 children yet unborn? She has been lucky. She has
19 one child that is okay, a little boy, but she
20 rolled the dice and I just about freaked, but she
21 explained it to me and I understood.
22 The point is are we still focused there.
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1 Here is what I see, and this is kind of a not too
2 long a conclusion, but a bit of a conclusion. I
3 told our board I do believe this is a committee cut
4 off from its client, and I don't think it's the
5 committee's fault.
6 I told the board I thought the committee
7 was being a bit insular when I saw the words
8 "strategic planning." In my seven years on the
9 California Prevention Committee, I had the honor of
10 working with Patricia Franks, Ph.D., heads up
11 strategic planning for the University of
12 California, and is a brilliant woman, and I had the
13 honor of her deciding that she liked me and saying
14 stick with me and you will learn a lot about
16 Well, I did, and for seven years, from
17 being a chair to a committee chair, I learned about
18 strategic planning. I have seen the word
19 "strategic" today, but I haven't seen the meat of
20 what strategic planning is really all about.
21 I feel, and this is more a feel comment,
22 the committee feels like it doesn't believe it has
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1 the power to change things, and granted, from our
2 perspective, we have had two clients, two
3 Secretaries of Health, that didn't seem as
4 interested as Dr. Shalala was in these issues, and
5 we have all had a rough time trying to keep health
6 on the agenda.
7 But I think what is lacking is leadership,
8 leadership about these issues, leadership about
9 strategic planning. The discussion I heard about
10 IVIG this morning, about immune globulins, I
11 mentioned to Marsha Boyle, we had that discussion
12 in 1998, when the committee was meeting I think
13 right on the Rockville Pike at one of the other
15 Those discussions were deep. That is when
16 everybody was upset that some of the home care
17 companies may have been hoarding or manipulating
18 supply. They were incredibly contentious meetings.
19 Where have we come since '98 on this issue, why are
20 we still talking about allocation of IVIG and
22 If we are really strategic planning, then,
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1 we are going to develop a plan that we pray is a
2 national blood policy and addresses these issues
3 now, so we are not reactive, we are not reacting to
4 a crisis, we are not reacting to a situation, we
5 have this overall plan for the nation.
6 How important is blood to this nation? I
7 can't answer that, but I think we have got to
8 strive harder to find out together. The committee
9 has to believe it can make change, and we have to
10 believe that we can work with you to do it, and if
11 that means those of us in hemophilia that did it
12 for the Ricky Ray bill back on the Hill, and beat
13 the pavement until we get a response, we are ready
14 to do that, but we need an ally.
15 We need an associate, someone to work
16 with, and we are not always going to agree on
17 everything, but I think we do agree that a national
18 policy is called for, and a nation of this size,
19 the world's leading nation does not have a national
20 blood policy.
21 I am not sure how you all feel about that,
22 but we continue to be shocked by that, and
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1 frustrated and ready to go do what we need to do to
2 make it happen, because at the end of the day, even
3 if she wasn't my daughter, I would want to do
4 something about it, but the fact that she's my
5 daughter makes it all the more critical that I have
6 some answers if she has a son with hemophilia.
7 So, I urge the committee to look at some
8 of these issues. I again thank you, Jerry, for the
9 time, and everyone else on the committee for
10 listening, and we are always appreciative to be a
11 part of this process, and have been here since the
12 beginning, and we will continue to be here.
13 The only issue is can we find enough young
14 people to reinvent ourselves and mentor ourselves,
15 because coming in today, I was saying, well, I was
16 a young turk 15 years ago coming in here, and now I
17 am getting to be an old man. It's a little scary.
18 Thank you very much. I really appreciate
19 your attention and your consideration.
20 DR. BRECHER: Any questions or comments
21 for Corey?
22 Okay. Thank you, Corey.
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1 Are there any other public comments at
2 this time?
3 Committee Discussion
4 DR. BRECHER: If not, we can begin sort of
5 our committee discussion. We have several things
6 we can talk about. We can go back to IVIG from
7 this morning. We can talk about the strategic
8 plan. I think it is probably worth spending a few
9 minutes talking about what Corey has just
11 So, what is the committee's pleasure,
12 where would we like to begin? Let's talk about
13 some of the issues that Corey has brought up first.
14 I think we can move that off the table first.
15 I think that his committee's perception
16 that the senior management at HHS is not
17 particularly paying attention to this committee is
18 an interesting observation. I was wondering if the
19 other consumer groups have that same feeling.
20 Maybe Mark for the National Hemophilia?
21 MR. SKINNER: Well, Paul is actually
22 president of NHF now. I don't want to usurp him.
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1 DR. BRECHER: Sorry. Paul, go right
3 DR. HAAS: I guess I am a little sorry to
4 admit that we haven't had this discussion as an
5 organization, but I personally would agree with
6 what I heard Corey say.
7 MR. SKINNER: The only comment that I
8 would add is i mean I think the committee in
9 general was extremely disappointed a couple of
10 years ago with the silence when we put committee
11 recommendations forward and we weren't getting
12 formal responses.
13 I do think that has changed, that we are
14 getting responses. Whether they are actually
15 translating into the actions that the committee had
16 contemplated, I think there is something still
17 missing there, but at least we are getting an
18 acknowledgment that we put a recommendation
19 forward, and there was a period where that wasn't
20 even occurring.
21 DR. BRECHER: Additional comments?
22 Why don't we move to the IVIG question. I
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1 am sorry, Jerry?
2 DR. SANDLER: I will give a personal
3 opinion that when we had a movement toward a
4 national blood policy, I had the feeling that the
5 Assistant Secretary of Health was given the charge
6 of a leadership position.
7 I don't see any leadership coming in this
8 area transfusion safety from above. I think we are
9 more engaged with them with Jerry Holmberg's
10 initiatives than we ever have been, and we are
11 exchanging an awful lot of communication,
12 recommendations, and we get the most wonderful
13 blowoffs I have ever seen, but I don't believe that
14 there is any major leadership in blood safety and
15 availability coming from above.
16 They are responsive to our initiatives
17 with communications that haven't taken a leadership
19 DR. BRECHER: Celso.
20 DR. BIANCO: I am trying to be careful
21 with my words.
22 DR. BRECHER: Aren't we all.
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1 DR. BIANCO: I am going to say what Jerry
2 said, but from a different perspective. I don't
3 think that the Secretary or HHS understands the
4 role of this committee. It has been a long time
5 between the IOM report and what the committee was
6 designed to do today, and I think that we are just
7 one of the committees that raises issues, comes
8 with points, but I don't understand that they see
9 the importance of what we do, and this is my last
10 meeting, so it's okay, I can say that.
11 DR. BRECHER: That's what you think,
13 MR. SKINNER: I just want to make one
14 other comment, because I do think Corey's comments
15 were very timely, and sometimes silence can be
16 misinterpreted either as agreement or disagreement,
17 and I think Corey's comments, particularly at a
18 time when we are talking about strategic planning,
19 bringing the committee back to why we were
20 originally created and for who we were originally
21 created is extremely important.
22 I mean there was very much a compelling
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1 need for the committee at the time we were created,
2 and the IOM study gave us that blueprint, and we
3 have been struggling with what is that blueprint
4 that we are working through an agenda, so we take
5 up a series of ad-hoc issues which are very
6 important, and we have drifted from perhaps that
7 original rallying cause that brought us all
9 It may be a natural evolution, but the
10 purpose of why we exist, I mean also comes from the
11 top down. It came from the outside in, and it was
12 created through the IOM study, and now keeping that
13 agenda focused.
14 So, hopefully, through this kind of
15 strategic planning process, we are going to be able
16 to get back to a template of issues then that we
17 are going to be able to work through, but I think
18 that is what has been missing, is that overriding
19 theme that has compelled us from each meeting to
21 DR. BRECHER: Judy.
22 DR. ANGELBECK: I have to say, as one who
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1 is charged with the topic on integration, I think
2 Corey's comments about the exclusion of the
3 grass-roots community in strategic planning is one
4 that we really need to take to heart, because
5 ultimately, if they are the receivers and the
6 citizens, they need to be part of the process, in
7 my view.
8 I have not been a participant in the
9 committee as a member since its inception, but I
10 have been an observer since its inception, and with
11 respect to that, I would say I think the committee
12 has lost its intensity and direction towards that
14 DR. BRECHER: Sue.
15 DR. ROSEFF: I have one question and a
17 First of all, what is the ability of the
18 committee to do something when we feel we are not
19 being listened to? We may talk about this
20 tomorrow, but with IGIV, we have seen that nothing
21 has changed since our last meeting, and there is
22 concern that things are going to get worse in
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1 January, and we have got no response from the
2 Assistant Secretary, so my first question is, well,
3 what do we do.
4 My second comment is basically I am
5 thankful that one of the issues that didn't come up
6 during Katrina was that there wasn't a blood
7 availability issue, but in a way, that sort of puts
8 blood in the background again.
9 I think what we are always doing is
10 responding to the disaster, and the hope is that
11 with the strategic plan, that we will not be
12 responding to a disaster, that we will have
13 something in place to be proactive.
14 So, I think it is our job to keep the
15 level of the blood supply, availability and safety
16 high on the agenda because again, I don't hear as
17 much about it as I did after September 11th,
18 because it doesn't seem that that has come up to
19 the same intensity.
20 So, first, my question is about what do we
21 do, and, second, is just a comment that I think
22 that the level of looking at the blood supply keeps
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1 dropping when there is not a big disaster upon us
2 that is affecting the blood supply.
3 DR. BRECHER: Jay.
4 DR. EPSTEIN: I think that there is an
5 inherent paradox, if you will, about the role of
6 the committee. It is true that the committee was
7 established in the wake of the IOM report about
8 decision-making in the HIV era.
9 It is also true that the IOM
10 recommendation was for the establishment of an
11 advisory council to the Secretary or to the
12 Department, and I think we need to remember that
13 the committee serves at the pleasure of the
14 Department and that essentially, the Department
15 decides that on which it wishes to be advised.
16 I think that the paradox and the tension
17 comes from the fact that the committee members
18 realize that they also need to lead the charge,
19 that they are not there just to answer the
20 questions posed by the Department, but that they
21 have taken upon themselves, or the committee has
22 taken upon itself a role of sort of taking a
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1 birdseye view and being more proactive on issues.
2 I am just not sure that that role and
3 mission is what is central to the committee
4 charter, and I think that is part of where the
5 tension comes from.
6 On the question is whether the committee
7 is effective, you know, we have had a number of
8 meetings where we have reviewed recommendations and
9 outcomes of recommendations, and I think that what
10 you really have is sort of a good news/bad news
11 story, that on some issues we have been able to
12 prompt quite a bit of response in not just
13 government, but also the private sector, and then
14 on other issues, there has been frustration because
15 we have not been able to see the outcomes that we
16 might have liked or the responses that we might
17 have liked.
18 But I guess my view is just a little bit
19 more colored because I just don't see it as all of
20 one stripe. I simply think we have had our
21 successes and failures.
22 DR. BRECHER: I would tend to agree with
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1 you, Jay. I think just in the last few years, the
2 Interorganizational Task Force, I think it has been
3 a success partly from this committee. I think a
4 lot of the issues over bacterial testing were
5 worked out in this committee. HCV lookback years
6 ago came through this committee.
7 So, I think there have been a lot of
8 successes, a lot of issues of reimbursement have
9 come out of this committee. Not all of them have
10 been resolved to the satisfaction of everyone, but
11 at least it has been in the avenue of getting those
12 opinions out there.
13 Any further comments or questions? Paul.
14 DR. HAAS: It's half a question and half a
15 comment, I guess. I think a major part of what
16 Corey was just saying to us was how do we, as a
17 committee, or maybe the Secretary, receive this
18 information from the grass roots.
19 I think as much as I agree with what I
20 heard Jay just say, and you have just said, in
21 terms of some successes, again, I am going to be a
22 little repetitive here, but the intensity of the
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1 original committee meetings, of which I guess I am
2 one of the few that is still here, that has
3 changed, and maybe that's good, but as it has
4 changed, I will use Mark's term, the focus of what
5 this committee is doing I think has changed.
6 Without the crisis out there, as we had
7 with AIDS first, and then understanding hepatitis,
8 what can we, as a committee, generate that type of
9 focus again, so that we have that type of--I won't
10 say the word excitement--that we had in the earlier
11 years, and I don't know if we can do that, but I
12 think it is an important part of I think what I see
13 this committee doing is keeping aware of those
14 issues just like the IVIG business coming through
15 here, and we want to stay focused on that.
16 DR. BRECHER: Yes, it is sort of like do
17 we really want to live in interesting times.
18 Other comments, questions? Merlyn.
19 DR. SAYERS: Corey and I go back to the
20 circumstances that you were talking about when
21 tension filled the air, and an urgent need to be
22 active was felt by everybody.
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1 I think one of the things that has
2 happened during the embryology of this committee is
3 that the sense of urgency has been reduced largely
4 because of gains in transfusion safety.
5 When we were talking about
6 transfusion-transmitted HIV, there was an
7 understandable national anxiety. It is not as easy
8 to develop as much energy talking about
9 transfusion-transmitted ehrlichiosis.
10 I think that is one of the sets of
11 circumstances which distinguishes our behavior now
12 from then. One other thing, Corey, and I have said
13 this to you before, when I have heard you talk, I
14 am sometimes left with the sense that somebody that
15 has an M.D. immediately has a net degree of filter,
16 which prevents him or her from understanding what
17 the issues are at the grass-roots level, and I
18 can't agree with that, essentially because many
19 physicians are themselves transfusion recipients
20 and dependent on transfusions, and many physicians
21 are treating physicians, and they certainly are
22 sympathetic, if not because they are transfusion
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1 recipients themselves, but certainly because they
2 might be treating individuals who are transfusion
4 So, I don't think an M.D. degree or Ph.D.
5 degree really superimposes some sort of censure on
6 your understandings.
7 DR. BRECHER: Art.
8 DR. BRACEY: One of the things that I have
9 just been thinking about as we have had this
10 discussion, clearly, what sparked this was adverse
11 outcomes released to transfusion, but the other
12 reality is that blood is to medicine as oil is to
13 armies. You can't fight a war without oil. There
14 are many things that you can't do in medicine
15 without an adequate blood supply.
16 I would think that the higher ups, if they
17 began to have some sort of strategic vision, would
18 see this and therefore would see that the work of
19 this committee, perhaps, you know, they are focused
20 on its origin as opposed to other possible
21 destinations, so again, to me, I think the key now
22 is to look at blood as a resource and to begin to
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1 focus on the good things that it can do and the
3 You know, we are in an era of advancing
4 aggressive medical therapies. We won't be able to
5 provide those therapies if we have an inadequate
6 supply. This is something that I think that the
7 higher ups would understand.
8 DR. BRECHER: Unfortunately, sometimes it
9 seems like you have to have a headline in the
10 Washington Post of the New York Times to get their
13 DR. EPSTEIN: I tend to think that it's a
14 good thing that the committee has evolved to taking
15 a global perspective about our system as a whole
16 and how it works in all its parts. I think that we
17 are in a position to do more long-term good from
18 that perspective than dealing, you know, urgently
19 and in a crisis mode with particular issues that
20 are pressing, not that that is unimportant when
21 important issues are pressing, we deal with them,
22 and we should, but isn't it a good thing to be able
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1 to take a step back and ask what are the problems
2 with our system and how can we make our system run
4 The second point I would make is that a
5 strategic plan for the Department to undertake
6 shouldn't be thought synonymous with a strategic
7 plan for the committee. I think it's an open
8 question what role this committee is advisory to
9 the Secretary should play in any such plan should
10 it emerge. It is not at all clear to me that it's
11 a plan that the committee should assemble, or the
12 committee should oversee, or the committee should
13 try to establish. I tend to think not.
14 Lastly, I think that Corey has again
15 reminded us of a very important thing, which is
16 that we shouldn't be out in the ozone, that the
17 concerns of the patients and the product end users
18 are our core business, and I think that is correct,
19 and I think that if we approach the development of
20 a strategic plan, it can't be in a vacuum. It has
21 got to be with a very real-world consideration of
22 how are people being affected in their daily lives
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1 by what we are doing with the U.S. blood system and
2 all its elements.
3 So, you know, I resonate to that very
4 strongly, and I agree that the empowerment of the
5 consumer community, the patient community, and the
6 advisory committee processes has been a tremendous
7 advancement in public policy.
8 I think that we don't want to lose that
9 element even if we now find ourselves, you know,
10 speaking calmly.
11 DR. BRECHER: Celso.
12 DR. BIANCO: Corey woke us up, and I think
13 that this is a wonderful opportunity, coinciding
14 with what we think now in terms of a strategic
16 I slightly disagree with Jay on who should
17 conduct such an effort, not necessarily the
18 day-to-day of getting days and nights talking about
19 the actual strategic plan, but I think that it is
20 necessary. This committee is supposed to set
21 national policy in blood, and it is necessary that
22 at least a guiding principle is the overall strokes
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1 be set by this committee.
2 It, I believe represents a lot of the
3 people involved, is an open committee. There are
4 many patients in the committee. By the way, I am a
5 transfusion recipient and lots of units, and the
6 public has access to this committee. So, at least I
7 think that the effort that we had this morning,
8 even if possibly or probably we didn't hit all the
9 right keys, is an initial effort, and we have to
10 put out, not necessarily the answers, but all the
11 right questions.
12 We don't have to respond to emergencies
13 only. That is what we have done always in the
14 past. I think that we have to ask ourselves are
15 those questions going to help us if we answered
16 them to do things right in the future, and I think
17 that is our role.
18 DR. BRECHER: Other comments or questions
19 on the subject? Corey, we are listening. We have
20 been listening to you.
21 MR. DUBIN: Two things. Merlyn, I would
22 never draw a line. You protected me on the podium
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1 when Paul Holland wanted to make a mess, and you
2 stepped up and said it wouldn't happen, and it is
3 not that I see a difference, because I don't,
4 because at the height of the crisis, you and I were
5 delivering a talk together when most people thought
6 we and you guys wouldn't talk to each other.
7 So, I didn't mean to juxtaposition it in
8 that way. I think we need to continue to work,
9 docs, us, researchers, CMS. Dr. Bowman, I would
10 love to hear more from you. I would love to
11 understand CMS better.
12 I look out. Jay, you know how I feel. I
13 think you are one of the best people out there in
14 the government, and it is not that I think the
15 committee should be the be-all, end-all, but I
16 think it screams for leadership, and I know there
17 is such good people at this table that know how to
18 lead - Celso, Jay. I mean I could go down the list
19 around the table.
20 So, I think we are calling for leadership.
21 Maybe guidance would be a better word, Jay, that
22 would be more comfortable, because I agree with
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1 you. The Secretary can wave his or her hand, and
2 it's over. We know that, but we also think you all
3 have so much credibility in the game, so to speak,
4 and we are ready to do what we can to support that
5 with the Hill, and, look, we may be small, but we
6 accomplished something on the Hill nobody said we
7 could ever do, and together we did it, all of our
9 So, I think from us, it's just a call, and
10 I don't want to go back. Somebody said thank God,
11 it's not I think the dialogue of the late '80s and
13 I don't want to go back to HIV, but there
14 are two crises out there. One is reimbursement,
15 and reimbursement is almost like the controlling
16 for allocation, and that is a crisis, and we are
17 all frightened about that, and hepatitis.
18 I really appreciate that you all
19 considered our words very carefully. That is clear
20 to us, and we will continue to be in the process as
21 long as we have got some breath going, and then
22 hopefully, my daughter will be standing up here,
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1 and she's tougher than I am, look out, but thank
3 DR. BRECHER: Jan, did you want to say
5 MS. HAMILTON: Thank you. I just wanted
6 to say several things were said this afternoon
7 about blood policy, and I was just trying to ask--I
8 can't remember how many years ago it was, but a
9 comment, I don't know, Celso, if it was you, or
10 somebody over here, said that this group should be
11 setting the blood policy.
12 I went to a meeting, I believe it was in
13 2000, if I am not mistaken, it was held by the CDC,
14 and a whole bunch of us sat in a room all day long
15 and talked about whether the national blood policy
16 needed to be updated, and nothing was done.
17 I sat here thinking why wasn't that being
18 done here. So, I think, if nothing else, you know,
19 I mean we support a lot of Corey's statements, and
20 things that Paul and Mark and everybody have said,
21 and I sat here and listened a lot of times when
22 this committee deliberated for long hours and never
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1 got an answer from one meeting to the next, to the
2 next, from the Secretary.
3 I see that changing to some degree, and I
4 am delighted with that, but maybe that's a good
5 project for 2006 for this committee, is to look at
6 the national blood policy. I mean it still says
7 something about plasma, and doesn't go any farther
8 than that, and that is sad.
9 We should be talking about the future and
10 about the things that we have, instead of just
11 going back just to plasma. I think you are right,
12 whoever said it, this is the place for that to be.
13 DR. BIANCO: Jan, it hasn't been revised
14 in 35 years.
15 DR. BRECHER: Any other comments or
16 questions? Why don't we take a 15-minute
19 DR. BRECHER: Could the committee members
20 take their seats, please.
21 We have two major topics to cover of the
22 remainder of today and basically, all day tomorrow,
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1 and that is, number one, coming to some conclusion
2 about the message we want to put forward about
3 IVIG, and, number two, the strategic plan and
4 policies for mitigating adverse diseases and other
5 things that could come into the blood supply.
6 So, I would suggest that we start with the
7 IVIG question. We have made strong recommendations
8 from this committee to the Secretary on two
9 occasions. I think that they have heard the
10 message, although we do not see definitive action
11 as yet.
12 I see us as having two choices. One, we
13 can come up with yet another resolution; or, two,
14 in the letter to the Secretary, well, we are going
15 to almost certainly make some sort of resolution
16 about the strategic plan.
17 We could simply state that the committee
18 remains concerned or even gravely concerned
19 regarding availability and reimbursement for IVIG,
20 and request that policy alternatives be considered,
21 and that is not a resolution, but I think it would
22 get the message across.
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1 So, I would be interested in hearing
4 MR. SKINNER: I do think there is one
5 thing that is new since we last met. I mean other
6 than there is more information than that, you know,
7 the pricing system does not work to support the
8 needs of the patients, the reimbursement system,
9 but the piece that I honed in on in the Secretary's
10 response, in the April 8th letter, was that they
11 find that there is sufficient supplies available
12 for the patients and that is marketplace
14 I am not sure whether or not the
15 information that we have heard agrees that there
16 actually is a sufficient supply out there. If it
17 is sufficient, it undoubtedly is extremely tight,
18 and there isn't much margin. So, I think the word
19 "sufficient," probably is overly generous.
20 There may be a supply out there if you are
21 in the right place at the right time, but I think
22 the evidence is it is getting tighter, and I think
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1 the new piece that we learned at this meeting, or
2 that has at least transpired since the last
3 meeting, we may have learned it before this
4 meeting, is that the companies have gone onto
5 allocation, which is further evidence that, in
6 fact, there is a supply problem. The companies
7 wouldn't have got into an allocation or a rationing
8 system in terms of the distribution of the product
9 if there was a supply problem.
10 So, the point that the Secretary came back
11 on, which I assume is the reason, then, that they
12 didn't choose to go forward with declaring a public
13 health crisis, was the supply piece.
14 So, my thought was that we should respond
15 by saying, you know, that there is, in fact, a
16 supply program, and it is further evidenced now by
17 what is happening in the marketplace in terms of
18 allocation, and then underscore and go back and ask
19 them to revisit the alternatives, which may include
20 declaring a public health emergency, so that we can
21 take short-term action until the reimbursement
22 pieces and the pricing and the data can catch up
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1 with what is occurring in the marketplace.
2 DR. BRECHER: Paul.
3 DR. HAAS: This really follows on Mark's
4 point. I think another piece that was driven home
5 today was the issue of where the treatment is
6 taking place. It is shifting, and those of us that
7 are accustomed to home treatment type of
8 phenomenons know that that shift is not good for
9 the patient.
10 And then to your question, Mark, about
11 should we attach this concern onto another, if we
12 think the IVIG is a is a significant concern, and I
13 happen to think it is, I prefer separate messages.
14 DR. BRECHER: Art.
15 DR. BRACEY: One other part that concerned
16 me is that in terms of the shift, there is an
17 assumption that the shift will, in fact, occur, and
18 one of the things that I was thinking of is that
19 clearly, since there is the capability of
20 contacting places where this shift would occur,
21 i.e., at the hospital settings, et cetera, would be
22 to ensure that we are not working on an assumption,
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1 so that we would end up in a reactive mode, but if
2 we could sort of prospectively go out and find out
3 if, in fact, this new business model would be
4 acceptable to those places where the shift is
5 assumed to go.
6 DR. BRECHER: I think also we have to be
7 clear to state that this is a non-sustainable
8 shift, that come January 1st, 2006, even this shift
9 will not support the patients.
11 MS. LIPTON: That is my concern, that
12 looking forward we don't really know what is going
13 to happen, and we are assuming that the shift is
14 occurring, but no one really was able to answer the
15 question that I think Jerry posed, which is are
16 those supplies that we previously went to the
17 physicians' offices, are those indeed being
18 allocated now to hospitals, and are they being
19 allocated to hospitals where they expect those
20 patient populations to show up.
21 You just have the feeling that there might
22 not be an overall supply problem, but you just get
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1 the feeling it is not showing up either where it is
2 supposed to, and it is causing serious problems for
3 those patients.
4 I will say it again, I said we keep taking
5 actions, and we don't realize what the tail is on
6 the end, and I almost think we are harming things
7 without stopping and saying, look, you have to look
8 ahead here, and if we don't think ASP plus 6 is
9 going to work in the primary care setting, why do
10 we think ASP plus 8 is going to even remotely work
11 in the hospital setting.
12 DR. BRECHER: Jerry.
13 DR. HOLMBERG: Julie, are you in the back
14 there? Can I ask you a question, please?
15 Allocations, when did they go in effect? I thought
16 that they were in effect way before our May
18 MS. BIRKHOFER: Yes, each company has made
19 their individual decisions based on their business
20 practices to put in place allocation, which again
21 is based upon historical order volumes. PPTA, as
22 you know, maintains a data gathering program, and
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1 for IVIG, since January, the data has been in the
2 yellow light, which is approximately four weeks of
3 inventory is available.
4 Comments made to the fact that the market
5 is dynamic and changing, and that companies have
6 streamlined their distribution practices is
7 evidence that four weeks in this market may be
9 So, from PPTA's industrywide data, there
10 is not a shortage, there is not a supply issue. We
11 are in the yellow. Yellow means four weeks. It
12 does not mean that there are shortages.
13 MS. VOGEL: Hi. Just to react to that
14 statement, IDF receives calls from all different
15 sites of care. At this point, I mean it is not a
16 matter of just a tightening market. I mean we
17 could talk about it in different terms.
18 The calls going, in the first place, about
19 shift of site of service, you are right. I mean
20 the allocations, I mean every product is on
21 allocation. When you are shifting a huge number of
22 patients from one site to another, the allocations
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1 don't follow the patients. So, the hospitals are
2 getting increases there.
3 However, and this is a big however, we are
4 seeing a trend right now of allocations being
5 reported into IDF being cut by 20 percent, and that
6 has nothing to do with the increase in Medicare
7 patients. Don't know why that is happening, it
8 could be with Red Cross leaving, exiting the
9 marketplace, I am making assumptions at this point
10 because I am not an expert on the supply area.
11 I am just reporting back to you what we
12 are hearing, but there is many, many hospital
13 systems who are taking on these patients who don't
14 have either product because of the increases, or
15 are talking about allocations being cut.
16 So, in this scenario, the best situation
17 is to get patients back into the right sites of
18 service and to treat them. Until we do that, we
19 won't know the true seriousness, if we have a true
20 supply problem or not. We have to get them where
21 they need to be treated, and at that point, we will
22 be able to tell if there are supply issues.
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1 DR. BRECHER: Julie, did you want to say
2 anything? Okay.
4 DR. EPSTEIN: I would like to ask Julie a
5 question. Can you confirm the assertion that the
6 distributions have been flat for the last six
7 months or so, because I think part of what concerns
8 me is that there was an historic trend of steadily
9 increasing utilization, and what has happened is
10 that in the face of that trend, there has been for
11 at least the last six months, flat distribution.
12 So, on the one hand, it may be that there
13 is, if you will, not a supply crisis in the sense
14 that there is enough supply for well-established
15 indications, for example, but the problem is, is it
16 sufficient in the face of the historically
17 accelerating demand, or is there a deficient supply
18 relative to the demand that exists, in other words,
19 we can't meet all prescribers' needs even though we
20 probably could for some subset of those
21 prescribers' needs.
22 I think that is part of it.
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1 MS. BIRKHOFER: I would completely agree
2 with you, Dr. Epstein. Distribution has remained,
3 as you say, flat, somewhat aligned over the past
4 six months. Demand, we know has increased 6 or 8
5 percent, and the companies, given the manufacturing
6 processes that it takes 6 to 9 months to bring
7 these therapies to market, the companies are all
8 individually looking at ways that they can
9 manufacture more.
10 But we can't make that prediction. All we
11 can base our comments on is what our supply data,
12 industrywide supply data shows, and also non-PPTA
13 member companies report this data, and again we are
14 showing inventory in the yellow consistently.
15 DR. EPSTEIN: If I could press the point a
16 little bit more, the yellow zone was defined, after
17 all, arbitrarily. In other words, how is the
18 supply stratification of red light, green light,
19 yellow light, designed in terms of demand? In
20 other words, what makes yellow yellow in comparison
21 to effect of demand? How do you know that what you
22 are calling yellow isn't really red?
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1 MS. BIRKHOFER: That is one of the things
2 that the PPTA North American Board of Directors is
3 looking at. The traffic light system and the
4 ratios that trigger those lights, that was put in
5 place about six years ago, working with member
6 company representatives, as well as Georgetown
7 Economic Services, GES.
8 Georgetown Economic Services are Ph.D.
9 economists that help look at the market, and
10 basically, they put ratios in place where about
11 0.25 equals about one week of supply, so right now,
12 when I say we are in the yellow, that is a ratio of
13 between 0.6 and 1.24. 1.25 and above is green and
14 0.5 and below is a red light.
15 Again, you know, these ratios were put in
16 place five, six years ago. What PPTA is looking at
17 now is yellow or new green based on the current
18 market dynamics, but that is something that we
19 can't change the ratios now, you know, as
20 arbitrarily. We need to have deliberation, we need
21 to work with the economists, we need to relook at
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1 I mean we can't, in the midst of this
2 question of is there supply issue, is it
3 reimbursement, you know, the perfect storm, is it
4 demand. We need to give this time to let the
5 market play itself out.
6 DR. BRECHER: I guess what people are
7 concerned about, it may not be red, but maybe it's
10 DR. BIANCO: Actually, for Julie, did I
11 understand you correctly that the ratios, they are
12 not adjusted for the increase in demand?
13 MS. BIRKHOFER: The current system in
14 place was put in place about 5 1/2, 6 six years
16 DR. BIANCO: So, you are using a week,
17 what was used, the IVIG that was distributed during
18 a week 5 or 6 years ago.
19 MS. BIRKHOFER: That's correct.
20 DR. BRECHER: Michelle.
21 MS. VOGEL: I would also like to make one
22 other comment. The other thing that is being
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1 reported in to us, many hospital systems are
2 starting to put in their disease state management
3 programs and putting pecking orders in place based
4 on who should receive IVIG first because of supply
5 issues in those hospitals.
6 So, that also brings concern issues to the
8 DR. BRECHER: Jerry.
9 DR. HOLMBERG: Just to point out on that,
10 that fact, in my discussions with the pharmacists
11 that have put in various prescription reviews, that
12 really the labeled uses are going first, and that
13 that is a high priority.
14 Actually, when they get a request for an
15 off-label use that does not match one of even the
16 30 that CMS has added to, that what they have done
17 is they then take it internally within their own
18 review process, but the pharmacists that I have
19 talked to in representing large hospital
20 organizations, have said that having this mechanism
21 in place has ensured that the people that need to
22 get the product get the product first.
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1 Can I add another comment? I see two
2 other issues here. We did hear comments--I could
3 guess three different issues that I would like to
4 talk about, and that is that, first of all, we have
5 heard this morning that there has been an increase
6 in the albumin utilization, which also drives the
7 economics on the manufacturer side, which may also
8 help correct the market.
9 But then also with the ITP, I saw that in
10 the booklet that AmerisourceBergen has given us,
11 that ITP accounts for 8 percent, and isn't there a
12 new course of therapy for the ITP that will be
13 moving away from the use of IVIG for ITP? So, is
14 there a potential gain of 8 percent?
15 DR. WONG: Are you talking about WinRho?
16 DR. HOLMBERG: Yes.
17 DR. WONG: There is a choice between the
18 two, and the side effects are different. So, some
19 patients may opt not to use WinRho even though, in
20 our hospital, it's the first line for ITP, because
21 of the IGIV issue.
22 DR. BRECHER: Of course, it only can be
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1 used on the Rh-positive individuals.
2 DR. WONG: Yes, but most people are.
3 DR. BRACEY: The other thing is that
4 recently, there are some negative reports in terms
5 of risk associated with WinRho, that are beginning
6 to come out, and I would think that that is going
7 to impact, to only increase IVIG requests.
8 DR. WONG: To clarify, the negative
9 reports were on intravascular hemolysis, is that
10 what you are alluding to? Yes, that is still under
11 investigation right now. Most of us have not seen
12 that. I just came from an expert panel looking
13 into the side effects. So, we are still monitoring
15 DR. BRECHER: I guess there also is
16 another IV preparation of anti-D that is on the
17 market, although I don't think it is approved for
18 the ITP indication as yet.
19 DR. HOLMBERG: There was a third point
20 that I wanted to make, and that is that we still go
21 back to what is ASP, and ASP is the average sales
22 price coming from the manufacturer.
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1 That is being calculated and monitored.
2 Now, it doesn't take a rocket scientist to be able
3 to figure out what is happening between the
4 manufacturer and the pharmacist.
5 Obviously, there is somebody in between,
6 and so how do we get a handle on the prices,
7 because I get reports every day that sometimes it
8 is up to $118 a gram, $120 a gram, and so that is
9 not coming from the manufacturer or else we would
10 see an increase in the ASP.
11 So, there is a problem here in the
12 distributor. Now, the manufacturers have two
13 different choices. They can go either through
14 their distributor or I think the AmerisourceBergen
15 says the unencumbered pathway, and through the
16 unencumbered pathway, that may be the free market
17 or the spot market approach.
18 But the bottom line is how do we get from
19 a system where it is being reported to CMS one
20 price, but then when it goes through a secondary
21 hand, there is an increase in price, and I think
22 that that is what we are all struggling with.
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1 DR. BRECHER: Karen.
2 MS. LIPTON: It is interesting you raise
3 that, Jerry, because I was struck by that, too, as
4 I was leafing through, and it says unencumbered,
5 which are mostly the primary care physicians'
6 offices are the ones who do not have a contract
7 price, so they are really floating more and go
8 through the distributors.
9 But again I think that that situation
10 still comes back to, that means that ASP probably
11 doesn't even work in a setting when you are dealing
12 with a primary care physician, because maybe their
13 prices are so volatile.
14 No matter what, it still affects where the
15 patients can get care, and I think that is what our
16 concern is.
17 DR. HOLMBERG: I think of one of the
18 things that I have heard from the grass-roots
19 people have been, especially clinicians treating,
20 is that shifting the patient from one location to
21 another, the iatrogenic problems, the infections,
22 and one physician that I talked to said yeah, you
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1 know, I did get treatment for this patient, but he
2 wound up one month on IV antibiotics. That is
3 another side effect.
4 So, you know, what are we doing here, and
5 also I think that Marsha Boyle, I think that you
6 did mention in one of your notes there about the
7 cost, that somebody had made a comment that it was
8 like 600-some plus dollars. I am sorry?
9 MS. BOYLE: It is much more expensive in
10 the hospital from what we are hearing.
11 DR. HOLMBERG: And that is because it is
12 under the AWP at 83 percent.
13 DR. WONG: Do we have any idea how much it
14 cost ASP Plus 10 percent, plus 15 percent for the
15 physicians to be able to administer it?
16 DR. BRECHER: That's a good question,
17 where would it break even?
19 DR. SANDLER: I would like to make three
20 comments. The first one relates to the letter that
21 we got back dated August 8th, addressed to you, Mr.
22 Chairman, and signed by the Acting Assistant
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1 Secretary of Health.
2 The third paragraphs says, "that after
3 discussions, we concluded that there are sufficient
4 supplies available." But when you get to the
5 fourth paragraph, the Assistant Secretary says, "We
6 believe that physicians should ensure that priority
7 be given to FDA-labeled uses and those diseases and
8 conditions that have been shown to benefit based on
9 safety and efficacy."
10 I find a little disconnect here, because
11 that last statement is, in effect, saying that the
12 current conditions require that we tell doctors not
13 to treat patients the way they best attempt to do
14 so. I mean a physician orders IVIG off label is
15 not doing something bad.
16 The FDA-approved indications evolve from
17 the experience that has been derived by treating
18 people in this way, and there are many people being
19 treated in my hospital with IVIG off label, who are
20 absolutely getting benefit.
21 So, going off label isn't a bad thing.
22 The second point I would like to make, I
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1 work in a hospital. I have been working in
2 hospitals most of my career. It is absolutely not
3 the optimal place for a doctor who is following a
4 patient with a primary immune deficiency disease to
5 be sending his patient.
6 Most patients wait for their appointment
7 to talk to their doctor or to talk to the case
8 manager or to the nurse practitioner, and at that
9 point say, by the way, you know, I have been having
10 this or that happening, and I was kind of waiting
11 until I come in.
12 The transfer of these patients is putting
13 them in a situation where they are going to be
14 losing contact on a regular basis with their
15 primary caregiver, and this is not a shift that
16 should be driven by economics. It is not going in
17 the right direction.
18 The third point I would like to make is
19 this whole issue I think underlines what Mr. Dubin
20 was pointing out in his very first opening
21 statement, which is, isn't there some loss of
22 connection between this committee and the people
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1 and the higher ups who make decisions.
2 This is something we communicated was
3 really urgent. We said this is really urgent, and
4 the people who are making the decisions are
5 handling this with a 5-page paragraph letter
6 saying, well, we heard what you say, but we have
7 done some other stuff, and our advice from what we
8 have done overrides the advice you are giving us,
9 which is exactly what Mr. Dubin was trying to say
10 about the discounting of the importance of this
11 committee at a high level, and this is a very good
12 example of how that discounting is taking place.
13 DR. BRECHER: Jerry.
14 DR. HOLMBERG: Dr. Sandler, I sort or take
15 a different view on some of your comments that you
16 have made there, and that is that in taking the
17 recommendation, there was a lot of investigation
18 done on the whole supply and demand and
19 reimbursement issue.
20 I can say that this is one reason why I
21 follow up on every call that I get on a complaint
22 that supply is not available, because when I do
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1 follow up on it, the supply becomes available.
2 So, you know, I don't understand why a
3 phone call has to be made to shake something loose,
4 what other dynamics are going on here, and so
5 really based on the evidence that has been
6 presented to the Department, yes, we have a
7 tightening of the market, but I don't think we have
8 a supply issue.
9 DR. SANDLER: Well, I want to go back to
10 that statement that says physicians should ensure
11 that priority be given to IVIG treatment for
12 FDA-labeled uses and conditions.
13 Inherent in that statement is it looks
14 like we are in a situation where doctors shouldn't
15 prescribe this medication the way they think they
16 should for all of their patients. That is what
17 that is saying, and it seems to me that it would be
18 really great if it said the United States of
19 America, with all of its resources, has enough IVIG
20 to provide for all of the patients including those
21 that doctors feel deserve it.
22 DR. BRECHER: All right, Committee, what
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1 are we going to do?
3 MS. LIPTON: I guess one of the things
4 again what we heard today is really this issue of
5 patients moving, so if we said something else, it
6 really is that patients can't receive the care that
7 they need to get in the primary physician's office,
8 and we don't know why, but that trend has not
9 stopped, and there still seems to be an erosion in
11 Now, maybe we can't really weigh in on
12 what we think it's the reimbursement or we think
13 it's the supply problem, because I am beginning to
14 think we don't really know if it's a supply
16 It certainly seems to be, if it's not
17 supply, an allocation. What we don't really
18 understand is reimbursement driving that issue or
19 is there something else at work. But at a minimum,
20 it seems to me we could still send an urgent
21 message that we have seen no positive change in the
22 very disturbing trend of patients being removed
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1 from their normal primary caregiving setting, which
2 we believe is beneficial to the patient, and it is
3 being transferred over to that hospital setting,
4 and we don't think that is in the best interests of
5 these patients.
6 DR. BRECHER: We also can reiterate that
7 the impending change in reimbursement in the
8 hospital setting will make this shift to the
9 hospitals non-sustainable, or we anticipate that it
10 is not sustainable.
12 MS. VOGEL: DR. Holmberg, you have done a
13 great job in following up on all these cases, and I
14 thank you so much. Just looking forward, I mean
15 January 1st, when the prices go down, no matter
16 what the supply issue is, you can make all the
17 calls you want, it is not going to open up the
18 doors to these patients.
19 So, I agree with what your statement is,
20 going forward with that. The other thing is what
21 we can say we do know is that since this past
22 January 1st was when we started seeing the shift in
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1 patients. When CMS increased their rates a little
2 bit January 14th, we saw a little bit of a
3 hesitation and patients were okay.
4 Once April hit and the products were
5 separated, and the prices crashed, all of a sudden
6 the shift happened dramatically overnight and
7 continued to spiral downward. So, no matter what
8 is going on with supply, we can definitely say that
9 reimbursement has affected the transitioning of
10 patients, and we know that this transition has
11 happened in Medicare patients, and is not happening
12 in the private insurance market.
13 So, in that, we can say that ASP plus 6
14 percent has caused this.
15 DR. BRECHER: Mark.
16 MR. SKINNER: Can I ask one more question?
17 I am going to try to ask, and maybe it's what Dr.
18 Sandler was getting to, and maybe this is a
19 question for Julie.
20 If the companies were not on allocation,
21 would there still be a 4-week supply, would they
22 still be in yellow, or is it the allocation process
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1 that is actually creating the artificial yellow
2 light--I should say is creating the yellow light
3 that is artificial?
4 MS. BIRKHOFER: That is a very difficult
5 question, and I really don't have a crystal ball, I
6 can't answer it. I can only tell you what I know
7 today, and based on our data today, there is
8 sufficient inventory that translate to 4 weeks, and
9 just to clarify Dr. Bianco's question, although
10 these ratios were put in place 5, 6 years ago, they
11 are obviously updated monthly. The ratio
12 represents distribution divided by the annual 12
13 month of inventory.
14 So, they are updated, they are rolling, if
15 you will, but I can't speculate, I just can't.
16 DR. HOLMBERG: Just to follow up on what
17 Michelle Vogel commented about, and again on what
18 Dr. Brecher has already suggested as far as
19 wording, any of my comments I really, you know, I
20 am trying to address the immediate need, and yet
21 what is in the back of my mind, and what we have to
22 keep thinking about, is what is going to happen
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1 January 1st, and I fully agree with that, is that
2 costwise, what is it going to cost the U.S.
3 Government for Medicare patients when they get
4 shifted over to greater than 24-hour care under a
6 I have not been able to get the answers
7 for that, but see, that's the next shift that you
8 are going to see, I mean as far as my opinion, in
9 predicting what is going to happen, is that you are
10 going to see--you have now seen it go from the
11 infusion centers, home care, physicians' office, to
12 the hospital outpatient. Then, it's going to shift
13 to the inpatient under a DRG.
14 That is a concern, and I don't mean to
15 discount, with some of my comments, the fact that
16 we need to be looking forward to what will happen
17 January 1st.
18 MS. VOGEL: And I agree with you, Dr.
19 Holmberg, and what is really scary with that is,
20 you know, medical necessity, do you have to be
21 admitted as an inpatient to receive IVIG, and the
22 answer is no, but what will it take before you can
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1 be, and how sick do you have to get before Medicare
2 will cover you as an inpatient to get your
4 At that point, it will be too late, so it
5 really gets to the point of how many patients are
6 we going to allow die before something can change.
7 I mean there are certain things that we can try to
8 prevent for the hospital reimbursement, what has
9 happened in the physicians' offices have occurred.
10 The only thing that can change that
11 immediately is the Secretary either declaring a
12 public health emergency or Congress making a
13 statutory change. I mean those are two options
14 right now for physicians' offices.
15 The other option for hospital outpatient
16 right now to prevent the hospitals from crashing is
17 for CMS to look during the proposed rulemaking and
18 to state either one of the options that we have
19 talked about, an add-on payment or dampening
20 provision or separating out these HCFA codes if
21 they are willing to do any of that.
22 If not, they go with ASP plus 8 percent,
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1 we have a disaster on our hands, and then what
2 happens in going to inpatient and when will the
3 hospitals be willing to allow the patients to be
4 admitted. So, we have a serious spiraling effect.
5 We know that there are many private
6 insurance companies have dropped their rates,
7 Medicaid has dropped their rates, the Federal
8 Employees Health Benefit Program has dropped their
9 rates, and we know Medicaid reform is about to
10 occur, that is going to mirror what Medicare has
12 So, for a population that is so fragile,
13 that needs this one therapy and can't get it, it is
14 devastating, and I don't understand what we need to
15 do to get this to change, but I am just hoping that
16 this committee will stand strong and stand behind
17 your recommendations that you made in May, and
18 continue to help push this.
19 We will continue to work with Congress to
20 push their support for whatever needs to go forward
21 and to get legislative change, but it is helpful
22 with your recommendations.
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1 DR. BRECHER: I think it is time to write
2 something, burn up a few pixels. We can either
3 just start by asking for suggestions, that we take
4 a five-minute break pull the subcommittee together,
5 throw a few words together to begin with, and then
6 we can play with that.
7 I would suggest that we get a core group
8 of maybe five people, maybe Paul, Karen, anyone
9 else, Jay. Jay is writing, even better, let's give
10 Jay five minutes and we will come back.
12 DR. BRECHER: The suggested initial
13 wording for this resolution, principally authored
14 by Jay, reads as follows:
15 The committee remains highly concerned
16 that disruptions to access for IGIV, including a
17 shift to hospital-based therapy, continue to
18 compromise quality of care for many patients. We
19 further are concerned that a change to hospital
20 outpatient reimbursement, to ASP plus 8 percent,
21 effective January 2006, will further aggravate an
22 already difficult situation and that this shift
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1 will not be sustainable.
2 We therefore recommend that the Secretary
3 take immediate steps to:
4 1. Increase reimbursement for
5 non-hospital IGIV therapy to a level consistent
6 with current market pricing.
7 2. Reconsider the current program to
8 hospital outpatient reimbursement to ASP plus 8
9 percent in January 2006.
10 3. Re-examine the extent to which current
11 IGIV supplies are or are not meeting demand.
12 So, we are open to suggestions.
14 DR. SANDLER: In our letter, we urged the
15 Secretary to declare a public health emergency, so
16 as to enable CMS to apply alternative mechanisms
17 for determining reimbursement schedule, et cetera.
18 Wouldn't that be a necessary component if
19 we wanted some action, in other words, shouldn't
20 this immediate request get back to that we are
21 requesting a public health emergency, so as to get
22 this stuff done?
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1 DR. BRECHER: I don't know. Jerry, does
2 it require an emergency, a crisis?
3 DR. HOLMBERG: Well, I think that the
4 letters that you have already seen from Congress,
5 that was provided by the IDF, and then also the
6 letter from the two congressmen, that I provided
7 you, it does show that Congress is very concerned
8 about this.
9 The thing is, though, that will a public
10 health emergency correct the problem, or will a
11 congressional change correct the problem, and you
12 have to understand that CMS's hands are tied.
13 Now, the public health emergency can
14 change some things, but it will not be a long-term
15 fix, and the thing is that I am concerned about is
16 that the direction here of calling a public health
17 emergency when we--well, first of all, when
18 Congress needs to look at the issue, and secondly,
19 I think that the letters that have been received
20 from Congress has caused CMS to very carefully
21 consider some of the changes.
22 Now, saying all that, I would stay away
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1 from a public health emergency, but I think that it
2 needs to be strong enough to be able to get the
3 message across that CMS needs to work through their
4 legislative avenues.
5 DR. BRECHER: Mark.
6 MR. SKINNER: I tend to think that the
7 concept of declaring a public health emergency
8 needs to stay on the table at this point. Between
9 now and January, there is not much time, and the
10 problem is only to get worse, and to expect
11 Congress to enact new authorization, or to take
12 action for CMS to change something in three months,
13 and to have it in place, to me, seems a little bit
15 I do recognize that the public health
16 emergency is a short-term solution or bandaid
17 solution until the real thing can occur, but I am
18 not sure that we shouldn't continue to argue that
19 all of the powers be used, because the situation is
21 DR. BRECHER: Jay.
22 DR. EPSTEIN: Perhaps we need to say
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1 something explicit about short-term measures. I
2 think part of the problem here is that there is, if
3 you will, a reasonable reluctance not to make the
4 system worse in the long haul by doing something in
5 the short haul.
6 But I think that part of the issue of
7 urgency is that one must do something in the short
8 run, and I think that that is perhaps yet another
9 message that needs to get communicated. We were
10 saying that, in essence, by calling for a
11 declaration of emergency, but we were doing it
12 because we thought that the legal framework didn't
13 allow for another remedy.
14 I think what the pushback is, which we
15 have heard from Jerry, is that there is a
16 reluctance for the Department to do that, because
17 it might tamper with the system in a way that is
18 adverse for the future.
19 But I think that the way around that is to
20 call attention to the need for short-term actions
21 independent of long-term solutions. I am just
22 concerned that if we call again for, you know, a
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1 declaration of emergency, it already fell on deaf
2 ears once, what exactly are we going to accomplish.
3 DR. BRECHER: Julie.
4 MS. BIRKHOFER: Thank you, Dr. Brecher, if
5 I could just comment. The public health emergency
6 that is language in the MMA, that could be used to
7 address the payment for the drug, and I certainly
8 am not disputing that at all, but another mechanism
9 that is available to CMS in the short term, as
10 well, would be the classification of biologic
11 response modifier, and that is a payment on the
12 physician administration side.
13 So, you have the payment for the drug,
14 which is the ASP, and then you have the cost of
15 services to physicians, so just respectfully, we
16 would also ask CMS--and I know they have had
17 meetings with IDF and Quad AI, and I believe the
18 AMA, or Quad AI and IDF--went in with a lot of
19 scientific and clinical information of why IVIG is
20 a biologic response modifier, and those of you
21 around the table that are physicians probably know
22 why it is, but that would be short term, as well.
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1 MS. VOGEL: I could further explain that
2 since I was in the meeting with CMS. Basically,
3 you have two different mechanisms. You have got to
4 increase the reimbursement for the drug, and I
5 think you can pretty much say that most providers,
6 physicians, or whoever it is, they are not going to
7 be buying product at a loss especially at the
8 number of grams you are talking about, so you have
9 got to get the reimbursement up to at least the
10 cost. I mean and that is where you are at.
11 Doctors are like if I could at least break
12 even, I would be taking these patients. Now, on
13 the administration side, they got hit both ways.
14 They have got hit on the drug side, they got hit on
15 the administration side.
16 The administration side applies to both
17 the physician's office, and is going to apply to
18 the hospitals, and so the highest classification
19 for IVIG is a biologic response modifier. It meets
20 the definition. It's a high complexity
21 administration product. CMS just needs to
22 recognize it as such.
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1 The meeting went well, and I think they
2 are open to it. They can accept it immediately.
3 They could put a transmittal out, and then we could
4 be reimbursed at a higher percentage, but I have to
5 still say with that, if you don't get the drug
6 price up, you are not going to succeed, and with
7 your language on increasing reimbursement in the
8 non-hospital setting, I think it is very important
9 to say that, but the only mechanism that CMS does
10 have currently, on a short-term basis, is through,
11 unfortunately, the language of a public health
13 Other than that, it is going to take an
14 act of Congress to change this.
15 MS. BOYLE: I would just like to reiterate
16 what Michelle has said, but as far as the public
17 health emergency, whether it is actually declared
18 or not, that has really raised awareness. You
19 know, members of Congress are signing on. If you
20 continue with that recommendation, it's putting the
21 emphasis on how important this is.
22 The biological response modifier, I think
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1 is something you could do right now. I think it
2 makes a lot of sense to put your wording in there,
3 but I still question why not recommending the
4 public health emergency.
5 DR. BRECHER: Jay.
6 DR. EPSTEIN: Mark, I would suggest that
7 you make the two points of reclassifying IGIV as a
8 biological response modifier, and exercising the
9 authority to declare a public health emergency to
10 provide CMS with alternative reimbursement
11 scheduling, as subpoints under No. 1, because they
12 are simply specific suggestions under No. 1.
13 Again, I am not close enough to the
14 subject to know whether those are the only
15 available tools, but there is no reason that those
16 can't be mentioned.
17 DR. HOLMBERG: I would like to ask a
18 question of our economist here. The way No. 1 is
19 worded, to a level consistent with the local market
20 or current market pricing, when you have a
21 distributor in the place there, in the middle, and
22 you have the pricing being determined by the
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1 manufacturer, how do you guarantee that?
2 I mean the formulas that are available do
3 not reflect the distributor.
4 DR. HAAS: Well, the guarantee is an
5 interesting word. As soon as you put it out in the
6 marketplace, the concept of guarantee disappears.
7 You have guarantee only if they are fixed prices,
8 and that, I don't think any of us would want to
9 look very seriously at unless it were--well, I will
10 just stop there. I don't think we want to look
11 very seriously in trying to fix prices.
12 You know, this is unresolvable problem in
13 the sense that we don't have a situation where the
14 seller and the end user are directly connected to
15 one another.
16 There are these intervening markets which
17 are not under any type of control, so I think we
18 have got to make the statement in such a way that
19 the doctor that prescribes the IVIG is paid enough
20 to cover the cost of his or her services, and I
21 don't know the right wording there. I am not close
22 enough either to give an answer to that.
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1 Jerry, it's the other thing. I want to
2 continue to reemphasize something Jay said earlier.
3 When we get through with this, I think it ought to
4 be set up in such a way there are short-term points
5 and some long-term points. I think they need to be
6 separated and clear.
7 DR. BRECHER: Art.
8 DR. BRACEY: One of the things I guess
9 that I am concerned about is that I would think
10 that on the other side, the decisionmakers perhaps
11 are not as sensitive to the quality issues
12 associated with the shift.
13 I mean they see it as a neutral. It would
14 be too detailed to go through the entirety of it in
15 this document, but I would wonder, is there a
16 chance for an interface to explain, you know, what
17 the quality issues related to the shift would be.
18 I mean is that something that can be done?
19 DR. BRECHER: We have done that before
20 where resolutions have gone forward to the
21 Assistant Secretary and felt that additional
22 explanation was needed, and we have had am a
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1 meeting with the Assistant Secretary with a
2 subgroup of the committee and other interested
3 parties. So, that is a possibility.
5 DR. LINDEN: This isn't really directly
6 related to that, but it is sort of related to who
7 understands what in terms of our position, but I
8 was looking at this web site printout that says the
9 Advisory Committee on Blood Safety and
10 Availability, but the text has a lot of things that
11 were in Dr. Beato's letter that I don't think we
12 really decided or necessarily agree with.
13 So, I am wondering if that's misleading to
14 people in how we make our thoughts known, if that's
15 not what is in the record on the web site.
16 DR. BRECHER: Specifically, are you
17 referring to the recommendation about off-label
19 DR. LINDEN: Yes, in terms of the supply
20 being sufficient, not having concerns in that
21 regard, and recommending that physicians would
22 better serve their patients by communicating their
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1 needs directly and focusing on approved label use,
2 not off-label uses.
3 DR. BRECHER: I think we have had concerns
4 about that, but I think there are bigger fish to
5 fry right now, which is the reimbursement. If we
6 could fix the reimbursement, I think that it would
7 all fall into place.
9 DR. SANDLER: I apologize I wasn't here
10 this morning, I wasn't able to be for the
11 presentation. Am I correct that the
12 representatives of the patients have not had a
13 direct audience with the Assistant Secretary of
15 The purpose of my asking that question is
16 that my advocacy for these patients is driven a lot
17 by the testimony as it is given very effectively by
18 the representatives, and I am hoping that this
19 committee isn't serving as a filter, preventing the
20 Assistant Secretary from hearing the heart-moving
21 stories of these people.
22 MS. VOGEL: We have not met with the
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1 Assistant Secretary. We have requested a meeting
2 with Secretary Leavitt, and we are supposed to be
3 part of a meeting with him or his chief of staff on
5 We have met with Herb Kuhn on many
6 occasions, and we have also put in a request to
7 meet with Administrator McClellan, but, no, we have
8 not met with the Assistant Secretary.
9 DR. BRECHER: But you do have a meeting on
10 Friday with a high-level official?
11 MS. VOGEL: Yes, we are part of a group
12 meeting with Secretary Leavitt or his chief of
14 DR. BRECHER: That should help drive home
15 the message.
17 DR. EPSTEIN: I think we should come back
18 to Art's point about the added negative effects of
19 in-hospital therapy, and at least flag the issue in
20 the first paragraph.
21 I am not exactly sure what specifically we
22 want to say, but let's see. Perhaps instead of
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1 saying "including a shift to hospital-based
2 therapy," we could say, "which are aggravated by
3 the shift."
4 DR. BRECHER: Is it that the disruptions
5 are aggravated, or is it that the risk to the
6 patient is increased by putting them in a hospital
7 setting as opposed to a doctor's office?
8 DR. EPSTEIN: We could add a second
9 sentence saying something along the lines that in
10 particular, we believe that hospital-based therapy
11 adds increased risks and costs to patient care,
12 something along those lines.
13 DR. BRECHER: Jerry.
14 DR. SANDLER: I think the words
15 "hospital-based therapy" may cloud a little bit of
16 the issue. Speaking as someone who covers one of
17 the infusion services here in town, I, of course,
18 wouldn't act as the technologist for the patient's
19 doctor and just give the infusions for a person who
20 is so precarious. We would require that such a
21 person transfer care to be using the infusion
22 services of the hospital.
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1 So, it is not just someone out there who
2 has taken care of a patient for the last 15 years
3 will write a prescription and have the person come
4 to the hospital and pay a little bit more and be
6 We wouldn't simply infuse. We would
7 expect the person who is being treated is our
8 patient, so it is really going to be the scenario
9 is that people will have to be transferred to
10 persons who will be on site to care for such
11 patients as they have been on site in the doctor's
13 I think we want to make it clear.
14 DR. BRECHER: Well, which may not be in
15 the best interests of the patient if they have to
16 travel a great distance to get to the hospital.
17 DR. SANDLER: Of course. I mean, of
18 course, it is not in the best interests of the
19 patient. These people have been cared for, they
20 have been cared for well. They belong in their
21 doctor's office where, in the long run, the United
22 States Government will pay less for their care, and
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1 the patients, as Dr. Bracey points out, are going
2 to get a higher quality of care in a doctor's
4 Hospitals aren't a place for routine
5 maintenance therapy.
6 DR. BRECHER: So, can we say, in
7 particular, we believe hospital care may not be in
8 the best interests of these patients?
9 DR. SANDLER: It is a little, it is
10 something my check payers wouldn't like me to
12 DR. BRECHER: Well, it may not always be
13 in the best interest, how is that, does that soften
14 it enough?
15 DR. SANDLER: Well, my point is to make it
16 clear that hospitals shouldn't be expected to
17 simply infuse, that if hospitals are going to be
18 the place where people are going to be treated,
19 hospitals are going to expect that the care of the
20 patient will be taken away from the person who has
21 cared for them up to this point, and delivered to
22 an on-site physician who will be there to take care
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1 of a person getting infused.
2 DR. BRECHER: I am more worried about the
3 immuno-deficient patients going to a hospital
4 setting where they may be--
5 DR. SANDLER: Oh, I get you, yes, and that
6 is an additional concern.
7 MR. SKINNER: There is two issues. It's
8 the transfer of the patient, and it's the setting
9 of care, and you only have got the setting of care.
10 I think you could fix it and cover both if you
11 would say in particular, we believe the transfer of
12 patients to a hospital-based care setting may not
13 be in the best interests, so you pick up the notion
14 of transferring the patients from their traditional
15 physician, as well as putting them in a hospital
17 DR. BRACEY: One of the things that I
18 thought that perhaps we could say is that it
19 disrupts the continuity of care, I think people buy
20 into the continuity of care, and exposes the
21 patients to new risks, you know, the hazards of the
22 hospital environment.
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1 DR. BRECHER: Increased risk of what, Art?
2 DR. BRACEY: Just say "increased risk."
3 DR. BRECHER: Jeanne.
4 DR. LINDEN: Instead of saying that the
5 hospital-based care is bad, can we say that the
6 loss of the continuity and benefits of the
7 community-based care could be lost, transferred to
8 less optimal care with increased risks, or
9 something like that?
10 DR. BRECHER: I don't think people are
11 going to say less optimal care. I don't think
12 Jerry would like to hear that.
14 DR. EPSTEIN: Just some suggested wording,
16 In particular, we believe that transfer of
17 care to a hospital or hospital setting may
18 interrupt continuity of routine care and may add
19 otherwise unnecessary costs, logistical complexity,
20 and risk.
21 If that sounds right, I will read it again
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1 DR. BRECHER: May interrupt continuity of
2 care and--
3 DR. EPSTEIN: May interrupt continuity of
4 routine care and may add otherwise unnecessary
5 costs, logistical complexity, and risk.
6 DR. BRECHER: Logistical?
7 DR. EPSTEIN: Complexity.
8 DR. BRECHER: And risk?
9 DR. EPSTEIN: And risk.
10 If we want to say infectious risk, that is
11 the main one are worried about.
12 DR. BRECHER: Does that get the sentiment?
14 DR. SANDLER: Maybe say care by their
15 primary physician.
16 DR. BRECHER: Transfer of care to a
18 DR. SANDLER: --may interrupt continuity
19 of routine care by their primary physician.
20 MS. BOYLE: It's not necessarily a primary
21 care physician. Sometimes it's a specialist in the
22 outpatient setting.
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1 DR. SANDLER: By their usual care
3 MS. BOYLE: Yes.
4 DR. BRECHER: Okay, we can do that.
5 Whoever succeeds me as chair of this
6 committee, typing is a prerequisite.
7 DR. SANDLER: Where it says "infectious
8 risk," do we want to say something like risk of
9 hospital-based infections, or nosocomial
11 DR. BRECHER: Yes, I think nosocomial.
12 DR. SANDLER: We are not talking about
13 common colds.
14 DR. BRECHER: What a surprise, Microsoft
15 Word doesn't recognize nosocomial.
17 DR. LINDEN: I am not sure that "routine
18 care" gets across that we are talking about care
19 actually being transferred, because to me,
20 "interrupted" may mean, well, they are getting part
21 of it now at the hospital and part of at the
22 doctor's office, and I wonder if we are really
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1 talking about loss of the benefits of the
2 continuity of routine care.
3 DR. BRECHER: I think when we put "usual
4 care provider," I think probably the need for the
5 word "routine" has disappeared. I think we can
6 probably get "routine" out of there. Does that
7 make it better?
8 DR. LINDEN: I guess "interrupt" is what I
9 have the most trouble with, if we are talking about
10 actually discontinuing it.
11 DR. BRECHER: Well, interrupt or disrupt.
12 Would that be a better fit, say "disrupt" instead
13 of interrupt"?
14 DR. BIANCO: "Disrupt" is in the previous
15 sentence. "Interfere with."
16 DR. BRECHER: So, would you prefer
18 DR. SANDLER: How about "impair"?
19 DR. BRECHER: I am sorry, "impair"?
20 Let me just read the paragraph, so
21 everyone hears it again.
22 "The committee remains highly concerned
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1 that disruptions to access of IGIV, including a
2 shift to hospital-based therapy, continue to
3 compromise quality of care for many patients. In
4 particular, we believe that transfer of care to a
5 hospital or hospital setting may impair continuity
6 of care by their usual care provider"--we certainly
7 say care a lot, don't we--"and may add otherwise
8 unnecessary cost, logistical complexity, and
9 nosocomial infectious risk. We further are
10 concerned that a change to hospital outpatient
11 reimbursement to ASP plus 8 percent effective
12 January 2006 will further aggravate an already
13 difficult situation and that this shift will not be
16 DR. SAYERS: Any interest in having a
17 preface which says something along the lines of,
18 "After new input from providers, manufacturers,
19 patients, and distributors, the committee remains
20 highly concerned"?
21 DR. BRECHER: After hearing input?
22 DR. SAYERS: New input, after new input.
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1 DR. BRECHER: Users or consumers?
2 DR. SAYERS: Users, patients, consumers.
3 DR. EPSTEIN: Can we put patients first?
4 DR. SAYERS: Yes.
5 DR. BRECHER: Always, patients always come
6 first. Patients, medical professionals, and
8 DR. SAYERS: Sure.
9 DR. BRECHER: Manufacturers always come
11 DR. SAYERS: And then remains highly
12 concerned about accelerating disruptions.
13 DR. BRECHER: Concerned regarding
15 DR. SAYERS: Accelerating.
16 DR. BRECHER: Oh, accelerating.
17 DR. BRECHER: Paul.
18 DR. HAAS: We also heard from distributors
19 this morning, too.
20 DR. BRECHER: Do they come before or after
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1 DR. BRECHER: Jerry.
2 DR. HOLMBERG: To drop one of the--oh, I
3 am sorry.
4 DR. BRECHER: Either Jerry.
5 DR. SANDLER: I want to pick up on Dr.
6 Linden's point about being a little more explicit,
7 and in particular, we believe that the transfer to
8 hospitals for IV infusions may require transfer of
9 these patients' care from their current providers
10 to new hospital physicians or to hospital
12 We haven't made it clear that just going
13 to the hospital for an infusion means we are going
14 to take them away from their doctor. I think we
15 should get that in.
16 DR. BRECHER: I don't know, I think that
17 is a little implicit, Jerry, when we say that
18 impair continuity of care, I don't know that adding
19 those additional words is really going to add that
20 much. I like keeping it simple.
21 MS. LIPTON: I am having trouble reading
22 that, but I think, should "about" really be a
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1 "that" instead of "about" in the third line?
2 DR. BRECHER: Wait a minute.
3 MS. LIPTON: We are concerned that
4 accelerating disruptions, including a shift to
5 hospital-based therapy continue to--
6 DR. BRECHER: So, where do you want me to
8 MS. LIPTON: The third line down. The
9 word "about," you should replace that with "that."
10 DR. HOLMBERG: After "concerned."
11 DR. LINDEN: And you should probably have
12 a couple of comments before and after the including
13 phrase, just to clarify it.
14 DR. BRECHER: "The committee remains
15 highly concerned that"--
16 MS. LIPTON: "That."
17 DR. BRECHER: Okay.
19 DR. HOLMBERG: I would recommend that you
20 drop some of the "cares" and transfer of patients
21 to a hospital or hospital setting may impair
22 continuity of care by their usual provider or
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1 medical provider, and get rid of some of the
3 DR. BRECHER: Why don't we say--to a
4 hospital or hospital setting, I think "to a
5 hospital setting" is sufficient. I don't think we
6 have to say "to a hospital."
7 MR. SKINNER: I was going to make a
8 comment there. Instead of saying, after the first
9 hospital, insert the word "physician." To say to a
10 hospital physician or hospital setting, that way it
11 picks up Dr. Sandler's comment.
12 DR. BRECHER: Okay. Now, we have to get
13 rid of some of these "cares," because we care too
14 much. Oh, we transfer to a hospital, that gets rid
15 of one. Thank you. I heard that .
16 Do we really need "usual care provider,"
17 or can we just say "usual provider"? Medical
18 provider or just provider? Okay.
19 MR. SKINNER: Provider in that context
20 could mean distributor.
21 DR. BRECHER: Medical provider?
22 MR. SKINNER: I think that's better.
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1 MS. VOGEL: I have a recommendation. What
2 we are hearing is that they are not seeing a
3 physician in the hospital, they are just being
4 infused with the product from a nurse. So, where
5 you have, "In particular, we believe the transfer
6 to a hospital physician," it really should just be
7 a hospital setting.
8 DR. BRECHER: I think in Dr. Sandler's
9 case, it would be transferred to a hospital
10 physician, but in other hospitals, it may not be.
11 MS. VOGEL: Okay.
12 DR. BRECHER: So, the question is which is
13 the best way to leave it.
14 DR. EPSTEIN: I think hospital setting,
15 because the reimbursement is geared to the setting.
16 DR. BRECHER: That's true.
17 DR. EPSTEIN: It is true that a lot of
18 things go along with the setting, but I think in
19 that sentence, it is the setting.
20 DR. BRECHER: Okay. The less words, the
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1 DR. SAYERS: I am getting down to the
2 picking of the nits now. You have got, "The
3 committee remains"--this is now the second
4 line--"highly concerned that accelerating
5 disruptions to access of IGIV, which include a
6 shift to treatment in hospital."
7 DR. BRECHER: Which include a shift to
9 DR. SAYERS: In hospital.
10 DR. BRECHER: That doesn't work.
11 DR. SAYERS: Why?
12 DR. BRECHER: Including a shift to
13 treatment in a hospital-based therapy?
14 DR. SAYERS: Oh, no, you would delete the
15 based therapy.
16 DR. BRECHER: Okay.
17 DR. SAYERS: Which include a shift to
18 treatment in hospital. I mean hospital-based
19 therapy sounds like--I mean it could be confused
20 with somebody going to the formulary and deciding--
21 DR. LINDEN: Then, you need the other
22 comma after hospital or a hospital, the hospital.
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1 Is it access of IGIV or to, and is it IGIV or IVIG?
2 DR. BRECHER: Treatment in a hospital
4 DR. BRACEY: I would say if we keep it
5 generic and say, "in a hospital setting," because
6 in truth, the way hospitals are organized these
7 days, they have outpatient activities that are away
8 from the inpatient, and, you know, you need to
9 leave it I think a little more general.
10 DR. BRECHER: Hospital setting, you
11 prefer? Okay.
13 DR. EPSTEIN: Just picking up on someone
14 else's earlier comment. "Accelerating disruptions
15 in access to IGIV," I think is a little bit better
17 DR. BRECHER: Accelerating disruptions--I
18 am sorry?
19 DR. EPSTEIN: --in access to IGIV.
20 DR. SAYERS: And it should be, "which
21 include" instead of "including."
22 DR. BRECHER: Which includes.
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1 DR. SAYERS: Yes.
2 DR. LINDEN: But it said "accelerating
3 disruptions," I mean that is the subject there.
4 DR. SAYERS: No, it's "which includes
5 shifts to treatment in a hospital setting."
6 DR. BRECHER: Let's read it from the
8 MR. SKINNER: I think you need an "s" on
9 continues now, too.
10 DR. BRECHER: Continues. Wait a minute.
11 DR. SAYERS: The other "s" is silent.
12 DR. BRECHER: Let's try that sentence from
13 the top. "After new input from patients, medical
14 professionals, distributors, and manufacturers, the
15 committee remains highly concerned"--or do you want
16 to say gravely concerned--"highly concerned that
17 accelerating disruptions in access to IGIV which
18 includes a shift to treatment in a hospital setting
19 continues to compromise quality of care for many
21 MS. LIPTON: It's "continue." It's
22 disruptions continue.
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1 DR. BRECHER: Continue to compromise
2 quality of care.
4 DR. EPSTEIN: Well, I have a substantive
5 question for the committee. Do we think that the
6 disruptions are accelerating, or just persisting?
7 I am not sure that I heard anything today that was
8 worse than what we heard.
9 DR. SAYERS: I would go for persisting.
10 MR. SKINNER: I think what is accelerating
11 is the transfer to the hospital-based setting, not
12 the disruption, so when we reworked the sentence,
13 the word "accelerating" is in the wrong place.
14 DR. BRECHER: I don't know that we know
15 that it is accelerating. It's continuing.
16 MR. SKINNER: Well, the percentages have
18 DR. BRECHER: It has continued to shift to
19 the hospital. I don't know that it's in an
20 accelerating rate, though.
22 DR. EPSTEIN: We could say, "which
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1 includes a progressive shift."
2 DR. BRECHER: Yes, we could say that.
3 DR. EPSTEIN: No, no, "persisting
4 disruptions which includes a progressive shift to
5 treatment in a hospital setting.
6 DR. BRECHER: That includes a progressive
7 shift in access--no, that's not right.
8 DR. EPSTEIN: It's the progressive shift
9 to treatment in a hospital. The word "progressive"
10 is part of the shift to treatment in a hospital.
11 DR. BRECHER: So, progressive shift--where
12 do you want me to move the progressive shift to?
13 DR. EPSTEIN: The next line, where the
14 word "shift" occurs, just put the word
15 "progressive" in front of it, and now we have to
16 fix "persistent disruptions." Take the article "a"
17 out of that. It says, "a persistent disruption."
18 Persistent disruptions, and again it was comma
19 which include--I am sorry--"disruptions in access."
20 The "that includes" comes out.
21 DR. LINDEN: Then, the next "includes"
22 needs to be just "include."
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1 DR. BRECHER: Right. Got it.
2 DR. EPSTEIN: I think it's time to go down
3 to the recommendations again.
4 DR. SANDLER: Hospital setting is in there
6 DR. LINDEN: The second time, you could
7 just say "such transfer," and don't have a comma
8 after it.
9 DR. BRECHER: I guess we don't really need
10 the word "setting." It doesn't add that much.
11 DR. LINDEN: You need to get rid of the
12 comma after "hospital." We are still saying
13 "transfer to a hospital" twice.
14 DR. BRECHER: We are. Well, we are saying
15 "shift to treatment in a hospital," and then we are
16 saying "transfer to a hospital."
17 DR. EPSTEIN: I think it's okay to repeat
19 DR. LINDEN: Yes, but you have to get rid
20 of the comma.
21 DR. BRECHER: I am sorry?
22 MS. LIPTON: Between "hospital" and "may,"
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1 you need to delete the comma, next line down.
2 DR. LINDEN: Yes, it's just the transfer
3 may impair continuity of care.
4 DR. BRECHER: Okay. Ready to go down?
5 Ready or not, here we are.
6 DR. EPSTEIN: Capitalize Secretary.
7 DR. BRECHER: Absolutely. Is there a
8 hyphen in short term? Yes. That was it for No. 1.
9 Let's go to No. 2.
10 DR. EPSTEIN: In No. 2, the word "change"
11 needs to be added. "We consider the current
12 program to change."
13 DR. HOLMBERG: It's unclear what your
14 recommendation is there.
15 DR. EPSTEIN: Oh, it's to withdraw the
16 regulation. I mean right now you have a regulation
17 in place that will cause outpatient reimbursement
18 to go from I guess AWP to ASP plus 8 percent. So,
19 reconsider. I mean we could be more directive and
20 say withdraw.
21 DR. BRECHER: Well, it's the current plan.
22 DR. HAAS: Would re-examine be a better
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1 term there than reconsider?
2 DR. BRECHER: I am sorry? What is the
3 word you want instead of reconsider? Re-examine?
4 DR. EPSTEIN: Well, again, we could say
5 delay or withdraw.
6 DR. BRECHER: Withdraw.
7 DR. EPSTEIN: I mean that's the strongest
8 thing, is just withdraw it. Again, it's a
9 regulation, if I am not mistaken.
10 DR. BRECHER: Withdrawing the current
11 plan. Does that get the sentiment across?
12 DR. HOLMBERG: Dr. Brecher, you have a
13 comment from the floor.
14 DR. BRECHER: Sorry. Yes, Julie.
15 MS. BIRKHOFER: Thank you, sir. On No. 2,
16 and I am just trying to serve as a resource here,
17 basically, the ASP plus 8 percent is in statute,
18 right? That's in the MMA. So, CMS--no?
19 MS. WEINSTEIN: Hospital outpatient.
20 MS. BIRKHOFER: Step up here, please.
21 MS. WEINSTEIN: Hospital outpatient
22 reimbursement in '06 has to be based on hospital
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1 acquisition cost, but one suggestion might be--I
2 mean a couple of the ideas, PPTA, excuse me, the
3 group together decided on was the dampening effect.
4 There is a precedent for that, but basically, it
5 would prevent the rate from dropping by more than
6 15 percent from the 2005. The current rate now
7 couldn't be reduced by more than 15 percent for
8 '06, and that would hopefully mitigate some of the
9 turmoil there would be, you know, if you reduce a
10 rate by more than that, that might create.
11 MS. BIRKHOFER: So, the MMA put in place a
12 provision that the hospital outpatient
13 reimbursement had to be based on hospital
14 acquisition costs, and that was to be done by the
15 General Accounting Office, the GAO.
16 The GAO transmitted their report in April,
17 and it was up to CMS to look at the GAO's
18 methodology to see if they wanted to use it or not,
19 they had flexibility. They chose not to use it,
20 which was a very good thing for access, because the
21 rates were abysmal, because of the trouble that the
22 GAO had was survey data.
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1 So, then, CMS put in ASP plus 6 percent
2 plus 2 percent, which comes to a total of ASP plus
3 8, so what Anna Weinstein, my colleague, just
4 explained, is that PPTA and this group of IVIG
5 community came up with these alternatives because
6 the framework of ASP is here to stay, and it's
7 accepted, and it's a shift away from AWP.
8 So, this group, along with PPTA, that's
9 where we put forward the concept of a dampening
10 provision, which Anna just explained. You could
11 freeze current rates until further knowledge was
12 gathered, data.
13 PPTA is working to collect data. The
14 biological response modifier, separating the J
15 codes, those are the types of things we discussed.
16 So, I just wanted to offer that.
17 DR. HOLMBERG: Mark, can I ask a question,
19 DR. BRECHER: Sure.
20 DR. HOLMBERG: Again, a question for our
21 economist. If you have one setting being given
22 this price, doesn't it have to be consistent in all
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1 of the settings, or else you are going to continue
2 to have the problem?
3 DR. HAAS: I would tend to think so, yes.
4 DR. HOLMBERG: I mean this is where we
5 have gotten the problem or CMS has gotten
6 themselves into a problem, is that it has shifted
7 and instead of the MMA making all the changes at
8 once, there has been a gradual shift, and so
9 therefore, the market is not going to--if I would
10 understand the economics correctly--the market is
11 not going to stabilize until all of the places that
12 it is being used is stabilized, are stabilized.
13 DR. BRECHER: Well, it is going to
14 stabilize to the point of least resistance to those
15 people who are willing to pick up the loss at the
16 current rate.
17 DR. HOLMBERG: The thing is that with what
18 is being recommended on the dampening, it is still
19 not going to correct the inpatient or the office
20 infusion sites.
21 DR. EPSTEIN: I agree with what you said,
22 Jerry, but I think again it's a short-term problem
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1 we are trying to, in this case, prevent, which is
2 that a sudden and precipitous drop in reimbursement
3 in the hospital outpatient setting can only make
4 the current situation worse.
5 Now, that in itself is not going to create
6 parity for the non-hospital setting, let alone how
7 it might reconcile the inpatient care, but the
8 short-term issue is not to have the precipitous
10 MS. LIPTON: And then we have to address
11 the long-term issue, which probably encompasses
12 what Jerry, that you said is it needs to be
13 stabilized at a reasonable reimbursement in all
15 DR. EPSTEIN: Right. That may be yet
16 another point, but I think we ought to modify
17 Recommendation 2 to say, "Modify the current plan."
18 DR. BRECHER: Consider modifying the
19 current plan?
20 DR. EPSTEIN: Yes, or consider modifying
21 or modify. Consider modifying the current plan,
22 and I would for the moment remove the
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1 parenthetical, and then to change, singular, to
2 change hospital outpatient--take out the word
3 "to"--to change hospital outpatient reimbursements
4 to ASP plus 8 percent in January in such a way as
5 to prevent any sudden and large decrease in
7 DR. BRECHER: Jerry.
8 DR. SANDLER: Mr. Chairman, about three
9 occasions now, people have suggested that you take
10 the word "consider" out. On three occasion, you
11 very politely have kept it in.
12 DR. BRECHER: Not intentionally.
13 DR. SANDLER: I am here representing the
14 American Hospital Association, and I can tell you
15 that their response to this is hell, no, and if
16 they were here, administrators in hospitals would
17 tell you, you don't want to be polite about this.
18 DR. BRECHER: Modify it.
19 DR. SANDLER: Yeah.
20 DR. EPSTEIN: You want to go back up and
21 do the same?
22 DR. BRECHER: Just because we are such
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1 caring people, we like to be polite.
2 MS. LIPTON: I thought this was different
3 because we weren't sure of what the right thing to
4 do, there are a number of options.
5 DR. BRECHER: That's right, we are not
7 MS. LIPTON: I think that No. 1 itself may
8 be--I will have to read it--well, we did say it, we
9 said flat out, increase reimbursement. I think
10 that's what we want to say.
11 DR. BRECHER: I think No. 1 can stay as
12 consider, but No. 2 is stronger as modified. We
13 are just giving them some options.
14 Okay. No. 3. Do we have a No. 4?
15 DR. SAYERS: This is about No. 3. Can we
16 just way "whether" instead of "the extent to
18 DR. BRECHER: I am sorry, modify it?
19 DR. SAYERS: I was going to say,
20 "re-examine whether" instead of "the extent to
22 DR. BRECHER: Oh, I see. Okay.
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1 DR. LINDEN: Is this something we want to
2 do just once, or do we want to say continue to
3 examine like on an ongoing basis versus a one-time
5 DR. BRECHER: Do we have to say are or are
6 not, or just say "are meeting demand"?
7 DR. LINDEN: You don't need are not.
8 DR. HAAS: Mark, I think there is further
9 questions as to what we mean by demand. There is
10 the demand for the label use, and the demand for
11 the off-label use, and since that came up in the
12 Secretary's letter, it would seem to me we should
13 be a little more clear.
14 DR. SAYERS: And say what?
15 DR. HAAS: Don't ask me.
16 DR. HOLMBERG: Mr. Chair, may I?
17 DR. BRECHER: Yes.
18 DR. HOLMBERG: The concern here, label and
19 off-label use, is the off-label use, are there
20 studies, clinical studies, to support the use of
22 DR. BRECHER: Some, some better than
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2 DR. HOLMBERG: Exactly, and the concern
3 here is where is the evidence-based medicine to
4 support the use of the off-label?
5 DR. BRECHER: What I hear you saying,
6 Jerry, is don't open this can or worms to say use,
7 demand, to meet demand?
8 DR. HOLMBERG: I would say demand, just
9 leave it as demand.
10 MR. SKINNER: I have two comments about
11 this. I am wondering if, instead of saying
12 "demand," the polite way to say it would be to say
13 talk about meeting prescribed treatment. That way,
14 you are saying the physicians should be in control
15 of the medicine perhaps.
16 The other thing that bothers me about this
17 is because the Secretary has already said we will
18 continue to monitor the situation, so basically,
19 what we are saying is do what you said you were
20 going to do.
21 So, I thought what we were doing here is
22 saying we are skeptical that there isn't a supply
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1 problem and that we think that there might be an
2 underlying supply problem that you haven't
3 detected, so go back and look again, not just to
4 continue to monitor it until you find one shows up.
5 So, I am not sure this says anything
6 different than what the Secretary responded in the
7 letter that they were already going to do.
8 MS. LIPTON: But, Mark, I think if you
9 look at the beginning where we said there is new
10 information, and then that, in combination with the
11 word "re-examine," it isn't just continue to
12 monitor, it's re-examine, and I think that that's--
13 MR. SKINNER: Okay.
14 DR. BRECHER: That is what I like to hear,
15 one lawyer talking to another lawyer.
17 DR. HAAS: The way we started to write
18 label and off-label, I was uncomfortable with that,
19 too, but I guess I am still a little concerned that
20 the Secretary's letter and what we heard from PPTA
21 is if there is a four-week supply, that that seems
22 to suggest that this statement doesn't say a whole
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2 DR. BRECHER: But it's a four-week supply
3 based on use five or six years ago.
4 DR. HAAS: I guess my thought would be
5 that they are looking for us to give directions, we
6 maybe want to be a little more specific in our
7 statement, because leaving it alone, then, I would
8 come back to you and say, well, what I am getting
9 from the manufacturer is that there is plenty of
10 supply out there.
11 DR. BRECHER: Why don't we move the word
12 "current," whether IGIV supplies are meeting
13 current demand," not demand five or six years ago
14 would be the implication.
15 MS. WEINSTEIN: Sorry, could I add one
16 point of clarification? The whole issue of the
17 yellow, red, and green light, it was decided five
18 or six years ago what each of those means, the
19 amount of supply available at each of those
20 different lights, but we are not talking about the
21 same amount, overall amount of supply that there
22 was back then.
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1 DR. BRECHER: The ratio?
2 MS. WEINSTEIN: Yes, it's a ratio of the
3 inventory to the 12-month average distribution.
4 DR. BRECHER: Okay.
5 MS. WEINSTEIN: So, just to clarify for
7 DR. LINDEN: I just have a question,
8 because I noticed in her letter, she talked about
9 being sufficient for availability to patients, and
10 I don't know enough about this, but were there
11 concerns that with decreased reimbursement, that
12 people might not have access, and therefore, it's
13 not available to them even though there is some out
14 there, but at twice the price, they can't afford
16 DR. BRECHER: Yes, that is the concern.
17 DR. LINDEN: I wonder if we want to get
18 that across, and she didn't just say total supply.
19 She said the supplies are actually available to
21 DR. BRECHER: Well, I think that gets to
22 the reimbursement costs with the prices going up,
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1 and you have to somehow match that, and what they
2 are currently paying is not enough, so that people
3 do purchase it.
4 DR. LINDEN: Right, but I am wondering if
5 we want to talk about this in terms of being
6 available to patients as opposed to just the
8 DR. HAAS: May I just add to Jeanne's
9 comment that when Jerry mentioned he would make
10 phone calls and then they would become available,
11 that, to me, is an indication that the patients
12 aren't getting it.
13 DR. BRECHER: It is certainly a red flag.
14 DR. HAAS: Yes. So, I like the idea of
15 getting the patient.
16 DR. BRECHER: Meeting patient demand, is
17 that where we would put it in there? I yield to
18 the economist in the group.
19 DR. HAAS: I can go into the economist
20 jargon just like medical doctors go into your
21 jargon. That word has a very specific meaning in
22 economics, so I think what we are talking about
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1 here is a need, and I would get away from the
2 economic term and just talk about the need.
3 DR. LINDEN: That is what Karen and I were
4 suggesting, maybe patient needs.
5 DR. BRECHER: Art.
6 DR. BRACEY: Back to Paul's point, I think
7 that if we don't hit this piece about off-label
8 use, I mean a big part of their argument is that,
9 well, you know, really, the reason that there is
10 increasing demand is that there are allow these
11 bozos out there using the off-label, the components
12 for off-label use, and perhaps we could add a
13 statement, "Although off-label use is a factor in
14 demand, there are a number of studies to support
15 the use of this agent in selected patients," you
16 know, something to support.
17 It just seems to me that their position is
18 that all off-label use is wrong, and I am not sure
19 that's something that should be left standing.
20 DR. LINDEN: Have we seen those studies
21 presented to this committee, Art?
22 DR. BRACEY: Well, not in the two times I
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1 have been here.
2 DR. BRECHER: No, we haven't specifically
3 looked at that data, but I think a number of us
4 reviewed papers on specific diseases, and it's a
5 mixed--the evidence is mixed. Some is better than
6 others for particular indications.
7 MS. LIPTON: Is it really the issue of
8 off-label use, or is it the issue that we don't
9 think that off-label use totally accounts for all
10 of these disruptions that we are seeing?
11 DR. BRACEY: Yes.
12 MS. LIPTON: I don't want us to get tied
13 up personally in off-label use, but I think that we
14 could send in a message that although we recognize
15 there is off-label use of this product, we don't
16 believe that that is--I don't know what the last
17 words are--but that isn't the sole reason that
18 patients are not getting access to this product.
19 DR. BRECHER: Celso.
20 DR. BIANCO: I would leave it as is.
21 Patient need is not determined by CMS or by some
22 authority. It is determined by the physician that
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1 prescribed, so I think it covers everything.
2 DR. BRECHER: Going once--does we need a
3 fourth point? Jay, you had mentioned a possible
4 fourth point.
5 DR. EPSTEIN: Well, I do think that we
6 need a fourth point, which has something to do with
7 the long term, and not being an economist, I am not
8 sure exactly what is the right thing to say here,
9 but it's along the lines of working together with
10 the Congress to establish a more stable pricing and
11 reimbursement structure for IGIV.
12 Now, again, others may get this a little
13 bit more on target, but I think that is what Point
14 4 is about.
15 MS. LIPTON: Stable and sustainable? I
16 mean we talked about sustainable.
17 DR. EPSTEIN: Yes, sustainable is good,
18 but the problem here is that you have got
19 dislocations of pricing and reimbursement that are
20 resulting in disruptions in the care system
21 including distribution and access, and that it's a
22 reflection of the legal construct presently in
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2 I think we have to recognize that the
3 Department, under the present law, is only capable
4 of the bandaid fixes, and that what is really
5 needed is for Congress to re-examine the system.
6 You know, Congress had a legitimate goal
7 of cost containment, but in this particular area,
8 it has had an unanticipated negative effect, and I
9 think what we are asking for is for the Department
10 to work with the Congress to achieve a more stable
11 and sustainable pricing and reimbursement scheme.
12 DR. BRECHER: I used the word
13 "government," but that would encompass Congress.
14 DR. EPSTEIN: Well, again, we are advisory
15 to the Secretary, and the Secretary has the ability
16 to lobby the Congress and introduce legislative
17 initiatives. The agencies do not, incidentally,
18 CMS cannot do this, but the Secretary can do this.
19 So, I think that is really what we want to
20 ask for.
21 MS. VOGEL: Can I make a suggestion? I
22 mean there is one vehicle that can be used during
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1 the fall, and that is reconciliation where we can
2 make the changes to the reimbursement structure for
3 IVIG, so it can be, you know, that the committee
4 recommends that the Secretary work with Congress
5 during reconciliation to establish a long-term
6 stable and sustainable reimbursement structure for
7 IVIG, something like that, because this is a
8 vehicle that can be done during the fall.
9 DR. BRECHER: So, you are suggesting that
10 HHS should work with Congress?
11 MS. VOGEL: During reconciliation.
12 DR. EPSTEIN: Could I just suggest that
13 where you are saying as a short-term measure
14 because reconciliation is a presently available
15 mechanism, but there are other ways, too. I mean
16 they could just introduce new legislation. I am
17 not sure that we want to narrow it. Maybe that's a
19 MS. VOGEL: Yes, I mean the only thing,
20 just looking at the climate, Congress doesn't want
21 to open up the MMA, so this is a way to be able to
22 make a fix for a specific problem, such as IVIG,
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1 which is a unique problem that is not occurring in
2 all the other products.
3 If we want to even mention the uniqueness,
4 and I know with Amy Pisano, when she testified in
5 May, she said, you know, of all the 450 products
6 out there, IVIG is the one product that they are
7 seeing problems with.
8 But, on the other hand, they don't want to
9 pass a technical bill, which is typically where you
10 would see a change in the Medicare, but they are
11 going to pass a reconciliation act, and that is the
12 vehicle where this can occur, if you want to get
13 that specific.
14 MS. LIPTON: But it still falls under work
15 with Congress, doesn't it?
16 MS. VOGEL: It does, it does fall under
17 the work of Congress.
18 DR. BRECHER: I think we don't have to be
19 that specific. We just tell them to do it, fix it
22 DR. HAAS: Mark, I think we do want that
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1 fourth one to read that the Secretary should work
2 with Congress, so it's direct.
3 DR. BRECHER: Okay.
4 DR. LINDEN: Mark, that is not parallel
5 construction, though. The other things are just
6 second person, you need to do this, so it really
7 should be work with Congress.
8 DR. BRECHER: Work with Congress, okay.
9 MS. LIPTON: We already instructed the
10 Secretary to take the following steps, the last of
11 which is work with Congress.
12 DR. LINDEN: Really, I mean it's not just
13 strive to. I mean it is to establish.
14 DR. BRECHER: Right.
15 Is everybody happy? Jerry is not happy.
16 DR. HOLMBERG: Should it be needs, plural,
17 or should it be patients' need? Needs probably.
18 DR. BRECHER: Okay.
19 All in favor of happiness, voting members,
20 raise their hand.
21 [Show of hands.]
22 DR. BRECHER: All opposed?
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1 It's unanimous.
2 It's 5:30, it's time to adjourn.
3 Tomorrow, we take on the hard part. I believe it's
4 9 o'clock. Yes, 9 o'clock.
5 [Whereupon, the proceedings were adjourned
6 at 5:32 p.m., to reconvene on Tuesday, September
7 20, 2005, at 9:00 a.m.]
8 - - -