Blood Safety Transcripts

DEPARTMENT OF HEALTH AND HUMAN SERVICES
ADVISORY COMMITTEE ON BLOOD SAFETY AND AVAILABILITY

Twenty-Seventh Meeting
Volume I
Monday, September 19, 2005
9:00 a.m.
Bethesda North Marriott Hotel
and Conference Center
5701 Marinelli Road
North Bethesda, Maryland 208852

PARTICIPANTS

Mark Brecher, M.D., Chair
MEMBERS:
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
Settings
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
2 affiliation.
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
13 immunodeficiency.
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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1 companies.
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
9 CDC.
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
19 IND.
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
13 sterility.
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
21 reconstitution.
22 DR. BRECHER: Is there any way to extend
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1 the outdate? Is it stored liquid or is it frozen
2 normally?
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
8 process.
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
8 me.
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
19 access.
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
12 therapy.
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
11 discretion.
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
21 survey.
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
16 time.
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
9 modifier.
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
4 product?
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
14 capacity.
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
13 relationship.
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
18 more?
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
9 tablets.
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
21 products?
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
14 price?
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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1 IVIG.
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
14 IVIG.
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
7 IVIG.
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
13 situation.
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
9 dramatically.
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
19 future.
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
20 patients.
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
14 batteries.
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
19 are!
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
21 go.
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
10 office.
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
17 costs.
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 th