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Blood Safety Transcripts

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

Eleventh Meeting

Volume II

8:18 a.m.

Wednesday, April 26, 2000

Hyatt Regency Capitol Hill Hotel

400 New Jersey Avenue

Washington, D.C. 20001

PARTICIPANTS

Arthur Caplan, Ph.D.
Stephen D. Nightingale, M.D., Executive Secretary

Larry Allen
James P. AuBuchon, M.D.
Michael P. Busch, M.D., Ph.D.
Richard J. Davey, M.D.
Ronald Gilcher, M.D.
Edward D. Gomperts, M.D.
Fernando Guerra, M.D.
Paul F. Haas, Ph.D.
William Hoots, M.D.
Dana Kuhn, Ph.D.
Karen Shoos Lipton, J.D.
Gargi Pahuja
John Penner, M.D.
Jane A. Piliavin, Ph.D.
Marian Gray Secundy, Ph.D.
John Walsh
Jerry Winkelstein, M.D.

Mary E. Chamberland, M.D.
Jay Epstein, M.D.
Col. G. Michael Fitzpatrick, MSC, USA
Paul R. McCurdy, M.D.
Eric Goosby, M.D.
David Snyder, R.Ph., D.D.S.

CONTENTS

AGENDA ITEM & PAGE NUMBER

Further Committee Discussion of Yesterday's
Agenda, P.4

Reimbursement Policies for Blood and Blood
Products in Response to the Introduction of
New Safety Measures

Statement of Issue - Stephen D. Nightingale,
M.D., Executive Secretary, Advisory
Committee on Blood Safety and Availability, P.119

Food and Drug Perspective, Jong-Hoon Lee,
M.D., Food and Drug Administration, P.126

Public Comment

Committee Discussion and Recommendations

Global Collaboration for Blood Safety - Jean Emmanuel, M.D., Director,
Blood Safety andClinical Technology, World Health Organization, P.311

Committee Discussion and Recommendations

Lunch

Update on Blood Availability -
Paul McCurdy, M.D., Consultant,
National Institutes of Health, P.334

Update on Plasma Availability - Dennis
Jackman, Executive Director, North America
Plasma Protein Therapeutics Association, P.337

Adjournment, P.352

PROCEEDINGS

DR. NIGHTINGALE: Will the committee members please take their seats? Could I ask the committee members to please take their seats?

Good morning. It is 8:15 a.m. For the record, I am Dr. Stephen Nightingale, and this is the second day of the Eleventh Meeting of the Advisory Committee on Blood Safety and Availability. Dr. Piliavin has agreed to act as the Chair of the committee until Dr. Caplan is able to join us this morning.

The first item of business today on the agenda is the conclusion of yesterday's agenda. At the conclusion of yesterday's meeting, for those of you who were not in attendance, there was a vigorous discussion by the committee of two resolutions. Those resolutions--I'm sorry. I was directed as the Executive Secretary of the committee to collate the recommendations of the Advisory Committee into a proposed document, which is posted on the screen behind me. I regret that it is not possible for all the members of the audience to read it. I have made 30 copies of it, and I will provide the copies that the Advisory Committee members do not need to the audience so they can follow along in the discussion.

It is my intent to ask each of the Advisory Committee members to make any comments they wish to make on this document, and then ask if either of the proposed recommendations would be, in fact, proposed and seconded by a member of the committee.

Dr. Piliavin?

DR. PILIAVIN: [Presiding.] It's been suggested that we go in reverse alphabetical order, but if there is someone who is moved to ask a question or make a comment first, I would certainly consider that.

I suspect we need to leave them time to read it.

DR. NIGHTINGALE: Okay.

DR. PILIAVIN: I'm assuming you're all reading it and that's why no one is responding.

DR. NIGHTINGALE: For the record, while you are reading it privately, I might read it out loud.

The first recommendation is as follows: The Advisory Committee on Blood Safety and Availability is favorably impressed with the concept and accomplishments of the error reporting systems that have been developed to improve the safety of air travel by the aviation industry and by the interactions of federal regulatory agencies with this system. The Advisor Committee believes that many of the same concepts successfully implemented by the Federal Aviation Administration, the National Aeronautics and Space Administration's Accident Safety Reporting System, and some airlines--notably, American Airlines' ASAP--could be applied fruitfully to blood banking and transfusion medicine.

Therefore, to reduce the morbidity and mortality associated with human errors and inherent organizational limitations, the Advisory Committee recommends that all blood establishments and transfusing facilities, or health care organizations in which these are based, adopt the MERS-TM or a comparable system as a standard error management system for transfusion medicine.

These error management systems should acknowledge the right of patients to know of any potential or actual harm as a consequence of any blood product received. At the same time, there should be statutory protection from disclosure for voluntarily reported information and of quality assurance activities that are not associated with potential or actual harm, provided that the information is also not associated with reckless or intentionally harmful acts.

These local error management systems would complement, and not replace, current regulatory activities, notably but not exclusively in the area of product safety. All analyses of collected data should be made available in a timely manner to regulatory agencies, national transfusion medicine surveillance programs, and other participants in a reporting program.

Congress should allocate a minimum of $2 million for establishment of these error management systems, and an infrastructure sufficient to support them, in the fiscal year 2001 budget. Congress should stipulate that these funds should not be reallocated for other purposes, and that no other funding should be reduced because of the availability of these funds.

The second discussed proposal reads as follows: Number 2, Because there is a small but non-zero risk associated with use of blood products or plasma derivatives that cannot be eliminated with current technologies, and because neither the donor nor the manufacturer of the blood product or the plasma derivative are liable for this risk as long as current technologies are appropriately used, the Advisory Committee supports the prior recommendation of the Institute of Medicine, and of others, for a national system that would compensate recipients for costs incurred because of injuries directly caused by the administration of blood products or plasma derivatives, and not associated with a reckless or intentionally harmful act, be enacted and funded by the Congress. I apologize for the grammatical error in the last sentence.

Do any members of the Advisory Committee not have a copy? Please raise your hand if you do not. If not, I'll distribute the remaining copies to the audience.

DR. PILIAVIN: So everybody thinks it's wonderful? Jim?

DR. AuBUCHON: Well, I don't wish to throw cold water on this, and I do think Steve did a very nice job putting a lot of different ideas together.

I am somewhat concerned over the fourth paragraph. With all due respect to our colleagues in the FDA, I am concerned that the FDA may become inundated with incident reports from transfusion services. I just did a quick calculation in my head yesterday, and based on our experience, the agency could see something on the order of 5,000 to 10,000 additional reports per month, and I believe the FDA currently gets something like 20,000 or 25,000 a year. Is that correct, Jay? Fourteen thousand, okay. So we're talking about a huge increase, potentially. And the $2 million that we're talking about here would be quickly consumed by the agency in trying to deal with this. I'm not sure that's really the primary focus.

I understand that the agency has a requirement to gather data to ensure the safety, potency, and purity of blood components, and that's appropriate and they certainly should continue that, and they should have access to the data necessary to give the American public confidence that their blood products do meet those standards. However, I would really like to see the data that are generated from hospitals, transfusing facilities, be directed toward some other agency or some other device to capture and analyze the data, similar to the split between the NASA system and the FAA, have the FDA continue to focus on blood products no matter where they're collected, and have another agency attempt to gather the information about how transfusion services are functioning.

DR. PILIAVIN: Where do you see reference to the FDA being responsible for this?

DR. AuBUCHON: Well, in--

DR. PILIAVIN: Paragraph 4 doesn't--

DR. AuBUCHON: Paragraph 4, the second line, particularly but not exclusively--

DR. PILIAVIN: That's analyses.

DR. AuBUCHON: Notably but not exclusively.

DR. PILIAVIN: But, Jim, that's analyses of the data. That's not the data themselves. This is once the data gathering and organizing--organization that's being set up with this money has done some analyses on the data, it's going to go to the regulatory agencies. That's the way I read this paragraph.

DR. NIGHTINGALE: May I clarify? In my attempts to incorporate all the suggestions of the members, both during and immediately after the meeting, I attempted to incorporate the concept that the error management system might be sponsored by a nongovernmental organization, and that the $2 million that was the figure tossed around yesterday afternoon--I won't identify the person who started tossing it because of my Fifth Amendment rights. But it is clearly the sense of several of the members and much of the blood community that the example of the aviation industry inciting the voluntary error management system outside of the Federal Aviation Administration has been an important component of the success of that system. And I hope to incorporate that thought in my draft of the proposal.

I would also mention that proposals at places like this are really interpreted in the context of the meeting in which they're approved--of the recommending meeting. We don't have the luxury here when we make proposals of having a long administrative staff, like the House and Senate does, to polish things. And I think that's part of the force that comes from these recommendations, is that they may be done on the fly, but they do represent the feelings, and we try to use the transcript to identify the context of those feelings.

DR. PILIAVIN: Steve, in other words, this paragraph 4 was never intended to say anything about the FDA getting raw reports?

DR. NIGHTINGALE: Oh, on the contrary.

DR. PILIAVIN: Okay. Ms. Lipton?

MS. LIPTON: But I think, Steve, though, that it can be misinterpreted, because what it does in this paragraph is say--I looked at analyses and thought, oh, that's the analysis that the transfusion service does, and then it goes equally to all those parties. And I did think that yesterday we were speaking of some sort of a hierarchical--you know, the error management system would allow data to go into one place, and then it would sort out this goes to FDA, this goes someplace else. And I don't think that's captured here.

DR. NIGHTINGALE: What suggestions would you make, then? We have plenty of time to get this right. I do believe we can get this right in the space of about an hour.

DR. PILIAVIN: It might help if you put the fifth paragraph before the fourth paragraph. That way the management system would be established, and then you could perhaps clarify the words that are currently in the fourth paragraph to say this management system will do analyses, blah, blah, blah, and send it to these places. Would that help?

MS. LIPTON: Yes. I guess my question is the national transfusion medicine surveillance program, isn't that what we're talking about as being the data collection center, or am I--I mean, a place where this would go? When you wrote that, Steve, what were you thinking?

DR. NIGHTINGALE: Which paragraph? I wrote it late at night?

MS. LIPTON: Paragraph 4, the second sentence. It says it "should be made available in a timely manner to regulatory agencies, national transfusion medicine surveillance programs..." Is that--

DR. NIGHTINGALE: Would it assist you if I were to say any national transfusion medicine surveillance programs? Because we're talking about hypotheticals here right now, and I'm trying to keep this inclusive.

I want to hold discussion, since we're talking grammar here, directly to the questions proposed, and I want to do the questions one at a time, because if we start talking about three grammar points, we'll get lost.

MS. LIPTON: Yes, I mean, I think we can fix the issue. I'm just saying that this I don't believe--and I would trust you to fix it. I just don't think it reflects what we were talking about yesterday. Maybe it would be good to get a sense from the rest of the committee members.

DR. NIGHTINGALE: Okay. Can we--

DR. SNYDER: I'm just a little confused. We're talking about making another local system in the--what is it?--the third paragraph or second paragraph? It seems like the focus is local rather than what I thought we also talked about yesterday, which was a national reporting system. It's not to replace the quality assurance activities in these facilities. It is a national system. That's where I'm confused, when you say these local error management systems.

DR. NIGHTINGALE: Actually, the thought that I was trying to incorporate was Dr. Hal Kaplan's, which is that there is a need for a national coordination of the efforts for one obvious reason, so the information gained at one entity can be shared among them. But the point that Dr. Harold Kaplan has repeatedly made is that unless actions are applied at the local level, they will be ineffective.

So I was trying to put more ideas into the sentence than perhaps it's comfortably bearing.

DR. HOOTS: I think it gets to the point about sequence again. Rather than just moving it up one paragraph, it seems to me that before you talk about in the second paragraph all blood establishments, would it not make some sense to talk about the creation of the actual national data collection and analysis to which they report before we actually talk about what their responsibilities are to report?

DR. PILIAVIN: I just noticed that. That's our problem, that that has been omitted, that the national system has been omitted, and all you've got in here is these little local ones.

DR. NIGHTINGALE: Okay. Would anybody like to--we have a proposal that I believe there is assent for, that the fifth paragraph be moved to the fourth paragraph, and seeing no objection--

DR. PILIAVIN: No, no. I think that's a little premature.

DR. NIGHTINGALE: That's premature, okay. Then--Mary?

DR. CHAMBERLAND: I think what I'm hearing and what I would also be supportive of is that--I think it's not simply a question of rearranging paragraphs. I think there needs to be up front a clear statement that if this is what the committee wants to recommend, the development and implementation of a national surveillance and reporting system, I think some of the language that I and others had worked on last night, you know, a national reporting and surveillance system needs to go first. And then because the second paragraph implies that--and there's no sense of structure. How are hospital or--it says blood establishments and transfusion facilities are all supposed to be adopting a MERS-TM system. Well, on their own? In a vacuum? I mean, there has to be some sort of--if this, again, is where I think we were going, some sort of a national structure to it, whether it's a national point of coordination that works with a group of hospitals in the development and implementation of this in a standardized fashion.

DR. NIGHTINGALE: Then Dr. Piliavin's suggestion is in the second line of the second paragraph, we would amend it to say after "the Advisory Committee recommends," we add the words "development and implementation of a national"--

DR. PILIAVIN: Error reporting system.

DR. NIGHTINGALE: "Error reporting"--

DR. PILIAVIN: Along the lines of what was proposed yesterday.

DR. NIGHTINGALE: Development and implementation of a national error"--can we use the word "error management system"?

DR. AuBUCHON: That's not really the national system. It's--

DR. PILIAVIN: It's really a reporting system.

DR. NIGHTINGALE: Okay. Then we want "reporting," "error reporting system for all blood establishments and transfusing facilities, or health care organizations in which these are based, that would employ the MERS"--

DR. PILIAVIN: Something like that. Dr. Davey--

MS. LIPTON: Could I just offer--

DR. PILIAVIN: Dr. Davey?

DR. DAVEY: Yes, since we're working on that paragraph, that sentence, I think it's important to, at least from my perspective, capture the sense of some of the discussion yesterday that there really are--there is a well-established and clear system already in place for the blood production side of the business where GCMP or CGMP and other structures are in place. And I don't think that paragraph captures that very well. We're really focusing on the transfusion side of the equation. So I would say that we also amend that sentence following the phrase "the Advisory Committee recommends," "that blood establishments and other facilities that transfuse blood and blood products, or health care organizations," rather than "all blood establishments." "Blood establishments and other facilities that transfuse blood and blood products," or "transfusing facilities."

DR. NIGHTINGALE: Give me the words one more time, if you would, please.

DR. DAVEY: Instead of "blood establishments," just say "transfusing facilities."

DR. NIGHTINGALE: So you would like to strike the words "blood establishments."

DR. DAVEY: Yes.

DR. NIGHTINGALE: Jay, because this is your bailiwick, I saw you nodding, and I wanted to make sure that your views were also incorporated in this.

DR. EPSTEIN: Well, see, I think there are two different points. First of all, I agree insofar as transfusing facilities are concerned. However, I think the recommendation for MERS-TM can apply equally to the collecting and processing establishments. So I would rather see us make an independent point about MERS-TM.

DR. NIGHTINGALE: Okay. So Dr. Davey's suggestion is to strike the three words "blood establishments and," and go, "recommends that transfusing facilities, or health care organizations..." I don't see any objection to striking that--

DR. PENNER: Leave "blood."

DR. PILIAVIN: Yes, blood transfusion.

DR. NIGHTINGALE: Okay, that "all blood transfuse"--so we strike "establishments and."

"The committee recommends the development and implementation of a national error reporting system for blood transfusing facilities, or health care organizations in which these are based, that would employ the MERS-TM or a comparable system."

DR. PILIAVIN: Why don't we stop the sentence at that point because Jay was trying to say, I think, that he would like something that says this MERS thing should be involved in the other kinds of reporting as well. It was getting too long.

Mary?

DR. CHAMBERLAND: Well, actually, my concern is that the committee not specifically recommend a named product or system of data collection, MERS-TM. I think at this point the committee should consider being more general rather than specific. We want to certainly come forward with a recommendation for a reporting and surveillance system, but I think there were many comments yesterday that this is not the appropriate venue to be designing that system. And not having familiarity with MERS-TM and what would be required, et cetera, to implement that, I would be more in favor of leaving this more generalized.

DR. NIGHTINGALE: Okay. The intent of the draft--

DR. CHAMBERLAND: It's nothing negative about MERS-TM.

DR. NIGHTINGALE: No, no. I think as everybody knows, certainly the committee that I've talked to this morning knows, the intent was to be inclusive rather than editorial in the draft. I think that to implement Mary's thought, then we might simply strike the last two lines of the second paragraph and just leave it--we've talked about "error management system for transfusion medicine," and not make a specific statement about MERS-TM.

Dr. Gilcher?

DR. GILCHER: Yesterday I had advocated that we state very clearly what the purpose of this was in the first paragraph. It's implied here but it isn't actually there, and could we put it right at the end of the last sentence of the first paragraph: "could be applied fruitfully to blood banking and transfusion medicine for the prevention of future errors," or something to that effect.

DR. NIGHTINGALE: Okay. I attempted to incorporate this with the first sentence of the second paragraph--was I successful?--when I wrote "to reduce the morbidity and mortality associated with human errors and inherent organizational limitations." Is there a thought beyond that?

DR. GILCHER: I would prefer to have it stated in the first paragraph so it's right up front.

DR. NIGHTINGALE: In that case, what we do is we drop a word. We drop the word "therefore," which was rather awkward to begin with. And after "transfusion medicine," we strike the period, and I don't think we need any punctuation at all there.

Dr. Piliavin is going to object to the length of the sentence, as would my high school English teachers.

DR. PILIAVIN: Yes.

DR. NIGHTINGALE: Dr. Gilcher, you are being attacked by the ghost of a very good English teacher.

[Laughter.]

DR. GILCHER: She can modify the sentence and shorten it, but I think it does need to be there.

DR. NIGHTINGALE: Be at the end of the first rather than the beginning of the second paragraph.

DR. GILCHER: Yes, I think it should be in the first paragraph.

DR. NIGHTINGALE: Then we should put a period--then if we put a period and then two words, it would say, "This would reduce..." Or, better still, "The purpose of this action would be..."

Any resemblance to the above-referenced English teacher on my right is purely coincidental.

DR. PILIAVIN: Then there's going to be a period after "organizational limitations."

DR. NIGHTINGALE: Yes.

DR. PILIAVIN: Then there will be a new sentence, which begins, "The Advisory Committee," and there we can have the "therefore."

DR. BUSCH: Perhaps a new paragraph. "The Advisory Committee" could begin the new paragraph.

DR. PILIAVIN: Yes, exactly. And then it would say, "The Advisory Committee therefore recommends," and we go into this thing that we're still crafting here.

DR. EPSTEIN: I'm troubled by some of the overall logic. What we're saying in the first paragraph is that we're endorsing the concept successfully implemented by FAA, the NASA ASRS, and some airlines. We don't, however, state what those concepts are. And the paragraphs that follow don't exactly mirror the concepts of the NASA ASRS system, so I'm left a little bit confused what we're really saying here.

DR. PILIAVIN: Okay. I think what Dr. Gilcher was trying to get us to say belongs in there, in terms of the idea that these kinds of precursor errors are a good predictor of more serious errors, and that's the way their system is set up, and we think a similar kind of system should be set up for blood.

DR. EPSTEIN: Okay. Except that if one is endorsing the concepts of that system, it has many, many other aspects, for example, you know, the consensus approach toward actions taken regarding reported incidents, for example, the exemption from punitive action for self-reported error, for example, the national acquisition and analysis of incidence data.

In other words, I'm simply troubled that if we endorse the concepts and don't state what they are, it's just not clear what we've endorsed. We could say something more general, that based on a review, we recommend the following.

DR. NIGHTINGALE: Okay. Let me try--I do want to respond simply because I felt it important to keep the recommendations to one page, if possible, at this point. My attempt was to keep this from becoming too prescriptive and that I had hoped that by mentioning the NASA aims, for example, that I would incorporate those ideas by reference.

DR. EPSTEIN: Again, I think--

DR. NIGHTINGALE: But it's the sense of the committee we're looking for, not the sense of a particular grammarian.

DR. CHAMBERLAND: I'm not actually sure the paragraph is necessary, or it could be greatly abbreviated, because I think it does lead us down some roads, as Jay has pointed out, that we don't necessarily need to go.

I think the record will speak for itself, the agenda, we heard about this. But it is confusing, and I'm not really sure that we need it.

DR. NIGHTINGALE: I find myself in the fortunate position of agreeing with Dr. AuBuchon, though perhaps not for the same reason, but Dr. AuBuchon and I have--and I want this stated for the record--arrived at the same conclusion. To me, one of the purposes of this particular document is to convey to people who were not here but are in the position of very substantial power what happened at the meeting. And one of the things that happened at this meeting was we heard very forceful presentations by representatives of these organizations or those who contributed to them, and I thought that reference to them would help the movement of this piece of paper through the government.

DR. CHAMBERLAND: I would rather see a reference to the Institute of Medicine report or something along those lines, which occurred at our first meeting, which I think--and as you brought up, our agenda was actually set before the release of that report. But I would like perhaps to see some language that ties into that report, which is really the springboard for a lot of congressional and other discussions that are going on now.

DR. NIGHTINGALE: It's also a springboard for a lot of controversy.

DR. CHAMBERLAND: Well, I stand by my original, which is I'm not sure we really need a preamble.

MS. LIPTON: I was going to suggest--I agree with Mary and Jay in that I think that some of the elements we've changed and I think we want to make sure that someone looking at this doesn't think we meant just adopt that system. But if you just use your first sentence, when it just says we were favorably impressed with the concept and accomplishments and the interactions, and then delete the rest so we don't say we take these concepts and we want to implement them. Instead we go on and then we talk about the elements of such an error management system that we believe, you know, that those elements are important. And that way I think it does tie us more into--I mean, I agree with Mary, but we heard a lot yesterday that I think was very stimulating and very exciting and carried us beyond what the IOM report did.

DR. NIGHTINGALE: That works for me. Dr. AuBuchon?

DR. AuBUCHON: Fine with me.

DR. PILIAVIN: So we're just removing the second long sentence in the first paragraph?

DR. PENNER: One other suggestion and that would be to remove "the concept" and just simply have that first sentence read "is favorably impressed with the accomplishments of the error reporting system," then you don't have to worry about concepts, which Jay is concerned about, and then you've got all that you need.

DR. NIGHTINGALE: Does that seem to work for you? Before Dr. Gilcher speaks, does Dr. Penner's suggestion meet with general head nodding?

DR. PILIAVIN: Yes. It looks okay.

DR. NIGHTINGALE: It would appear to.

DR. PILIAVIN: All right. Dr. Gilcher?

DR. GILCHER: Yes, I was going to recommend something to this effect: "The Blood Advisory Committee recommends the development and establishment of an error reporting system for actual and near misses for the purpose of preventing future errors in transfusion medicine."

That really states very clearly up front what we were told to do.

DR. PILIAVIN: Say that again.

DR. GILCHER: I probably--

DR. NIGHTINGALE: Are you suggesting a first sentence or a second sentence?

DR. GILCHER: A first--well--

DR. PILIAVIN: He's rephrasing--

DR. GILCHER: I was going to make that as the very first sentence, and then we--because the first sentence then states very clearly what we were asked to do. And then we can say that we--if you want to, you can put in that there were other agencies that contributed to the production of this.

DR. NIGHTINGALE: Paul?

DR. McCURDY: I think that whenever you use the term "reporting system," I would very much prefer to put "reporting and analysis." I think the error management implies that and would be satisfactory. But if you say "reporting system" separately, then you ought to include "analysis."

DR. NIGHTINGALE: Ron, while we're getting that set up, and I think Jane is going to volunteer to be the typist, your first sentence would read, "The Advisory Committee recommends the establishment and implementation"--hold on--"of an"--

DR. GILCHER: Whatever words--

DR. NIGHTINGALE: "Error management system."

DR. GILCHER: I think it has to be a reporting system--

DR. NIGHTINGALE: Error reporting.

DR. GILCHER: It's an error reporting system--

MS. LIPTON: I think that's including "analysis" because--

DR. GILCHER: Yes, I agree.

MS. LIPTON: --one thing that's missing that we hopefully will get to later is this concept of corrective action that we said was so important.

DR. GILCHER: As a prior English teacher--joke--the point that I'm trying to make is that the first sentence should very clearly state exactly what we're doing, and then we can put everything after that, because many people will read that first sentence and then bounce around, and then we've established clearly what our intentions are.

DR. NIGHTINGALE: "The Advisory Committee recommends the establishment and implementation of an error reporting and analysis system for transfusion medicine." Do you wish--now where do you want to go from here?

DR. GILCHER: "And the purpose of that is to prevent"--that's the whole issue here, is to prevent future errors in transfusion medicine. It's very clear then exactly what our purpose is.

DR. NIGHTINGALE: I think you would say "to reduce and, where possible, prevent"? I think "to reduce and prevent morbidity and mortality due to human error or inherent organizational limitations."

The first sentence: "The Advisory Committee recommends the establishment and implementation"--

DR. PILIAVIN: You don't have to read it.

DR. NIGHTINGALE: Sorry. My teacher couldn't read my handwriting.

[Pause.]

DR. BATTLES: [inaudible comment off microphone.]

DR. NIGHTINGALE: Would "human and organizational error" do want you want?

DR. BATTLES: I think just error doesn't say where the error occurs. Human error assumes that it was all, you know, human.

DR. PILIAVIN: I kind of like that.

DR. NIGHTINGALE: I think we--Dr. Battles, would you approach the microphone, please? The--

DR. BATTLES: Yeah, I--

DR. NIGHTINGALE: Excuse me for a minute. There is a sense, at least at the head of the table here, that incorporating explicit reference to both human error and organizational limitations would be appropriate in the first sentence, and you are the expert here. Enlighten us regarding your concern.

DR. BATTLES: Yes, I--we want to avoid the concept that all error is human so that we include, you know, the aspect of the latent--some people call it system. I prefer latent organizational--

DR. NIGHTINGALE: How about human and system error?

DR. BATTLES: That would be fine, as long as it's not just the one--

DR. NIGHTINGALE: Human and system? We're okay with human and system around the table?

[No response.]

DR. NIGHTINGALE: Let's do it.

DR. PILIAVIN: Error? Errors? I'm going to.

DR. NIGHTINGALE: The second sentence then would be--or the second paragraph, "The Advisory Committee is favorably impressed with the accomplishments..."

DR. PILIAVIN: Every now and then it pops up to the top of the screen, and I don't understand why.

DR. NIGHTINGALE: While Dr. Piliavin is typing the second sentence, Dr. Epstein has a comment.

DR. EPSTEIN: I wonder if in the first sentence we shouldn't add the notion of a nationally organized system.

DR. NIGHTINGALE: Would you like to put the word "national" before "error"?

DR. EPSTEIN: If you say "a national system" or "a nationally organized system"?

DR. NIGHTINGALE: How about "national" right before "error" and change "and" to "a"?

[Pause.]

DR. NIGHTINGALE: Dr. Caplan has joined us.

Dr. Epstein?

DR. EPSTEIN: And, again, I think we want the notion reporting, analysis, and corrective action system.

DR. HOOTS: I would propose--I think that's a fundamental principle that needs its own statement, and I would propose that we insert a sentence that says, "A fundamental expectation of the implementation of this system is that analyses will lead directly to corrective action."

DR. NIGHTINGALE: There is at least one nod for that as the second sentence. I see two. Do I see any heads shaking?

The nods have it.

DR. GILCHER: Steve?

DR. NIGHTINGALE: Dr. Gilcher?

DR. GILCHER: Again, I want to keep that first sentence short, concise, clear, so that when you read it, you know exactly what the mission is.

DR. NIGHTINGALE: Are you happy with the first sentence as written?

DR. GILCHER: Pretty much. And then I endorse what was just said for a second sentence.

DR. NIGHTINGALE: Okay. Dr. Gilcher, we've got two sentences up there. Have we approximated your intent? Jim, I'll get to you in just a second.

DR. GILCHER: Yes.

DR. NIGHTINGALE: For those in the audience, we apologize for the type that we have used. There's going to be more, and we're going to try our very best through the microphone to keep you appraised of what's going on while we do it.

Dr. AuBuchon?

DR. AuBUCHON: I'm not an expert in error management, but my conception is that corrective action is what a facility does when it uncovers a problem in its own system, and all corrective--to paraphrase Tip O'Neill, all corrective action is local; corrective action is not likely to be national and is not likely to come out of this kind of system. I would--to my mind, the fundamental expectation of the implementation of this system is a better understanding of the problems that lead to errors, allowing each individual facility to make improvements in their own system.

DR. NIGHTINGALE: I appreciate your comment, but sitting slightly to your left is Dr. Epstein and to his left Dr. Nightingale, and those of us who are feds would probably take some issue with the notion that the only corrective action is local. We can do it if we have to. I mean that actually substan--what I'm trying to do is keep this inclusive. We recognize the desirability of the local loop being the primary loop in error management, but we also recognize the necessity of a national loop, and there's tension around the table about which loop is the one that gets the grease.

DR. GOMPERTS: But, Steve, on the second line, you may be able to deal with this by stating, "implementation of local and a national reporting system."

DR. NIGHTINGALE: I'm sorry. In the second sentence, it would lead directly to corrective action. You wanted to add local--

DR. GOMPERTS: No. Second line, first sentence, "implementation of local and national reporting analysis systems."

DR. NIGHTINGALE: Local and national? The nods seem--Karen?

MS. LIPTON: My only problem with local is it sounds like then we're setting up a local reporting system and regional, and I don't think that's the intent. I think what we want to do is say primary responsibility--you know, that the institution itself has a fundamental responsibility here but that we think there is benefit to this national reporting because you can see things that you could not otherwise see and corrective action can sometimes be, you know, something that you don't see in an individual center. You know, Paul and I were talking; it could be a design defect in a product that you don't really see at your local level until you collect sufficient data about the problem.

So I'm really just worried about local because it makes it sound like it's city or state. I think it's institution.

DR. GOMPERTS: But the national system is dependent upon local responsiveness.

DR. NIGHTINGALE: Let me ask if it is the sense of the committee that if we spend too much time on the first two sentences, we won't have the energy to the smithing down at the bottom of the page that might be necessary. I think we're as close as we're going to get myself, and if we spend more time on this, we will run out of gas before we get to the end of this, because we've got a long day ahead of us.

DR. PENNER: Steve, I don't think you need the second sentence. For corrective action, it's assumed in the first sentence, to reduce morbidity and mortality. How are you going to do it? It has to be corrective. And leave that sentence--and that leaves the first sentence as I think was originally planned, is the overall structure, and that's all you need to lead in, and then you can detail it later on as you go down. And that sentence would be redundant.

DR. NIGHTINGALE: Is there a sense that it's redundant? I can't remember who proposed the second sentence, but if that person would identify him- or herself?

MS. LIPTON: Can I--one of the things that concerned me yesterday was just in listening to the MERS-TM was that there was a suggestion in that system, in that reporting system, that there was some benefit to not correcting things immediately that would allow you to see trending. And that concerns me. I think that we don't want people to just think their job is done by reporting a problem as opposed to understanding that there's a responsibility to correct.

Now, maybe that's inherent, but I don't think it's an inherent necessarily in that reporting system.

DR. PENNER: But I don't think that has to be in the first sentence. That can come later on.

DR. NIGHTINGALE: I would suggest a compromise. Since it's not in the first sentence, would you accept the second sentences later on?

DR. PENNER: Yeah, I think we have an opportunity to complete that, but that is just, I think, expanding on the first sentence, and it doesn't need to be, if the plan is to at least set the pattern by the first sentence and then go from there.

DR. NIGHTINGALE: Jay?

DR. EPSTEIN: Perhaps we can address this point in the first sentence where we say, "A national reporting analysis for transfusion medicine," add the words "as a basis for actions to reduce and to prevent."

DR. NIGHTINGALE: Dr. Penner and Ms. Lipton.

DR. PENNER: Yeah, I think if it's all right with the originator of the first sentence.

DR. NIGHTINGALE: Dr. Gilcher, is that--

DR. GILCHER: Yes.

DR. NIGHTINGALE: Okay. The reporting and analysis system. After "transfusion medicine" insert the words "as a basis for action." Are we there, folks, with the first two sentences? I believe we are, and we can move right along to the third.

DR. PILIAVIN: Have we kept this sentence?

DR. NIGHTINGALE: Yes. Did we keep the second sentence or did we strike the second sentence? We struck--

DR. GILCHER: I don't think we need the second sentence with the inclusion of what Dr. Epstein has suggested.

DR. NIGHTINGALE: We struck the second sentence, the sentence that says, "A fundamental expectation..."

Does the now second sentence meet with approval or with nods? Remember, we are in the discussion phase and the nods are for editorial purposes only. We will go through standard committee procedures in proposing, seconding, and discussing, and calling a vote.

Dr. Davey, then Dr. Epstein.

DR. DAVEY: Just one minor change. I think in the second line if we change "transfusion medicine" to "transfusion facilities," then that whole second paragraph of the original falls out, which is already looks like it's fallen out. "Transfusion medicine" is pretty vague.

DR. NIGHTINGALE: Well, it's also pretty broad.

DR. AuBUCHON: To avoid confusion with blood establishments, maybe "transfusing facilities" is the same concept, but different enough language.

DR. NIGHTINGALE: Dr. AuBuchon makes a point that I would support and Dr. Epstein nods, so let's put "transfusion facilities."

DR. EPSTEIN: I wasn't commenting on that.

DR. NIGHTINGALE: Sorry. Jay, you are the regulator. You get the last word on this.

DR. EPSTEIN: Well, I just think this paragraph is so broad, I mean, part of the system's already under national reporting but part isn't. If we're calling for national reporting analysis for the whole, we're pushing toward unifying the databases, and I'm not against that even though I don't think FDA owns the whole ball of wax. So I'm not uncomfortable with this. I think the distinctions--what we're saying that's new regarding transfusing facilities comes later.

DR. NIGHTINGALE: All right. Then we've left "medicine" there because this is broad and we'll get specific later. So we're done with the first couple of sentences. And Dr. Piliavin is typing, I think--

DR. PILIAVIN: I'm just typing from your memo--

DR. NIGHTINGALE: That's right.

DR. PILIAVIN: --until other stuff happens.

DR. NIGHTINGALE: Until there's comments. I think at this point, while Dr. Piliavin types, the committee will reflect, and if anybody has a comment to make, they should make it, Dr. Epstein.

DR. EPSTEIN: Well, in the second paragraph--I'm sorry, the second sentence of the first paragraph, again, I would like to suggest adding the words "and correction." So it would be "error reporting and correction systems."

DR. NIGHTINGALE: Error reporting and correction.

DR. EPSTEIN: Because, again, part of the unique character of what we've heard was how they went about correction. It wasn't merely reporting.

DR. NIGHTINGALE: Jay, it's okay as we are now? Okay. Then Dr. Piliavin will continue to type, and Dr. Snyder will have the floor.

DR. SNYDER: Yes, I'm just a little confused. I thought that we're capturing everything. The FDA has a corrective system. The local facilities have a corrective system. Nowhere yesterday did I get the impression that the near misses that went into the system resulted in that national center doing corrective actions. The corrective actions were always at the company level, the local level. So I'm getting really confused by this.

DR. NIGHTINGALE: No. Later in, for example--I think an important component of the system that we're talking about is that there is a local system that shares information, as we say it later, with other local participants, with regulatory authorities, and with any nongovernmental third party that might administer this system in a timely manner.

Dr. McCurdy?

DR. McCURDY: I think we may be a little hung up on this corrective action issue. I think that there are both local situations and national situations that cry for corrective action. I think that Hal Kaplan overstated the issue yesterday when he talked about doing corrective action after every incident. And, actually, you wouldn't do that unless--well, it would depend on the incident.

But I think there are design flaws that are picked up in aircraft from near misses and actual crashes, and those are national; even worldwide corrective action go into those. There are also procedures locally that change. So I think you need both. I think the ultimate has to be done locally, but I think you have to have a national, a much wider focus.

DR. NIGHTINGALE: Dr. Snyder, did Dr. McCurdy address the issue that you were raising? Because I clearly did not.

DR. SNYDER: In a form. You know, the near misses, I don't know what percentage of those lead to corrective action. It's the crashes that, you know, give the--once they determine the cause of a crash, give the cascade to a corrective action.

DR. NIGHTINGALE: Yes, well, I believe the basic principle, not so much necessarily just from Dr. Kaplan, but certainly what Dr. Westrum and others were talking about yesterday, was if you take--the guiding principle is if you take a near miss as seriously as you take a direct hit, you'll get fewer direct hits.

Dr. Epstein?

DR. EPSTEIN: I think the first paragraph should make a remark about MERS-TM. I think that just as we have been impressed at this meeting and the prior meeting with the model of error management in aviation safety, we were impressed at the last meeting and this meeting by the utility of the MERS-TM system and its approach.

So I think one could separate it from the specific recommendations, but it's worth a remark. I mean, after all, the government on behalf of the people invested handsomely in the development and piloting of an error management system for transfusion medicine, and I think it merits the acknowledgment of this committee that it shows merit. So I think we should craft a sentence after the second sentence of the first paragraph.

DR. NIGHTINGALE: I think if we will wait just a minute, Dr. Piliavin is approaching the bottom of the page.

Give us just a minute and I think we'll go faster in the long run.

DR. PILIAVIN: I can stop this any time.

DR. NIGHTINGALE: Okay. I think we want to go back to the first paragraph and add a third sentence that Dr. Epstein will help us draft.

DR. EPSTEIN: Just a suggestion. In addition to the MERS-TM, I think we also have an excellent example in the blood establishments where the introduction of continuing good manufacturing practices in sort of partnership with FDA has been extremely successfully introduced, and we should acknowledge both in the blood GMP environment and through these preliminary programs in transfusion medicine service environments that there's a good success model.

DR. NIGHTINGALE: Then you might want to begin this new third sentence by saying, "The Advisory Committee is also favorably impressed with the results to date of the MERS-TM system." Let's do that. And--

DR. EPSTEIN: I think we should take the FDA system first. It's, you know, been around for 50 years.

DR. NIGHTINGALE: Dr. Epstein once again makes a point that is difficult to refute. "Favorably impressed with the results of"--I'm sorry, "with the results of"--the first pass would be the FDA's regulatory efforts. Dr. Busch, can you give us better words than that?

DR. BUSCH: Perhaps "the resultsÊof implementation of error management systems in blood establishments under FDA oversight, and blood collection"--

DR. NIGHTINGALE: We might even put a deserved plug in here for the accomplishments of the Food and Drug Administration and the record note that it is not a representative of the FDA who's making that suggestion. "With the accomplishments of the Food and Drug Administration in reducing errors and accidents." Am I okay? To date.

DR. EPSTEIN: Well, yeah, through enforcement of CGMP practices in blood collection facilities.

DR. NIGHTINGALE: Do we need to go into that much detail? This is a preface.

DR. EPSTEIN: As Jim said, the FDA per se didn't reduce the errors and accidents. They encouraged industry to introduce programs that have successfully done that.

DR. NIGHTINGALE: We want to plug in for industry as well as FDA.

DR. CAPLAN: [Presiding.] Do you want to say the FDA and--

DR. NIGHTINGALE: I see representatives of industry nodding.

DR. EPSTEIN: In the blood collection industry, perhaps. Blood and plasma.

[Pause.]

DR. CAPLAN: I think what's missing, Steve, after our statements of impression, is a transition statement. What we want to say is the Advisory Committee recommend this, we're impressed, and it's time, we believe that we can move forward now even further. In other words, the declarations are good. What we want to do is move it toward--

DR. NIGHTINGALE: Okay. Give us words to start the second paragraph, Dr. Caplan.

DR. CAPLAN: At the end of the first, I would just say--we can say something like a great deal has been accomplished, the committee believes-

DR. NIGHTINGALE: "While a great deal has been accomplished, the committee believes"--

DR. CAPLAN: Right. "The committee believes the opportunity"--"now believes the opportunity exists to do even more." And then start the next sentence, you might take out the--

DR. PILIAVIN: What we still need to have is--

DR. CAPLAN: You see what I'm saying? In the next paragraph, first sentence, take out "these" and make it capital E on error. "Error management systems should acknowledge..."

DR. NIGHTINGALE: Art, for the record, your microphone is on. Pull it around.

DR. CAPLAN: What I'm saying is--I'm just trying to make the prose move here so that we just start the next sentence with a capital E, "Error." We'll be syntactically there.

DR. NIGHTINGALE: I guess Dr. Gilcher and then Dr. AuBuchon.

DR. GILCHER: It's very clear, I believe, that on the collection side the errors are as low as they are because of what the FDA has done in terms of guidance and regulation and that that guidance and regulation has not been there on the transfusion side, which is what we're addressing.

I would change the words that say "The Advisory Committee is also favorably impressed with the accomplishments" to "acknowledges the accomplishments of the FDA", because we clearly have, on the blood establishment side.

DR. NIGHTINGALE: So, "The Advisory Committee acknowledges the accomplishment of FDA", rather than "is favorably impressed with."

DR. GILCHER: Yes.

DR. PILIAVIN: I got it, I got it.

DR. GILCHER: You could move the "favorably impressed" down.

DR. NIGHTINGALE: One at a time.

Are we close to the end of the first paragraph? Dr. AuBuchon has a frown.

DR. AuBUCHON: I'm sorry, but the conclusions are not supported by the data. This committee has not been presented with information that allows us to conclude that FDA action or implementation of CGMP has reduced errors, nor to my knowledge are there any papers in the peer review literature that show that blood centers' implementation of CGMP have reduced error. I fully support the use of CGMP and all aspects of transfusion medicine, and I think it is the appropriate thing to do, and it is the least costly thing to do in the end. However, I do not believe that we can say that we have either been shown the data or that we can acknowledge--

DR. NIGHTINGALE: Fine. In that case, let me suggest that we change the word "accomplishments" to "efforts."

MS. LIPTON: Maybe the issue that was in errors and accidents, maybe you can say "in improving blood safety", because I think the issue that people are struggling with is we don't have data on it.

DR. NIGHTINGALE: But "efforts"--

DR. CAPLAN: "Efforts" is good.

DR. NIGHTINGALE: "Efforts" addresses your concern and moves us along.

DR. CAPLAN: We've seen data on efforts.

DR. NIGHTINGALE: And it moves us along which is something that we are going to do today. Underneath this velvet glove--Michael then Mike.

DR. BUSCH: I think that first sentence, personally, I think that should be the beginning of the second paragraph, the preamble of the Advisory Committee is impressed, and then--because you need to then transition to "now we recommend." And I think that first sentence in the first paragraph is really a very strong follow up to the end of the first paragraph, just taking us to the point, "We now recommend establishment of a system", so--

DR. NIGHTINGALE: What Dr. Busch wants to do is to take what is now the last sentence in the first paragraph, and make it the first--

DR. BUSCH: No, the first sentence of the first paragraph, which is really saying what our recommendation is--I know this is going back and forth, but I think the rest of that paragraph is really a summary of "We were favorably impressed with these examples we've been presented with. And in light of this, we now recommend establishment of a program for transfusion medicine." So just move that first sentence, and it becomes an excellent beginning to what we're actually recommending.

DR. NIGHTINGALE: It may, but we do not have unanimity there. Dr. Gilcher wanted that first sentence to be the first sentence, and he spoke for the reasons behind that.

DR. GILCHER: And I still feel that way. I don't mind repeating it to some degree. I would have--I actually would have favored having the first sentence be a first paragraph, period. You read it, you understand, and then it's very clear, because that is what people will look at, is the first sentence should really tell you everything that's going to come after that.

DR. NIGHTINGALE: Then Dr. Busch.

DR. BUSCH: That's fine, but I do think then we need to in essence repeat it or somehow say, "We now recommend." There's a little redundancy to it, but that's fine.

DR. NIGHTINGALE: Okay. Are we done with the first paragraph?

DR. CAPLAN: Yeah, move it.

DR. NIGHTINGALE: Colonel Fitzpatrick?

COL. FITZPATRICK: In listening to all the conversation, the last sentence talks about accomplishments, but the accomplishments you're referring to are in the blood collection arena, and you want to move to transfusion medicine. So I would suggest you say, "Well, a great deal has been accomplished in blood collection. The Committee now believes that the opportunity exists to apply those principles to transfusion medicine as a whole", and there's your transition.

DR. NIGHTINGALE: We're getting long. This first paragraph is getting long.

DR. CAPLAN: Let's let it stand. Let's move to the next one. We'll come back and see how it works.

DR. EPSTEIN: I would like to concur with what Colonel Fitzpatrick said. I believe that Dr. Davey agrees as well.

DR. NIGHTINGALE: Give us language, the last sentence of the first paragraph.

COL. FITZPATRICK: Just "has been accomplished in blood collection and processing. The Committee now believes that the opportunity exists to apply those principles to transfusion medicine as a whole."

DR. AuBUCHON: Transfusion facilities.

COL. FITZPATRICK: Well, I used "medicine." I think we ought to stick to "medicine."

DR. NIGHTINGALE: Are we done with the--we need to be done with the first paragraph soon.

DR. HAAS: Can we just go back to Ron Gilcher's point though, the first sentence, the first paragraph? I agree with him strongly that that sets them up.

DR. NIGHTINGALE: All right. We can insert a new paragraph after the end of the first sentence of the first paragraph. Going once, going twice. Okay.

The third paragraph is--

DR. PILIAVIN: Do we want to have a "1" here because we've got a "2?"

DR. NIGHTINGALE: No--yes, that's correct.

Okay. Dr. Epstein.

DR. EPSTEIN: It's a small point, but where we say "FDA working with the blood collection industry", I think we should say "blood and plasma."

DR. NIGHTINGALE: Okay, and that "industry" should be plural, "industries."

The third--let's move to paragraph number 3. Comments on the third paragraph? Dr. Kuhn?

DR. KUHN: I was just wondering if the word "potential" needs to have more explanation. "Potential or actual harm." Is "potential" what we are trying to convey?

DR. NIGHTINGALE: Yes. And I have a suggestion that--why don't we put "risks?"

DR. KUHN: Or what I was wondering is if it's a duty to disclose. Is that--

MS. LIPTON: Yeah, but I think that's right. Risks is really what we're talking about.

DR. NIGHTINGALE: "Risk" was the word we were talking about. "Should know of any risks", or "risks or actual harm?"

DR. KUHN: Why not "harm?" Why do we have to have "actual harm?"

DR. PILIAVIN: We know about risks, but--

DR. NIGHTINGALE: Okay, "Risks or harm." "Risks or harm?" Nods. Let's go with "risks or harm."

MS. PAHUJA: Are we going to take out the word "actual" or--

DR. NIGHTINGALE: We're taking back "actual."

DR. PILIAVIN: Are we leaving "potential" in there?

DR. NIGHTINGALE: We're getting rid of--"potential" is being replaced by "risks", and "actual harm" is being replaced by "harm" for the reason that harm is harm.

Let's see. Dr. Winkelstein and then Dr. Chamberland.

DR. WINKELSTEIN: I'm pleased with the change to "risk" instead of "potential", but I'm still unclear as to whether we're talking about something that could happen, that you would notify before the transfusion, or once there's a call-back for a new infectious agent that you may have received in the past? Which way--I'm unclear as to when to--

DR. NIGHTINGALE: Well, "risk" does both, actually.

DR. WINKELSTEIN: Pardon?

DR. NIGHTINGALE: I believe that "risks" would incorporate both those concepts; would it not?

DR. WINKELSTEIN: Both.

DR. NIGHTINGALE: Yes.

DR. WINKELSTEIN: Okay.

DR. CHAMBERLAND: Well, currently, I guess--and I share Dr. Winkelstein's confusion because currently we require informed consent, don't we, prior to the administration of? Is this what you're getting at?

MS. LIPTON: This isn't informed consent. This is--

DR. CHAMBERLAND: But that way it sounds like duty to disclose information afterwards. I mean, informed consent is what you get from someone before you undergo some kind of treatment. This addresses the issues of if we find out something, that we are saying we think that there is a duty to disclose that, whether it was that we know that something happened to you in the hospital, or we now know something about a donor who came in. My only concern with "risk" is I wonder if we want a qualifier on that, because you know, there are risks to everything, and do you want to put in "material risks" or, you know?

DR. WINKELSTEIN: So my understanding is that this clause refers to something that might be discovered by science as a consequence of a given transfusion, not informing the patient that all of these things might happen before. I think that ought to be very clear in here. It's just not as clear as I would like it to be.

The other question I had, which is--I didn't know whether I should bring it up now or later, but perhaps now will save us time later. Are we excluding IV gamma globulin in all of this? And the reason I bring that up is at the moment we have no informed consent for intravenous gamma globulin, yet for obvious reasons there is some risk to it. Is it included within the whole document?

DR. NIGHTINGALE: This is the reason why we're using the word "risk" and the word "harm" without either a definite or an indefinite article before it.

MS. LIPTON: And again, this doesn't address informed consent. This is an issue of the duty to disclose something afterwards.

DR. WINKELSTEIN: Well, I understand that, but I'm still confused as to whether IV gamma globulin would be included in this definition of "blood product."

DR. NIGHTINGALE: Sure.

MS. LIPTON: Sure.

DR. CAPLAN: Absolutely, yeah, absolutely.

DR. WINKELSTEIN: Because at the moment there is no legal requirement for disclosure before or--well, clearly after, but not before.

DR. NIGHTINGALE: And of course, this is not--the fact that we recommend something is not the statement that it is a legal requirement. We are making a recommendation. We are not interpreting existing law.

DR. WINKELSTEIN: I understand. I'm just reflecting practice here.

DR. NIGHTINGALE: Yeah. Dr. Kuhn and then Dr. AuBuchon.

DR. KUHN: Would it be acceptable to put--after the rights of patients, to put in, "And the duty to disclose any risk or harm?" Then you're naming the duty to disclose. You're naming it.

DR. CAPLAN: You could just say--

DR. NIGHTINGALE: I think the right to know and the--the right to know is in fact distinct from the duty to inform.

MS. LIPTON: I think I see the issue that's happening here. It's really, "that have been suffered as a consequence" is really what you're talking about. I think what the--the use of the language up there does suggest that, well, do you have to inform them ahead of time too and afterwards. I think what we're focusing on is if you suffered, actually suffered risk or harm as a result--

DR. NIGHTINGALE: Yes, if you enjoyed risk or suffered harm. No, the point is, I think the reason why we're focusing on the--

MS. LIPTON: Not "that may be suffered", "that has been", because you're trying to address the after.

DR. NIGHTINGALE: I think actually the reason why I would suggest we continue to focus on the right to know rather than the duty to inform in this particular place, is that the right to know is easily identified with an individual person. The duty to inform is much more--there's many more people involved than we're going to cover in a sentence here.

DR. CAPLAN: Actually, the real issue here is not just the duty to disclose, it's to make sure that the patient knows.

DR. NIGHTINGALE: Yeah, and we're focusing--

DR. CAPLAN: We can satisfy by the disclosure--

DR. NIGHTINGALE: And we're focusing on the patient here.

MS. LIPTON: Yes. And that's why I think we're trying to get away from informed consent and everything. We're saying this person needs to have this knowledge personally about this.

DR. CAPLAN: Right, right. So it's not enough to have disclosed it; we've got to know it. I could disclose it in the obscure press.

MS. LIPTON: Just because I told you you might get hepatitis B, we're saying that's not good enough. If we know you had it afterwards, then you have to tell that person. It's not enough to say, "Well, you may get this from it."

DR. NIGHTINGALE: Okay. After we hit the back arrow key here, we'll--

DR. CAPLAN: Yeah, Jim.

DR. AuBUCHON: I would like to make sure that this discussion about patients' rights to know and this recommendation is focused on errors and accidents that may occur. I'm concerned that the statement, as it currently stands there, is a blanket endorsement of any and all look-back that may come up in the future. And I view look-back as a public health effort which has some laudatory goals and some results that may, frankly, not be worth the effort in some cases. I don't think it's the--I don't think it should be this Committee's approach to just endorse all potential notifications. We should focus on errors.

Therefore, rather than--at the end of that first sentence there, rather than saying "suffered as a consequence of any blood product received", "suffered as a consequence of any error or accident in the transfusion process."

DR. NIGHTINGALE: Okay.

MS. LIPTON: I think Jim's right. This was we'll give--you know, patients are willing to give up not having access to this database and having that disclosed, as long as on the other side, they know that if there has been an error or accident that causes potential harm or actual harm, that they will be told.

DR. CAPLAN: So we're looking for a sentence here that says, "Error management system should acknowledge the right of patients to know of any risk or harm suffered as a result--as a consequence of any error or accident, period?" Is that where we want to be?

DR. NIGHTINGALE: We've suddenly changed the concept here, folks, and we may want to have changed that concept, but I don't think that all of us would. We originally had "blood product received." If you've got a blood product received, and there was something that happened as a result of it, the patient wants to know that.

MS. LIPTON: But that's not--we were focusing on--

DR. NIGHTINGALE: The error accident--patient rights here.

MS. LIPTON: But we were focusing on the collection of the database. What is the information that's going to be in that database? That was the tradeoff we were looking at yesterday.

DR. CAPLAN: Is this handled by the error management systems part? Presumably they're collecting error.

DR. AuBUCHON: This refers to the error and accident system. That's why we're putting it in here.

DR. CAPLAN: But I mean, the error management system is only going to have data on error, is my point, most likely, so there's no worry that it's going to be a broader mandate.

DR. NIGHTINGALE: Yeah. Dr. AuBuchon just wiped out product liability here.

DR. AuBUCHON: I don't believe so. All I'm saying is that patients should be told if they have suffered some harm as a result of an accident or if they're at some increased risk as a result of an accident. The issue of whether or not we should conduct look-back for Disease X 30 years from now, should be discussed 30 years from now.

DR. CAPLAN: Right, but that won't be in the error management system. That's my point.

DR. AuBUCHON: It would not be part of the error management system.

DR. CAPLAN: So that it's narrower that way. That's what I'm saying. Know what I mean? I understand what your point is, but if we just say "Error management systems should acknowledge the right of patients to know of any risk or harm suffered as a consequence related to blood products received." It is in the error management at the end, so I suspect we can do a little deletion there somewhere. We don't need "the consequence of any error or accident" part.

MR. WALSH: I think it's more--you know, it's error reporting, not error management, first of all. And I also think that we should go to extremes not to limit the right of the patient to know, and the error may occur as the result of a bad product, and the patient should be advised of that. Are you suggesting otherwise?

DR. AuBUCHON: No. I agree that if after transfusion it is found that the infectious disease testing performed on the unit was not accurate and the patient may be at increased risk, by all means tell the patient, get them tested. But as it was originally worded, I believe that first sentence was an endorsement for all look-back programs in the future, and I don't think that that is appropriate or would be supported by public health officials, that those look-back decisions should be handled independently, and we should not as a committee give a blanket endorsement for time immemorial of look-back purposes.

DR. CAPLAN: I want to jump in on this one, because I don't think that's what we've got here. We still have what the error management system has to acknowledge about the right of patients to know, and the broader look-back question isn't at issue here I would say. It's not going to follow out of there as a consequence one way or the other. Jay?

DR. EPSTEIN: It does follow. It's a somewhat obscure reason, but the FDA regards post-donation information as an accident, okay? That's the reason that post-donation information such as that which may cause look-back is being captured in this text.

DR. NIGHTINGALE: So in fact, Dr. AuBuchon's suggestion had the unintended consequence of achieving just what he wished not to achieve. Is that correct?

DR. AuBUCHON: I agree with his point. I just don't think that we've completely remedied the paragraph yet.

DR. NIGHTINGALE: Did you agree with mine?

DR. AuBUCHON: Well, yes. And I agree that post-donation information, in some circumstances, is something that should be conveyed to the recipient if there is really harm or risk that the recipient may have suffered.

DR. NIGHTINGALE: Do we leave the sentence as it is? There's agreement that we leave the sentence as it is? Dr. Snyder has a comment.

DR. SNYDER: Yes. Why are we calling it error management, rather than error reporting? We're shifting gears here again. I thought everything was error reporting and analysis.

DR. NIGHTINGALE: That is error management. Reporting analysis and correction is management. No or yes?

MS. LIPTON: I think what Dr. McCurdy had suggested, that it's not just reporting, that he was--as long as it said error management he was happy.

DR. NIGHTINGALE: Yeah. Let's go back to management if we could.

DR. WINKELSTEIN: I think it's more precise to say "reporting and analysis" than "management." "Management" implies an active process that I'm not sure--I agree with the folks to the right. I think it's "error reporting and analysis." "Management" is a different concept.

DR. NIGHTINGALE: "Management" is reporting, analysis and correction. I think that's what we're looking for, and that's what we're using the word "management" here to incorporate three--

DR. WINKELSTEIN: An active process--

DR. CAPLAN: Why don't we put in--later, not now--"Management is reporting"--

DR. NIGHTINGALE: That's already in the transcript of the meeting and will be understood and will save us a few lines here, perhaps even a few minutes.

Okay. Are we--we may be close to the completion of the wordsmithing. Before this section we need to be, even if we're not. Are there any other--let's review it and keep going.

DR. PILIAVIN: This is where we are.

DR. NIGHTINGALE: Okay. "At the same time there should be statutory protection from disclosure for voluntarily reported information and of quality assurance activities that are not associated with potential or actual harm, provided that the information is also not associated with reckless or intentionally harmful acts."

I see nods. We could move on to, "These local error management systems would complement and not"--

DR. PILIAVIN: You mean "local", not--

DR. NIGHTINGALE: Local, local error. All right.

DR. CAPLAN: Keep going. You were humming.

DR. NIGHTINGALE: We're going. "Local error management systems would complement"--Dr. Gomperts?

DR. GOMPERTS: This is the first time in this document you're mentioning local error management systems.

DR. NIGHTINGALE: Dr. Epstein?

DR. EPSTEIN: I would suggest striking the word "local."

DR. NIGHTINGALE: Let's strike it. And do we see nods? We see nods.

DR. CAPLAN: Doesn't matter.

DR. PILIAVIN: And we're going to have to go back to "these."

DR. NIGHTINGALE: "These errors?" Yeah, we do. Jay, is "these" okay?

DR. EPSTEIN: I would change "would" to "should."

DR. NIGHTINGALE: "Would" to "Should." That's an important change. Okay.

"All analyses of collected data should be made available in a timely manner to regulatory agencies, national transfusion medicine surveillance programs and other participants in a reporting system." Are there concerns? Dr. AuBuchon?

DR. AuBUCHON: Well, this is back to the word "local." I understand that the focus of this Committee is federal primarily. We're making recommendations to the Secretary, but through HCFA, if not the FDA, the Secretary can certainly have an impact at the local level in each individual hospital. And I'll return to my previous comment, that corrective actions need to be local, that we do need to have local management--error management systems. And it seems that we aren't mentioning that.

DR. NIGHTINGALE: Well, we did in January. How would you want to incorporate it here, Jim? This is not a new concept.

DR. CAPLAN: It probably doesn't need to get mentioned explicitly though. It's sort of--it's implicit again in that error management systems topic.

DR. NIGHTINGALE: That would by my suggestion. Then we move on to the portion to which we provide unsolicited advice to the Congress. "Congress should allocate a minimum of $2 million for establishment of these error management systems and"--perhaps, "and for an infrastructure sufficient to support them in the FY 2001 budget."

I see Dr. Epstein's hands, for the folks in the back. I will read the second sentence and then ask him to comment.

"Congress should stipulate that these funds should not be reallocated for other purposes. No other funding should be reduced because of the availability of these funds." Dr. Epstein?

DR. EPSTEIN: I don't know where the number, 2 million, comes from based on any data that we're reviewed. I would rather just say "sufficient funds", and I would add at the end of that paragraph a statement such as, "Funds necessary to maintain these systems should be appropriated annually."

DR. NIGHTINGALE: Okay. If the Committee does not wish to specify a specific number, the Committee would leave it to the discretion of others to do so. Does the Committee wish to leave it to the discretion of others to do so?

DR. GILCHER: I agree with Dr. Epstein.

DR. CHAMBERLAND: I concur. I don't think we--and I'm actually not entirely comfortable with the language that--and I think, Jay, were you in favor of striking "Congress should allocate", that it be a more general statement, "That implementation and maintenance of this would require adequate--identification of adequate resources", or something like that. But there are a lot of different funding paths this could come down from, so I would be more in--making it a little bit more general, but nonetheless acknowledging--

DR. CAPLAN: What's the case for mentioning a number?

DR. CHAMBERLAND: Because we haven't designed the system.

DR. CAPLAN: No, no, no. I'm just asking what's the case for the number? Is there any reason to specify any--

DR. NIGHTINGALE: The case for is that if someone decides that this is a good idea, and decides that Dr. Epstein can do this for 100,000 bucks, then Dr. Epstein's going to have to do it for 100,000 bucks, or encourage a lot of people to work overtime without pay.

DR. WINKELSTEIN: But doesn't his suggestion to substitute the word "sufficient funds" cover that contingency or possibility? I'm in favor of saying "sufficient funds."

DR. CAPLAN: Uh-huh.

DR. SNYDER: If I can throw something in, you know, you're better off that it's authorized up to--you know, a number, because otherwise, you're not giving Congress any direction at all. They may be $10 or $100 or $1,000. You just don't know, number one.

Number two, just because they authorize it means nothing. And the word "allocation" doesn't mean a lot to me. It's appropriation. And if you don't ask for subsequent years, which is Jay's point, you're not going to get them.

DR. CAPLAN: Paul?

DR. McCURDY: I think that if there--if the figure 2 million came from other than Mount Sinai, then it should be included as something for Congress to shoot at, but if it doesn't have some reasonable analysis and support behind it, then using "sufficient funds" or some words like that would be desirable. It can be added, if you wish, after analysis by the executive branch, or something along that line. That is, I think the department might be encouraged to determine what a reasonable sum was and request it. But I think it depends on what the support for the 2 million is, at least for me.

DR. NIGHTINGALE: I think one of the figures that was quoted around yesterday was that the Food and Drug Administration might be faced with 100 times the current number of error and accident reports if some of our goals were to be implemented, and round numbers, that would cost a lot of money.

DR. EPSTEIN: And it wouldn't be sufficient.

DR. SNYDER: One other point. You've got--of "these error management systems", are you talking about the local level, the state level, the federal level?

DR. NIGHTINGALE: The whole thing.

DR. SNYDER: So you're proposing to have a national--

DR. NIGHTINGALE: Expenses would be incurred, both by the Food and Drug Administration, by the local transfusion facilities, and by any intermediary that might administer the voluntary aspect of this program.

I think one of the things I might suggest is this: the Institute of Medicine recommended $35 million to establish error management programs throughout the medicine industry for the first year, and recommended a higher figure, actually 100 million, for subsequent years. The administration recommended $20 million. Blood and blood products collectively are about 5 percent of the whole health care budget. Blood's about 5 billion. The whole health care expenditure's a trillion. So to benchmark, you might ask for a proportional share of the error management budget, and that would be about $1 million for what was proposed by the administration and closer to 2 million for what was proposed by the IOM.

DR. DAVEY: I understand your reasoning, Steve, but I do think those of us that have kind of been in the business of setting up studies, $2 million won't go far. I mean, it will get a couple good pilots to check out this system, but for the scope of what's being proposed, that's a small number, and I think we should not specific a number, say something like "appropriate funds to be allocated."

DR. NIGHTINGALE: There seems to be a sense for appropriate funding. Dr. Linden has a comment.

DR. LINDEN: I think at the previous meeting I gave a figure related to the New York system. If you annualized--excuse me, not annualized--extended to the entire country what we've been doing in New York, it would be about a million dollars a year, but that's for basic recording and tracking with some assistance in terms of corrective action. The 2 million would be twice as much as that, and would include start-up costs, which would probably be considerable, but that's really equivalent to what you're talking about for a national reporting system.

I'm very concerned about the nomenclature of the error management systems. I see that as what the local facilities are doing and what--the national reporting system, whatever that's going to be, is really truly a reporting system. If this is intended to include support for hospitals implementing or enhancing their existing error management system, then 2 million would be grossly insufficient. If it is for the national reporting system, it's probably in at least an order of magnitude ballpark based on our experience.

DR. NIGHTINGALE: Thank you very much.

DR. CAPLAN: Let's go down. Mike, you were waiting?

DR. BUSCH: I basically have the same thing to say. I think we should focus this on supporting the national reporting system, and exclude the considerations of cost, because I don't think we have any sense of what the cost will be to the local hospitals, and that's where the bulk of the costs will be, and some mechanism--for them to really be able to afford to do this, some mechanism needs to be developed to do that.

DR. NIGHTINGALE: So is there a sense for "sufficient funds?" Perhaps "sufficient funds to support this program, including a minimum of $2 million for establishment of a national data reporting system."

DR. CAPLAN: I'm happy with that.

DR. CHAMBERLAND: I would like to check my facts here, but I mean if you truly want an active hospital-based system of surveillance, AIDS reporting in this country, which is done out of state and some selected local health departments, I believe has been in excess of $100 million. I mean, if you're truly talking about implementation of standardized active systems of surveillance, $2 million is really, as Rick said, is going to buy you pilots maybe in 5 hospitals for a year or two, so--

DR. CAPLAN: The political goal is to make sure it's adequately funded, so the only question is: does mentioning a number get there? And my sense is that what's likely to happen here is that this recommendation is going to get chewed on all over the place by other hands--that's a mixed metaphor--but it will probably be about as good as we're going to do to say--to get sufficient funds.

DR. NIGHTINGALE: All right. Do we leave 2 million in or do we take 2 million out?

DR. CAPLAN: I'm voting for taking it out.

DR. NIGHTINGALE: Taking it out? The taking-it-outs have it.

DR. CAPLAN: I would like to see--a suggestion I have is the establishment of the system, and for an infrastructure sufficient to maintain and support it.

DR. NIGHTINGALE: To support or support and maintain.

DR. GUERRA: Mr. Chairman, I think that one could perhaps footnote that with the example of what it costs to support and maintain the Vaccine Adverse Event Reporting System that has a lot of experience now over quite a few years, and costs a lot of money to establish.

DR. CAPLAN: We can do that. We don't have to put it up here, but we could in the text mention Dr. Linden's point, what Mary had to say about the cost of this vaccine reporting thing. We'll get that into the--

DR. PILIAVIN: I can just write "footnote" in brackets there.

DR. CAPLAN: Yeah. And we'll cite that in a text discussion. That's ball-parking better for numbers.

DR. NIGHTINGALE: Are there any other comments for this--that people wish to make on this resolution at this time?

DR. PILIAVIN: Wait a minute. We've got the last part.

DR. NIGHTINGALE: Do you want to do--okay, let's go--

DR. CAPLAN: Just go to 2.

DR. NIGHTINGALE: I will read for those--Dr. Epstein?

DR. EPSTEIN: Well, we're saying "appropriate sufficient funds to support and support." I think it's "to develop and support." "Congress should appropriate sufficient funds to develop these systems and for an infrastructure sufficient to support and maintain them."

DR. NIGHTINGALE: Thank you.

DR. PILIAVIN: I should take out that "support."

DR. NIGHTINGALE: Yes. Okay. We'll go to the second one then. "Because there is a small but non-zero risk associated with the use of blood products or plasma derivatives that cannot be eliminated with current technologies, and because neither the donor nor the manufacturer of the blood product of the plasma--of the blood product or plasma derivative are liable for this risk as long as current technologies are appropriately used. The Advisory Committee supports the prior recommendation of the Institute of Medicine and of others for a national system that would compensate recipients for costs incurred because of injuries directly caused by the administration of blood products or plasma derivatives and not associated with a reckless or intentionally harmful act", period.

DR. CAPLAN: Karen?

MS. LIPTON: A couple things. I would recommend that we delete the phrase "because neither the donor nor the manufacturer of the blood product or plasma derivative are liable for this risk as long as current"--I mean, I just think what we're trying to do is just say, you know, we can't get to zero risk. We need a compensation program. I think getting into those statements of liability is just going to dig us into a hole.

DR. NIGHTINGALE: Take out the second "because" is what you're saying?

MS. LIPTON: Take out "because", all the way down to "used comma", and then just say, "The Advisory Committee supports the prior recommendation of the Institute of Medicine and others, that a national compensation--that a national system to compensate recipients"--and I think we need the concept prospectively.

DR. NIGHTINGALE: Okay. But take out the first two "becauses", take out both "becauses" and add "prospectively?"

MS. LIPTON: No, you can leave the first because, "Because there is a small but non-zero risk", although I, as an English teacher, I would never start a sentence with "because."

DR. NIGHTINGALE: Okay.

MS. LIPTON: But I don't care.

[Laughter.]

MS. LIPTON: I just want to get those over with.

DR. CAPLAN: Let's get rid of the "because." We can simply have a sentence, "There is a small but non-zero risk associated."

MS. LIPTON: You could just say then, "The Advisory Committee supports the prior recommendation of the Institute of Medicine and of others."

[Pause.]

DR. NIGHTINGALE: Karen, where do we put--

MS. LIPTON: So you can say, "The Advisory Committee, therefore, supports the prior recommendation." I mean--

DR. PILIAVIN: I don't think we need the commas.

DR. CAPLAN: Dr. Piliavin's suggestion carries. She is at the typewriter.

MS. LIPTON: "And of others." Instead of--"of others, that a prospective national system"--comma, national system.

DR. CAPLAN: What's the prospective?

MS. LIPTON: Well, I think what we were talking about was that we don't want to get this hung up on dealing with past injuries. We want this to be successful, and I think to do that, we have to say, "Going forward now, this is what we'd like to do", rather than dragging in a lot of stuff from the past that we've been dealing with. I'm just concerned that we won't get anywhere in Congress.

DR. CAPLAN: I understand what you're saying. I don't think it's a prospective--

DR. BUSCH: Yeah. How about saying that the national system to compensate future recipients.

DR. PENNER: But this is not related to administration of blood. It's due to the inherent problems of blood itself, so this is misphrased. You're not trying to compensate people for the administration process. It's for the inherent risks involved with the blood products themselves.

MS. LIPTON: Yeah. Actually, I wasn't going to use "administration." I was going to say "transfusion" rather than--

DR. PENNER: Then be specific. It's really "inherent risks involved with the blood products."

DR. PILIAVIN: Take out "administration", "caused by blood products."

MS. LIPTON: Right.

DR. PILIAVIN: So how much do I take out?

DR. NIGHTINGALE: Take out "the administration of."

MS. LIPTON: But you were questioning where "prospective" goes? Right, you could use "future."

DR. DAVEY: Steve, I think--I agree with Karen, that we've got to have "prospective" in there somewhere. Where it is it seems fine. I think it's a good idea. But I do think that Dr. Penner--and Mary and I have been talking about it--are getting into something too. When we say about risks or :injuries caused by blood products or plasma derivatives", we've got to be a little more specific. Are we talking about hematic transfusion reactions? Are we talking about fevers? Are we talking about unknown viruses? There are a lot of risks of blood products, and I think we should be more specific. We're not compensating patients for all of those risks, are we, future risks?

DR. NIGHTINGALE: Well, actually, I think so, and I think there was an inference that I don't think you intended in your remark there, that there would be thousands of transfusion reactions, because as we know, there are a lot of serious ones. There may be--it is expected that there will be fewer if we adopt universal leukodepletion.

DR. DAVEY: But how is that different than anesthesia risk or something? I like the idea of this, but--

MS. LIPTON: I think yesterday we were talking about no-fault compensation, which means that--you know, to get out of litigating all of these issues, we've set up a system that compensates people for injuries related to transfusion. I mean, that's what I thought the discussion was.

DR. NIGHTINGALE: We're crossing a rubicon here, Dr. Davey.

DR. CAPLAN: However, it doesn't mean that there would not be review of administrative assessment, so having this is not an open door to every fever claim. Mary?

DR. CHAMBERLAND: I think Dr. Penner is on the right track, that we have to provide a little bit more justification or background for this, because--I mean, I think we're familiar with the issues in this Committee, but you know, why shouldn't people be compensated for anesthesia-related injuries, you know, a national system for this and such. So I think, you know, we talked about the uniqueness of blood, that it's a biological product, et cetera. So some of that kind of language that we try and set blood a bit apart from the usual medications or procedures that people might undergo.

DR. CAPLAN: Larry?

MR. ALLEN: I'd just like a little clarification on the part that says "compensate recipients for cost incurred." That's, to me, kind of vague, and I think that's kind of limiting the compensation.

MS. LIPTON: I was going to suggest--I had done some edits, and I was just going to say "compensation for injuries." I was going to leave the "cost" out of it. I mean, that's what most systems kind of figure out. They have to go through and attribute a dollar value to certain types of injuries.

DR. NIGHTINGALE: Yeah, costs incurred, yeah.

DR. CAPLAN: We'll go down--

DR. SNYDER: Yeah, I've got one question. Is there a pressing reason why we're moving forward with this? Because it would make sense to have the vaccine injury compensation people come in, because this is a lightning rod, and I think forewarned is enough. The first part, we know--we don't want to tie them together, in my opinion.

DR. CAPLAN: Just two comments about this. On Mary's point, is blood different, or does the world need no-fault in health care? My attitude is, on the blood side, it's been a mess, so it's time to say that no-fault is applicable here. I remain neutral about whether the world would benefit from having no-fault across health care.

And on the lightning rod aspect, I mean, the Committee should be advised that it is being a lightning rod.

[Laughter.]

DR. NIGHTINGALE: Not for the first tie.

DR. PENNER: Could we add "unavoidable" to "injuries?" Would that maybe clarify it a little bit, "unavoidable"--

MS. LIPTON: I think then you'll litigate forever what "unavoidable" is.

DR. CAPLAN: I mean, again, remember, when you have a system that's no-fault, it doesn't mean having been through automobile accidents and no-fault places, that no one reviews it. It just moves out of a tort framework to something different.

DR. KUHN: Maybe like we had said before, we could reference--although it's not a perfect system, it is a good model--the Vaccine Injury Act, and maybe we could reference that.

DR. NIGHTINGALE: I think that since we have not had, as Dr. Snyder pointed out, an explicit two-day discussion of the Vaccine Injury Act, that the point of putting it in here is dubious to me. Everybody--people who will be reviewing this do have intimate knowledge of the strengths and weaknesses of that program, and there are both. And we are not wedding our recommendation to that prior knowledge. This recommendation would come on its own merit from the Committee, and on the merit of discussion we had yesterday.

MS. LIPTON: And i think also it's important that we just said "a national system." It's not necessarily going to--I mean, it could be a privately-administered system. I think we should just, you know, leave that all to further study.

DR. CAPLAN: My only gripe about this is I'm still not happy that we've wordsmithed correctly the prospective or future-oriented--"prospective national system" isn't the right phrase. I know what you're looking for. We'll work on it, I guess, if we can't wordsmith is--

DR. HOOTS: What about "future recipients" then?

DR. CAPLAN: No.

DR. PILIAVIN: What's wrong with "future recipients?"

DR. NIGHTINGALE: "Future recipients" excludes someone who might be harmed today by something we discover down the road. If that's the intent, so be it. I'm not sure that's everyone's intent here.

DR. HOOTS: Can I comment? I understand what you're saying, but I think you have to start somewhere. I mean, it becomes infinitely--as long as someone survives--well--

DR. CAPLAN: See, my comment would be, you do have to start somewhere, and the "future" and the "prospective" drop out, and you just set the system, and let them wrestle with when their start date is.

DR. HOOTS: Okay.

DR. CAPLAN: I mean, it's not--I don't think the thing's going to do anything till it exists, till it starts, and starts doing something. It won't do anything before any day.

DR. NIGHTINGALE: Dr. Caplan's logic would appear to be persuasive. Are there any other comments--

DR. PILIAVIN: The word "future" is in there. Do you want me to take that out?

DR. CAPLAN: Yeah. Take out "future."

DR. PILIAVIN: Take out "future."

DR. NIGHTINGALE: Take out "future."

DR. CAPLAN: The start date for this system, I promise, will be when it comes into existence in the future.

DR. NIGHTINGALE: In the future. It is 5 minutes 10:00, and we are 2 hours and 5 minutes behind schedule, and that is an unavoidable consequence of democracy, and I beg the forbearance of the audience. I'm not expecting to get it, but beg it anyway. And ask are there any other suggestions here, because we're going to go to a motion?

DR. PENNER: I'm sorry. But I've got one other concern. I still see we have eliminated "local" out of this, or am I missing something, because I don't want us to recreate the wheel when we already have a system intact as we've spoken about yesterday, and recognize that this is going to build onto the local capabilities for correction as well as reporting, as opposed to creating a whole new system at every hospital.

DR. CAPLAN: Like the opportunity exists to apply those principles globally to transfusion practice?

DR. PENNER: But you already have--

DR. CAPLAN: It's American hegenomy.

DR. PENNER: And before we had local--

DR. BUSCH: How about to enhance principles--"apply those principles to enhance existing local transfusion community activities or existing hospital-based programs?"

DR. PENNER: Yeah, something on this order I think would really carry the fact that one doesn't have to create something from cloth at this point.

DR. CAPLAN: "The opportunity exists to build upon this success and apply these principles", something like that?

Well, if you want, instead of getting hung up on that one, why don't we wordsmith a little bit in the bullpen here and then--because I know what you're fishing for.

DR. NIGHTINGALE: At this point we need to move on.

DR. CAPLAN: Yeah. But I understand what you're asking for, it's to build upon the local--

DR. NIGHTINGALE: The concept that you're articulating, John, is well recognized by the Committee.

DR. PENNER: Okay.

DR. CAPLAN: We'll build some language in there, to build upon local success and then take it nationally. And Jane's got a reminder there to do that for you.

DR. NIGHTINGALE: All right. So do we want to move this?

DR. CAPLAN: Dr. Gilcher?

DR. GILCHER: I have a question. The errors are potentially quantifiable. Those things that we don't recognize are not. And not that I want this in the body of this, but potentially as a footnote. If we taken the 44 to 98,000 deaths that occur in the country because of medical errors, can we quantify the cost of the errors in transfusion medicine, because that may help those organizations that are going to appropriate the monies.

DR. NIGHTINGALE: But not here, not now.

DR. GILCHER: No, I'm just asking the question.

DR. NIGHTINGALE: Down the road, staff will attempt to do so.

DR. GILCHER: All right.

DR. AuBUCHON: Well, that's a nice idea, Ron. My concerns is that actually if we look at the number of deaths out of the 44,000 that are related to transfusions is going to be vanishingly small. Important, regrettable, and in most cases, avoidable, but vanishingly small.

DR. GILCHER: Right, and I agree with that, but there is more than just death. There are other losses that occur, and that's really what I wanted to attempt to quantify.

DR. CAPLAN: All right. I think the Chair is ready to entertain a motion. Do you want to move the adoption of it?

DR. PENNER: Yes, move the adoption of the proposal.

DR. WINKELSTEIN: Could we all just read it? Could we just scroll from top to bottom in way of review?

DR. CAPLAN: The motion is seconded, so we will read the statements. Steve will read the statements.

DR. NIGHTINGALE: With appropriate wordsmithing.

DR. CAPLAN: With appropriate wordsmithing. Just taking on more elements of the Bulgarian Communist Party. You vote it. We'll write it later.

[Laughter.]

DR. NIGHTINGALE: Listen, I am extremely uncomfortable with changing a single word of a document that has been discussed this extensively in public. My own belief is that this document and the transcript that surrounds it, and the summary of this meeting that will be approved by the chairman in accordance with the Federal Advisory Committee Act, will provide more than sufficient guidance to the government officials who will review and potentially act upon this document, and I would strongly request the Advisory Committee's indulgence that we work in the summary of the meeting, rather than in the text of the resolution, to incorporate the suggestions that we were not able to include so far, because to do otherwise would, in my view, violate the Federal Advisory Committee Act, which I am in no mood to do.

DR. CAPLAN: We can do the wordsmithing in the supportive text that accompanies the recommendations.

DR. NIGHTINGALE: Can you cut me that slack? Thank you.

DR. CAPLAN: Read on.

DR. NIGHTINGALE: Okay. "The Advisory Committee on Blood Safety and Availability recommends the establishment and implementation of a national reporting and analysis system for transfusion medicine as a basis for action to reduce and prevent morbidity and mortality due to human and system error.

"The Advisory Committee is favorably impressed with the accomplishments of the error reporting and correction systems that have been developed to improve the safety of air travel by the aviation industry and by the interaction of the Federal Regulatory Agencies with this system.

"The Advisory Committee acknowledges the efforts of the FDA working with the blood and plasma collection industries in reducing errors and accidents, and is favorably impressed with the results to date of the MERS-TM error management system. While a great deal has been accomplished in blood collection and processing, the Committee now believes that the opportunity exists to apply these principles to transfusion practice."

DR. PILIAVIN: Those brackets are for their wordsmithing of the stuff to go with it.

DR. NIGHTINGALE: Yes.

"Error management systems should acknowledge the right of patients to know of any risk or harm suffered as a consequence of any error or accident related to blood products received. At the same time, there should be statutory protection from disclosure for voluntarily reported information and of quality assurance activities that are not associated with potential or actual harm, provided that the information is also not associated with reckless or intentionally harmful acts. These error management systems should complement, and not replace current regulatory activities notably but not exclusively in the area of product safety. All analyses of collected data should be available in a timely manner to regulatory agencies, national transfusion medicine surveillance programs and other participants in a reporting system.

"Congress should appropriate sufficient funds to develop these systems and for an infrastructure sufficient to support and maintain them in the FY 2001 budget. Congress should stipulate that these funds should not be reallocated for other purposes and that no other funding should be reduced because of the availability of these funds. Funds necessary to maintain these systems should be appropriated annually."

Number 2. "There is a small but non-zero risk associated with the use of blood products or plasma derivatives that cannot be eliminated with current technologies. The Advisory Committee therefore supports the prior recommendation of the Institute of Medicine and of others, that a national system to compensate recipients for injuries directly caused by blood products or plasma derivatives and not associated with a reckless or intentionally harmful act should be enacted and funded by the Congress."

A footnote says that, "Error management is to be understood as including reporting, analysis and correction."

It's been moved and seconded.

DR. CAPLAN: Discussion?

DR. DAVEY: Are we voting on 1 and 2?

DR. NIGHTINGALE: We're voting--

DR. CAPLAN: Actually, I think--

DR. NIGHTINGALE: --on the packages, but the packet was proposed.

DR. CAPLAN: You want to do it that way; you want to go 1 and 2? Would you accept a little friendly modification to the motion?

DR. PENNER: What the heck?

DR. CAPLAN: All right. So let's move 1 first. We'll vote that. Discussion on 1? Mary?

DR. CHAMBERLAND: Just a small point. The first--I guess it's the first paragraph recommended the development of a national system of surveillance, and then subsequent paragraphs refer to plural, "these error management systems." And then the next to last paragraph talks about "the data should be reported in a timely manner to the national surveillance programs." And I guess I'm just a little confused, because are we--if we're advocating recommending for a national system of surveillance, should it be singular, and you would not be reporting to yourself?

DR. NIGHTINGALE: I think we're anticipating a variety of outcomes.

DR. PILIAVIN: I think that comes from the fact that we're sometimes talking about this national system, and then we're talking about the local systems that interact with it. And we've never really talked about the local things in this particular thing, but we want to--we want some of the stuff to refer to the whole conglomeration of it. I think that's where the distinction is coming from.

DR. CHAMBERLAND: I agree with your--yeah, with what you said, but we eliminated the word "local." So there's no tie-in to that. So it's just confusing if you read it at face value. You're advocating for a system, and then it suddenly becomes plural in subsequent paragraphs, and then it appears the system is being asked to report to itself.

DR. CAPLAN: I don't know. I guess I don't find it as vague as that, but it's partly because we have that context in the supporting discussion about what we're talking about with local and national. We will get that into the text.

DR. NIGHTINGALE: Into the context. I am trying, as gently as I can, to move you all along. We need to get going here, folks.

DR. CAPLAN: All right. In that spirit, end of discussion.

Moving on, one, vote. All in favor?

[Show of hands.]

DR. CAPLAN: Opposed?

DR. NIGHTINGALE: I see no votes in opposition. It was unanimous.

DR. CAPLAN: All right. Motion on two.

DR. DAVEY: Any opportunity for final comment on two? I feel fairly strongly still that there should be a starting line and that we should, in some way, incorporate in this major, major new initiative that we're looking for prospective situations, for future recipients, otherwise we're not only crossing the rubicon, we're crossing a Mississippi. We're including everything that ever happened, as well as what might happen in the future. So I can't particularly support it as written unless there is at least something along those lines.

DR. CAPLAN: "In the future"?

DR. NIGHTINGALE: Would you be satisfied with "future" back in?

DR. CAPLAN: I mean, if we say, "To compensate, in the future, recipients for injuries"?

DR. PILIAVIN: Why not future recipients?

DR. CAPLAN: Future recipients?

DR. WINKELSTEIN: Future recipients.

MS. LIPTON: I agree about the concept. Do we need to absolutely worry where the word "prospective" or "future" comes in? I mean, is that absolutely?

DR. NIGHTINGALE: No.

MS. LIPTON: Can we just vote on the concept?

DR. NIGHTINGALE: This isn't going to make a great deal of difference.

DR. WINKELSTEIN: It will financially. Financially, you're opening up a Pandora's box.

MS. LIPTON: I just meant, I mean, I think it makes a difference to include prospective or future. I don't care how it gets worded in--

DR. CAPLAN: My only point again is, to see if this addresses your concern, this system is going to get set up, and it's going to have to start to function to decide how to compensate people for injury. It is absolutely beyond any possibility that it is going to be able to compensate backwards based on this statement. You are calling for the creation of something. But they may decide at that point, once they get it, to do that, but that's going to happen, once it's sort of in motion. The principle here is start the system off.

DR. PILIAVIN: Art, I think the point is that nobody that I know on the committee wants it to conceivably be used to--

DR. CAPLAN: To go backwards.

DR. PILIAVIN: To go backwards, and that's why we want the future in there.

DR. NIGHTINGALE: I think that is, Dr. Davey's point. Dr. Davey, if the word "future" is in as it is right now, would it meet with your support?

DR. CAPLAN: Future recipient.

DR. DAVEY: I would agree that somewhere in there, Karen's point, we have to capture that concept. The wording, where it is, we can quibble about. Future recipients sounds fine to me, but I want to capture that concept.

DR. NIGHTINGALE: I think that the specific wording of this resolution will mean much less than its unanimous endorsement by the committee.

DR. CAPLAN: Dana?

DR. KUHN: I believe that the recommendation of the IOM proposed perspective, that when the time did come, that this would be established it would be prospective.

DR. PENNER: And there's no indication of retrospective at this point, which is not mentioned. And I think "prospective" is still the appropriate word.

DR. NIGHTINGALE: Dr. Davey, is prospective--

DR. DAVEY: Yes.

DR. NIGHTINGALE: Dr. Davey would live with prospective.

Dr. Piliavin--

DR. PILIAVIN: Prospective recipients.

DR. NIGHTINGALE: Prospective recipients.

DR. PILIAVIN: It started with a prospective system.

DR. NIGHTINGALE: Prospective national system for recipients?

DR. CAPLAN: Go back to that, the spirit of what we're hunting for.

All right. The Chair will entertain two more comments.

DR. BUSCH: Just I want to respond Jane's comment that nobody on the committee would support reimbursing past recipients. I completely disagree. I would completely support a program, and do, that would reimburse past recipients. But I think what we're looking at is a feasible program here that would implement prospectively.

DR. GUERRA: Two points: One is that the National Vaccine Compensation Act did do it retrospectively, but they did set a time limit for it.

The others that, I don't know that we want to necessarily limit to compensate recipients for injuries. Perhaps we could also add "or death," not necessarily the recipients, but their--

DR. NIGHTINGALE: For injury or death? Injury or death?

DR. CAPLAN: Okay.

DR. PENNER: That's the ultimate injury.

MR. WALSH: I'd just like to go on record as supporting we should compensate--it doesn't need to be prospective--we should compensate people that have been affected, as Dr. Busch mentioned.

DR. CAPLAN: All right. How about we see a vote on, as phrased, adding "death" and putting a "prospective national system" in. All in favor?

[Show of hands.]

DR. CAPLAN: Opposed?

DR. NIGHTINGALE: For the record, no one was opposed to the vote. So the vote was unanimous.

DR. CAPLAN: We had unanimous votes on both one and two.

And at this point, I think what we'll do is just take a 10-minute break.

[Recess.]

DR. CAPLAN: Okay. At this point, we are going to switch over to the always noncontroversial subject of money, and compensation and reimbursement. And I think the way we're going here is Steve is going to say a word or two about where we are with respect to earlier recommendations and responses. Then I think we've got a comment from the FDA coming and then some industry perspective. Then we'll strive for lunch after all of that.

So, Steve, do you want to say a word about where earlier recommendations fell on the fertile soils of the Secretary's Office?

DR. NIGHTINGALE: Good morning, once again.

You will recall, I believe, almost everyone in the room will recall that in August of 1999, the Advisory Committee met to discuss the issue of reimbursement. The discussion was focused on the proposed outpatient prospective payment system that had been proposed by the Health Care Financing Administration, and the Advisory Committee at that time made the following recommendations: This is number three. I am summarizing.

"The Advisory Committee hereby recommends that the Secretary of Health and Human Services exercise her existing statutory authority to exclude plasma-based therapies, their biotechnology analogues and blood therapeutic alternatives from the definition of covered OPD services under the Medicare Hospital Outpatient Department Prospective Payment System.

"The committee further recommends that the Medicare program separately reimburse for these therapies when furnished in a hospital outpatient department, including the emergency room, on a reasonable basis and that the Advisory Committee recommends that the Secretary use her existing authority to exclude therapies under APC 369 from the Prospective Payment System for hospital outpatient services and reimburse them on a reasonable-cost basis.

"In addition, the Advisory Committee recommended that the Secretary work with Congress to seek additional resources to support the introduction and maintenance of mandated blood safety measures."

I can assure both the members of the Advisory Committee and the public at large that these recommendations and the recommendations of the public in other forums were very carefully and very exhaustively reviewed within the Department. I can also assure the members of the public and the members of the committee that while there is never complete agreement on an issue, there was great effort made to achieve a consensus within the Department that would reflect, as best as possible, the stated interests of the parties who had responded to earlier versions of the final rule.

And that was why on February 3rd the Secretary wrote to Dr. Caplan the following paragraph, in regard the your third and fifth recommendations, which were the ones that I just read:

"The President announced on October 19, 1999, several measures to correct unintended consequences of the Balanced Budget Act of '97. These include creation of separate APCs to pay for blood and blood products, of allowance for temporary cost-based APCs for certain new technologies."

I believe these measures are responsive to your concerns and that the Secretary's words there reflect the intent of the Department.

You are also aware that on April 7th, the Health Care Financing Administration published a final rule on our Patient Prospective Payment. It is brief, and it is sufficiently brief that for those of you who have not had the opportunity to see it, I am going to read it into the record and then turn the floor over to Dr. lee.

The HCFA response to the concerns about packaging costs of blood and blood products, HCFA says, and I am quoting: "Many commenters, including the American Red Cross, a major medical association, teaching hospitals and community oncology centers, believe that the payments we propose for blood and blood-related products and for APCs that require the use of blood and blood-related products were too low. Commenters claimed that the proposed payments were so much lower than actual costs, that hospitals might be forced to stop providing a range of blood services, especially those more complex than a simple transfusion. The commenters were concerned that our proposed payment would not allow hospitals to furnish the most clinically appropriate blood products and services.

"The commenters also stated that blood and product exchange were not assigned to appropriate APCs, thus skewing payment rates and not recognizing the true costs of services with which blood and blood product exchanges are associated. Commenters attributed this deficiency to the fact that certain blood-related products were incorrectly billed in the 1996 data we used as the basis for pricing APCs.

"Commenters were also concerned that we excluded procedures whose costs are outside three standard deviations of the mean cost. One major organization recommended that we separate payment for blood and blood products from the service with which it is associated. This commenter also recommended separate payment for infusible blood-related derived drugs and that we base payment for transfusible blood products on cost.

"Some commenters recommended a transition period prior to the full implementation of the proposed PPS.

"Response: Based on the recommendations of commenters, we have created separate APC groups to pay for blood and blood products. We agree with the commenters that blood use varies enough that packaging blood units with their administration could lead to inequities. Because we were not able to capture enough claims data in the base year to accurately price the blood and blood product APCs, we have based payment rates for these APCs on data provided by commenters, including suppliers of blood and blood products.

"We have based payment on current costs rather than 1996 costs, so that we recognize the costs of recently developed blood safety tests. The safety of the nation's blood supply is a major concern of the Department of Health and Human Services, and we want to encourage appropriate testing and follow-up care."

The details cover a large chunk of the April 7th FederalRegister. And in view of the time and limitations of my throat, I will not read them. I believe that they are familiar to most of the members of the Advisory Committee, who have received a copy of this document, and to most of the participants at this meeting.

The purpose of holding this particular meeting then is as follows: The Department committed itself when it adopted the blood access components into the Blood Action Plan on November 24th, I believe it was, of 1999, to attend to the economic concerns of the blood industry, broadly defined. The purpose of this meeting is to listen to the concerns of the blood and plasma industry and of other interested parties in light of the publication of this final rule. This is an open-ended invitation. The goals of the Department were accurately represented in the last sentence that I just read, and we wish to take all appropriate measures to achieve those goals.

One issue that we do feel should be addressed before we take public comment is for the Food and Drug Administration to comment on the issue of universal leukodepletion. This has been a very complex issue on many levels. The Department, and particularly Food and Drug Administration, has struggled mightily to achieve the appropriate outcome. And Dr. Jong Lee will speak on behalf of the Food and Drug Administration. It is my pleasure to welcome Jong.

DR. LEE: Thank you, Dr. Nightingale. Thank you for the opportunity to present the FDA perspective on universal leukoreduction. In the way of a brief review, I would like to just step through four milestones that happened within the last decade.

In march of 1995, a public workshop was held. At that workshop, the major discussion was focused on recognition of a new class of blood products; that is, blood products that are now leukocyte-reduced below a specific threshold of five times ten to the sixth or less residual leukocytes per unit. And the major focus of that discussion was how to regulate this new class of products. So after recognition of this class as a special product, the FDA guidance was generated, which resulted in the 1996 memorandum, which outlined the regulatory perspective on that.

Following that, in 1997, at a Blood Product Advisory Committee meeting, this special product was discussed further in terms of its effectiveness against a special indication. Now, in 1995, the special product was recognized only for its merit in terms of its leukocyte-reduced content. But the way in which that leukocyte-reduced content was to be medically applied was really left as medical discretion and escaped the purview of regulatory jurisdiction at that point.

In 1997, the indication of the leukocyte-reduced products being effective against CMV was discussed, and the committee voted in favor of its effectiveness. However, it voted that it is not necessarily--it may not be concluded that leukocyte reduction by filtration is equivalent to other methods of leukoreduction or even with infiltration that it's equivalent to all uses of different kinds of filters. So this was the first step towards recognizing special indication for leukocyte-reduced products.

And it was anticipated that other indications for using leukocyte-reduced blood products would follow after this discussion; namely, it would be HLA allo immunization, the potential effectiveness against the reduction in the incidence of HLA allo immunization; the effectiveness against the reduction of other infectious agents, and also the major indication that was already recognized, the reduction in febrile nonhemolytic transfusion reduction. That particular transfusion, reduction in febrile reactions was the only indication that was generally recognized and actually written into blood labeling up to this point.

Despite the anticipation that other special indication discussion would follow, that was actually sort of cut short by a new topic that surfaced, and that topic was discussed in 1998 in front of the Blood Products Advisory Committee, where rather than having to discuss each special indication, why not move towards universal leukoreduction in recognition of the fact that the indications for use is growing and that there is really no downside to providing leukocyte-reduced blood. In other words, move from the leukocyte-reduced products as a special class of products, move from that to recognizing leukocyte reduction as blood GMP in that old blood should be leukocyte- reduced.

With a unanimous decision based on this recommendation that all blood cellular blood products, actually, as you say, all platelets, and red blood cells, and whole blood should be leukocyte-reduced. FDA held a public workshop in December of 1999 in which the major point was not whether or not to implement Universal Leukocyte Reduction, but to derive public consensus on how to best implement Universal Leukocyte Reduction. And since then, FDA's current thinking has been in favor of implementing Universal Leukocyte Reduction in the near future.

Having provided as a brief background, I would like to simply just revisit the actual voting process in 1998 BPAC deliberation. The question posed, the committee read: Is the benefit risk ratio associated with leukocyte reduction sufficiently great to justify the universal leukoreduction of all nonleukocyte transfusion blood components, irrespective of the theoretical considerations for transfusion-transmitted Creutzfeldt-Jakob disease?

Now, I pointed out that the discussion of special indications was cut short by a discussion of whether or not everything ought to be leukocyte-reduced. And part of the impetus for that came from the fact that many of the European national blood authorities have implemented Universal Leukocyte Reduction partly in recognition of the fact that this may be affected as a precautionary principle against this theoretical risk of transfusion-transmitted CJD.

In the United States, we elected to separate the two problems in view of the fact that it is difficult to scientifically demonstrate the effectiveness of leukocyte reduction in the CJD setting. Is it possible to move towards Universal Leukocyte Reduction, excluding that consideration, recognizing that there is very little downside and that indications for using leukocyte blood products are growing? And henceforth, the phrasing of the question.

And the committee voted unanimously or at least I would say the overwhelming majority, 13 yes votes to zero no votes, with only three abstentions, that FDA should move forward with ULR, and the answer to this question is yes.

The consumer and industry representatives both concurred with the yes vote, and comments provided in support of the yes vote and of the abstention votes pointed out that this question was answered in the absence of cost considerations, in addition to the lack of attention to its effectiveness in the CJD setting.

The charge of the FDA has been not to consider costs, but FDA recognizes that public health and costs are inseparably intertwined and FDA has, therefore, kept an eye on cost considerations, although we were careful not to directly consider cost considerations.

Having heard a unanimous support from the committee with which FDA agreed, a public workshop was held, as I alluded to earlier, in December of 1999. The objectives of that workshop was, again, not to discuss the merits of ULR, but to discuss how best to implement ULR, Universal Leukocyte Reduction; it was to discuss the U.S. experience available to date, up to that point, on Universal Leukocyte Reduction; it was meant to exchange ideas; it was meant to provide a forum in which ideas on how to best implement Universal Leukocyte Reduction could be discussed. This was really meant for the industry to guide industry, not necessarily only to speak to the FDA. But also it was a major forum in which the industry and the public could provide comments to the FDA for future consideration in generating some sort of a formal statement.

The outcome of that workshop was that the participants clearly agreed that the full implementation of Universal Leukocyte Reduction within 2 years of FDA's statement is a reasonable time frame. Anything longer than that was considered inappropriately long; yet anything shorter than that, was considered as a potential risk in terms of careful and seamless transition process to a new GMP standard.

It was stated and generally agreed that each center was best able to design its own specific implementation plan within an overall general framework established by the FDA and that the FDA take this opportunity to update the current recommendations on QC testing, as outlined in the currently effective 1996 memorandum on leukocyte reduction.

And it was also pointed out that this would be a nice opportunity for FDA to institute a pilot program in which the licensure of leukocyte-reduced products could be streamlined and expedited based on self-certification to a set of standards that FDA would establish.

So having clearly heard the message from the BPAC, and also having clearly heard the message from the Universal Leukocyte Reduction Workshop, the FDA's current thinking on the topic has not changed and has remained solidly in favor of implementing Universal Leukocyte Reduction.

Now, the key point here is that this position is based on the fact that the burden of proof, with respect to the growing list of indications, given that the use of leukocyte blood products is increasing, and given that the adverse effects of leukocyte reduction, limited to the process itself per se, there are no adverse effects associated with leukocyte reduction per se, although there may be some associated with the ways in which the devices that may be used in achieving that goal, given that basis, the burden of proof whether or not leukocyte-reduced blood products are to be used, really switched from having to demonstrate its effectiveness in certain settings to actually now demonstrating its ineffectiveness in certain clinical settings. And there are actually small groups at this present time who are trying to establish just that. Its ineffectiveness in major clinical settings, however, it's difficult to say how successful that will be.

The general public consensus remains that leukocyte reduction is effective in all clinical situations, although the list of FDA-approved specific indications remain small at this point. And that default mode of decision also recognizes that there is a clear strong international trend towards universal leukocyte reduction as a blood GMP; that at least nine national blood authorities have favored Universal Leukocyte Reduction, and the list is growing.

Then why the delay of 2 years to full implementation, as outlined in the previous-mentioned workshop? Well, this 2 years was arrived at, at first, in recognition of the fact that the filter supply may not be available to meet the demands if overnight implementation was instituted. However, 2 years was felt to be more than sufficient to allow this to happen and also allow the center-specific implementation plan to be developed.

More importantly than these two issues is the fact that implementation of leukocyte reduction does cost public health dollars, and that although the charge to the FDA is not to consider cost concerns, that we recognize that the cost concerns and public health are inseparably intertwined and that there ought to be some time for reimbursement concerns to be worked out with the Health Care Financing Administration.

And it was hoped that the 2 years would allow adverse impacts on blood availability and health care delivery, the potential adverse impact of implementing ULR will be worked out in that time.

So given that basis of selecting this time frame, the task remains before the FDA as to how to make this formal statement. And initial thinking was to use the guidance mechanism under good guidance practice to make a public statement. However, it's been argued that the correct mechanism is actually the formal rulemaking process. And that's an issue that the Agency will have to settle on how to proceed forward.

So in summary, the FDA remains fully supportive of ULR, as it always has been, as evolving blood GMP; it agrees with the workshop outcome of the 1999 December workshop in how to implement Universal Leukocyte Reduction, selecting 2 years as the time frame to full implementation, allowing blood center-specific implementation plan under FDA oversight; and I might at this point take just one sentence to point out that we are trying to take advantage of this situation to update the QC requirements that are perceived as being deficient in the current memorandum on leukocyte reduction and to use the opportunity to promote the self-certification licensing mechanism for leukocyte-reduced blood products.

So in terms of making a formal statement about this, we have clearly articulated our current thinking. In the future, in terms of formally capturing this current thinking, it's a little bit unclear as to whether we will proceed with a guidance mechanism under GGP or under formal rulemaking process, but that is to be worked out and not necessarily be an obstacle to effective and expedient implementation.

The timing of this depends on several concerns, as I mentioned earlier--most notably with the coordination with the Health Care Financing Administration. The coordination has really been provided by this committee as the focal point, and I think sufficient time has passed and the fact that the new codes for reimbursement under the ambulatory care setting has come out. And although some questions remain as to concurrent, analogous reimbursement system, it is absent for the inpatient setting.

The time is probably ripe to move forward, that cost considerations have received enough indirect attention from the FDA and that we are now ready to move forward with a formal public statement. That may or may not be tempered by future Advisory Committee deliberations that are forthcoming in this committee and also in the upcoming TSE Advisory Committee.

With that, I will close.

DR. CAPLAN: Thank you. Comments?

DR. AuBUCHON: Mr. Chairman, I am amazed that this approach has been taken on this issue. In the past, the FDA has always required that a new product be shown to be safe and efficacious before being licensed. Apparently, the playing field has now been change,d and we need to show that a product is safe and that there is no data to show the lack of nonefficacy or some double negative that, frankly, does not make sense to me.

I think the status of the situation is nicely summed up in a letter that I recently received from the senior vice president for Biomedical Services of the American Red Cross, one of the major proponents of Universal Leukocyte Reduction, where she notes that it may be that the scientific debate about universal prestorage leukoreduction has been superseded by the time frame of implementation.

In contrast to Dr. Lee's statement that there is broad public acceptance of the value of leukocyte reduction, I contend that there is still a large scientific debate about the efficacy of Universal Leukocyte Reduction. There are clearly some individuals who will benefit from leukocyte reduction, but we do not know whether the practice of Universal Leukocyte Reduction will really reap all of the benefits that it may provide.

There are currently studies underway which will help address that issue. There are huge economic issues attached to that. Because the price tag for Universal Leukocyte Reduction in this country is approximately half a billion--yes, with a "B"--dollars a year. The filter manufacturers are grinning broadly at this possibility, seeing their sales expand markedly overnight. I have no objection to them selling filters and making money, providing that what we are doing for our patients will really be beneficial.

When the Blood Products Advisory Committee looked at this issue, they indeed looked at it absent issues of cost. I would think it would have been appropriated for the FDA to bring the entire issue to this committee, since cost is within our purview, and we have to make many decisions based on the availability of resources.

Universal Leukocyte Reduction may well provide additional benefits, but I can list at least ten other things that we could be doing today in transfusion medicine that would also provide benefit. We could provide all of our red cells as frozen deglyceralized because the red cells would last longer and patients would need fewer transfusions possibly. We should do quality control on every leukocyte-reduced unit to make sure that the filter really did what it was supposed to do. Perhaps we should do all infectious disease testing twice on each unit to make sure that no random analytic error caused an infectious unit to creep through the system.

So there are many things that we could propose that would potentially provide some additional benefit. I think it is interesting that the FDA is allowing the manufacturers to push this through without having the scientific debate reach a conclusion that really allows us to say whether or not the expenditure is worth it. I find that amazing.

DR. CAPLAN: Jay?

DR. EPSTEIN: Well, I certainly recognize, and I think anyone who has looked at the literature recognizes that there is an ongoing scientific debate. But that's precisely why the question was brought to a scientific advisory committee in September '98. Now, Dr. Lee has explained that the outcome, absent consideration of cost, was essentially a unanimous view that on the whole the benefits of Universal Leukoreduction merited its implementation, although it was clearly stated that in the opinion of many of those voting, no single purported benefit was sufficient in its own right to justify that measure.

Now, I think that what has happened is that the FDA is fully aware that this is a major step economically for the industry and that the costs have to be absorbed somehow and in the real world. And I guess that the issue that we are really bringing to this committee is the question whether the reimbursement problem is now solved. And I think it would add some clarity if we were able to separate the scientific arguments from the issues of reimbursement.

I cannot figure out, reading the various letters that have come to the Agency, to what extent the opinions are governed by the fear that there will be no way to pay for this and, therefore, it could have drastic impacts on blood centers versus the scientific opinions, whether were the costs covered it should be implemented. And I think that's the problem, and that's why we're here to talk about reimbursement. The issue of how to pay for blood safety and quality innovations has been in front of us for some time. It would seem that the system has been able to absorb the costs of investigational NAT. Leukoreduction is more costly and has been daunting. But I think that we have to try to separate out these parts of the debate.

I would go further and say that we, generally speaking, need to separate out the rationale which is scientific, from the rationale which is purely political, from the rationale which is more directly economic and that we do ourselves and our public health community a disservice when these issues get too intertwined. Because what happens is that everyone presents the opinion couched in the currency of good science, and yet we are really talking about other things. We are either talking about social points of view, policy positions and/or economics.

So what the FDA tried to do was separate it. Our charge is not the cost; it's the safety and efficacy, as you correctly state. That's why we brought the issue solely in terms of safety and efficacy to the Advisory Committee. The opinion, which can be debated, was nonetheless unanimous in favor, on strictly scientific grounds. And what we are trying to do is get a clear pathway toward practicality. We see the cost issue as an implementation issue. We have been of the position that it couldn't be implemented yet. But we can't entangle that with the issue over whether it should be done.

I think that, just to give you an analogy the way we look at the question of GMP and product quality, we see it somewhat analogous to, say, taking human host cell DNA out of a vaccine. There's little demonstration of harm, there's a broad consensus that you don't want it there, and over time the standard for residual DNA keeps being lowered, and everybody thinks that's good.

Well, where we stand with leukocytes is we know they can cause some harm. There's no evidence that for a nonleukocyte product they are of any benefit to the patient, and so we see it as a purity issue; you know, safety, purity, potency. That's our charge. And so we, therefore, see this as an advancement in product quality related to purity. And that's the way the scientific issue is framed in our minds.

So I understand the points that you've made, that we're not dealing with a well-established clinical impact outside of certain defined recipients at particular risk. I understand that. On the other hand, I think that we have vetted that issue in a scientific forum, and there are recognized definite and theoretical benefits, and there's the underlying concept that we are making a purer product. We are, in essence, removing an adventitious contaminant.

So that's how I would respond, Jim. And I say that fully respecting the individuals who have been vocal recently in the literature against leukoreduction, universal leukoreduction. But I think that our job here is to try to look at the cost issue because this is really the only forum where we can. And I would hope that we could then look at the cost issue, see if we've made sufficient progress and try to disengage that from the scientific issue.

DR. CAPLAN: Steve?

DR. NIGHTINGALE: Steve gets to make the next comment.

I want to make it even clearer than I hope it already is that the Department of Health and Human Services has and continues to very strongly and unequivocally support the position that Dr. Epstein just articulated. This is superfluous, but to support Dr. Epstein's efforts in this regard. The Department understands fully the complexity of the scientific debate. The Department's concern include, though are not limited to, separation of a debate on the scientific merits of a public health policy from the economic consequences of that policy. And the Department's prior silence, if, in fact, that was, reflects not a wavering in that commitment, but in the difficulty that the Department has had internally struggling with the very issues that Dr. AuBuchon raised.

The issue, for example, of whether or not to separate blood and blood products from prospective payment in the outpatient area was very, very vigorously debated within the Department. We believe that we have made progress towards resolving the issues that Dr. AuBuchon and others have raised. We do not kid ourselves that we have completed that task. And that is why we are having this discussion here today. We recognize that we have further to go. A major concern that we have is that in the area of economics, there are very strong opinions and there is very little data to back up many of those strong opinions.

We right now are embarked on an experiment to see what will happen to the price of blood and blood products now that we have dissociated them, even in a limited economic domain, from prospective payment. We will have data in 6 months that we do not have on the market or even spot price of blood and blood products that will be most certainly used in future deliberations by the next administration on how to deal with other areas of blood reimbursement.

That is why we have invited you here to give us your guidance. And that is not a cynical term. That's exactly the term that we're looking for, on how to proceed in this transitional time. We want to do a better job, and we solicit your support. And while we solicit yours, we express our very strong support for Dr. Epstein and his agency.

DR. GILCHER: As the director of a moderately large blood center that actually collects 1 percent of this nation's blood supply, approximately 135,000 to 145,000 units of red cells, I can tell you very clearly what the costs are. The cost of a nonleukocyte-reduced red cell has been vastly underpriced and has driven many blood centers into practically bankruptcy. If we take the differential cost, and I can give you the numbers, the real cost to my blood center to leukocyte reduce a unit today, based on the fact that the cost of filters, the technology is decreasing, is approximately $20 a unit. That is our cost.

Therefore, using 14 million as the maximum number, and it's actually less than that, of units of red cells collected, we're really looking at a total cost to the system of approximately $280- to $300 million. So I take issue with the half-a-billion-dollar number.

One of the worst situations for a blood center to be in, and this is where costs are driven up, is when you reach the midway point. And our blood center has reached that point. At 50 percent, leukocyte-reduced and 50 percent nonleukocyte-reduced, our costs are clearly driven up. Our communities have made the decision, based upon medical input and endorsement by our count medical society, to go 100 percent universal leukoreduction as of July 1st.

Now, I want to point out that platelets already in this country collected by apheresis technology are already leukocyte-reduced. The technology is in place virtually for 100 percent of those products.

In addition, when you compare bedside against prestorage leukocyte reduction, there is no quality control, there is no process control at the bedside. And the product that is administered, and there are studies to support this, is clearly inferior. Whereas, when this is done as a prestorage process within the blood center, where there is process control, where there is then quality control of the product, we can really assure the quality of that product.

Those are my comments.

DR. CAPLAN: Karen?

MS. LIPTON: I just wanted to make a comment. I, too, think that the issue we need to address here is the cost. But just a couple of concerns. We heard from Ron about the blood center concerns. But I really want to emphasize that the hospitals are really suffering from this. And appreciating that we have had some relief on the outpatient side, that is only about 10 to 15 percent of a true cost for a hospital.

I don't think we can wait 6 months to see how this goes. I think that in 6 months we are going to see some real problems in transfusion services and safety that affects what I call the administration and process rather than product. We have put a lot of money into making the product as safe as possible. We need to make sure that we don't start cutting money out of the system so that we are giving the wrong unit to the wrong patient, we are making other compromises to give us a perfectly safe unit that does absolutely no good. And I really do believe that on the hospital side, we are there. The way DRGs and Prospective Payment System is structured, transfusion services and blood banks are a cost center. As the cost of blood goes up, they don't get more money. They are just told to take it out in some other forum, and I think it is really important for this committee today to come out with a statement that really says we need a change to this. I understand that that cannot happen at the department level because there are congressional limitations under the Balanced Budget Act.

But I think that we need a strong statement from this committee to take to Capitol Hill, to talk about the urgency of this situation and to put us in a position where we are promoting blood safety and transfusion safety and not saying we are with one hand and taking away with the other.

DR. CAPLAN: Keith?

DR. HOOTS: Yes. I guess I want a little clarification perhaps, in terms of there was originally on our agenda, I guess, what, 18 months ago, there was to be an intensive look at this, and then we got supervened by a relook at the Hep C. So we really haven't had an opportunity to look at all, well, not even all of the data, much, very little data except what each one of us brings to the table.

From my own personal experience, I actually have had the opportunity to work in two separate institutions; one who has been universally depleting for about 10 years and one who is kind of phasing it in slowly. And I am not sure that even that amount of dual experience is enough data for me to make a real cost efficacy determination, although I must confess I do have a little bit of bias that is weighted kind of towards the BPAC direction, just because I have a bias towards what those filters have the capacity of doing. But I don't have enough data I think to say whether that translates into a true cost efficacy number that we can live with.

And as much as I agree with Karen that I don't want blood banks to be in the lurch out there, I just wonder if this is a--if making a blanket statement about reimbursement, if we will really have fulfilled our full fiduciary responsibility without gathering some more data and hearing a lot more dialogue than we've already had.

DR. CAPLAN: Dr. Davey?

DR. DAVEY: Yes. I fully appreciate the comments of Jim, and Karen and others, also. And if we do make statements on this matter, I would suggest we keep the reimbursement statement separate from perhaps what I would recommend as an encouragement to move forward with some kind of regulatory guidance in this matter. Right now there is a fair amount of confusion, I guess would be the word, in the profession and the industry about what is the framework for moving forward with its initiative and issues of politics, and cost and science are kind of tangled up. But the BPAC did make a fairly clear statement, and the FDA I think has, through John, has made their position clear also.

So I would like to propose that the committee make a recommendation that the FDA really act expeditiously to issue some kind of a regulatory statement regarding universal leukocyte reduction probably in accordance with the recommendations of--not probably--but in accordance with the recommendations of BPAC so that there can be some guidance for us in the field on how to move forward.

DR. CAPLAN: Mike?

DR. BUSCH: Yeah, I am really torn on this issue because I have been very involved in a number of the studies that are trying to document whether or not this approach is efficacious. And it is very difficult to do, and internationally it's been very difficult to get a consensus position scientifically.

My biggest concern, though, is sort of what Jim started out with, which I feel is that the role of this committee I think has been bypassed for the second time. I think we were completely bypassed on the issue of British donor referral. It was brought to us as a done deal, and I think that is what's happened here. This has been moved forward, and in essence almost regulated as a done deal in the absence of this committee taking its responsibility to bring cost and overall issues into the decision. And that's my biggest concern. I think we need to take this on as a significant full committee meeting agenda item and really look at the overall issues.

We are talking about $3- to $400 million a year, compared to what we talked about earlier of maybe $2 million a year to implement a complete error management program. And will hear later about international blood safety issues, where countries aren't even screening for HIV and HCV. And that's the role of this committee is to bring that balanced, cost-effectiveness perspective to these decisions, and I think it's really wrong that this program has moved forward, independent of this committee taking on its responsibility.

DR. CAPLAN: Let's do Paul and then Jay.

DR. HAAS: I can't comment on the quality of leukoreduction. That's not what I know. But if you listen especially to what Karen has said and has been said around the room, the very fundamental economic concept of opportunity cost is real. Cost-benefit analyses are important, but they are hard to capture.

And, again, Karen's comment I think is incredibly important. We can see a benefit in one area that if we are not seeing the cost, it means that the hospitals or whatever aren't doing other things, the very things we've spent a day-and-a-half on here, then that opportunity cost or that cost-effectiveness analysis or quality, whatever we want to use to get there, is something that does need to be addressed. And I think I agree with Mike that this is something that shouldn't be decided in a half an hour, but some serious data, if we can get it.

DR. CAPLAN: Jay?

DR. EPSTEIN: Well, just to comment that, you know, the functions of the different committees have been crafted to be somewhat complimentary. And we have generally taken what we thought were purely scientific issues to the FDA Scientific Advisory Committees. Where we think that there are global public health issues and/or societal choices and/or cost issues or ethical issues, we then have taken them to this committee. And I would just contend that we have, in fact, not just gone forward. I mean, it's been almost 2 years, and we have not issued a recommendation, and that's very unusual behavior for the FDA.

So i think that we have taken the time or committed the time to try to resolve to let the system, in the large, not just the FDA, but the larger system, deal with the cost side and the cost-effectiveness side of the equation. You are correct that we didn't come to this committee asking the committee to solve the problem, but that's because we already had recommendations made a year-and-a-half ago to address mechanisms to find funding for blood safety advancement.

So what's been going on is that the Department has been trying to accomplish that. And really what the committee is being asked today is have we gotten close enough to that end to proceed, not just on this issue, but potentially on other issues. So I don't think it's quite fair to say that we've simply left this committee out of it. In fact, what we have been doing within the Department and the agencies has been trying to follow the proscription that this committee has articulated at previous meetings.

DR. CAPLAN: Just to put my two cents into it, I would say that we've tried to argue that compensation should be more reflective of safety. That's really what the drive has been from the Advisory Committee. Leukocyte reduction is one of the ten things that we'll challenge Jim to do at lunch to make blood safer.

But the list is there, and the compensation policy decision, in some sense, has opened it up. I don't think, in my view, we've ever had a discussion back and forth about the evidence pertaining to leukocyte reduction per se, but we have had a discussion about compensation and safety, NAT testing, whatever it's going to be. So, in that sense, what we've done is urged HCFA or funders to make more room for safety. Leukocyte is in the middle of that.

DR. BUSCH: I understand all of that. My concern is that somebody needs to weigh the relative merits. I mean, we're talking about a minimum of $20 per unit. We struggled over a dollar unit to add an enhanced antibody test, and we're trying to find the system to pay for $5 to eliminate these infectious residual risks. And here we're talking about an investment of $20 per unit; you know, orders of magnitude greater than we've been looking at in the last 10 or 15 years. And there's nobody making the weighing assessment.

DR. CAPLAN: Steve?

DR. NIGHTINGALE: I would simply add, at this point, that the decision not to take leukodepletion here is, first of all, one that has been extensively and continuously discussed in the Department. Those of you who participate in our monthly blood conference call have had several such discussions. The decision has not been made never to take it to the Advisory Committee; the decision has been what time, if any, it might be most useful to do so, recognizing that this Advisory Committee is part of a very complex decision-making process within the Department that ends up, as a consequence of the Institute of Medicine report and Secretary Shalala's adoption of it, before the Shays Committee on October 12 of 1995, straight to the top.

There is, I can assure you, no absence of senior oversight for blood policy in the current administration, nor will there be. So the one place I would respond to Dr. Busch is that the lack of a formal committee meeting on this is not intended, first of all, as a slight or denigration on this particular committee, but I would ask you to realize that there are a lot of different decision-making processes that go on.

The specific policy, however, that I would like to reemphasize at this point is we, the Department, nobody within the Department sees this committee as a scientific review body for the Blood Products Advisory Committee. That is not this committee's role. And the idea at one point--I mean, Jay and I were really going back and forth on this issue, one of the arguments that I believe prevailed was one that I did not want this committee to be seen in that light, as sort of a counsel, such as they have at the NIH. This is not the equivalent of counsel, and if it tries to do so, it will fail.

DR. CAPLAN: John?

DR. PENNER: About 12 years ago or maybe it's 15 we decided the problem was sufficient that we would like to leuko-reduce all of our blood products at our regional Red Cross center by centrifugation, alone. And of course this was not as efficient as using the filter. As the filters came through, we decided, well, we would use these, employ them, recognizing that our major problem that we were having in the hospitals was the leukoagglutinin; in other words, white cell sensitization, and that any reactions that we were seeing in our patients was not due to a mismatch on the red cells, it was due to the leukoagglutinins that were producing fevers or stopping our use of the blood product, and then following that, a subsequent workup, which was costly, and then having to revert again to some removal of the white cells.

So there is a cost in the hospital for any of these reactions which do occur not uncommonly because we have more and more patients who receive blood transfusions on many occasions. So our approach then was very simply to any patient who had received previous blood transfusions would then get the leukodepleted program.

The unfortunate thing, to get this information across to the staff, the medical staff, was obviously inadequate, and we continued to have this sort of thing because it is hard to at least reinforce the situation to our physicians that they should ask the patient if they've had previous blood transfusions. And then if they have, say we will use leuko-poor blood. And I think this is the whole contention, that there is no disagreement that this would be a safer, with respect to these reactions, approach. And in some cases leukoagglutinins are severe enough to produce a pulmonary problem where white cells are lodged in the pulmonary bed and caused severe morbidity, if not, on occasion, mortality.

So my feeling from the standpoint of my patients is I want leuko-free or leuko-poor blood, and I order it for every patient that receives it, so I don't have any conflict. So that it comes around to the same fact as cost factors that have to be implemented as part of this, and it would seem to me that cost is a significant aspect and should be handled with recommendations of the agencies that are providing the funding, that costs will have to be procured or managed appropriately for this safety issue to be employed.

COL. FITZPATRICK: Since we are addressing costs, I feel the need to express DOD's problem in this area. We are mandated to maintain the same standard of care as our civilian agencies, and we are doing that and we have been successful this month in implementing NAT, several months behind other people. Part of that is a cost issue. The health care dollars for DOD are fixed, and I was successful in lobbying and obtaining $1.7 million from that fixed budget to implement NAT. That is going to be at the decrement of some other aspect of health care within the Department of Defense.

If ULR becomes the standard of care within two years from some date, DOD will be mandated to respond and continue to do that. That will also be at the decrement of some other form of health care within the Department of Defense because of a fixed budget.

So I would ask that this committee consider that in their recommendations, and if language can be crafted to help offset that in their recommendations for us, I would appreciate that. That's my DOD statement.

Now, on the other side of the coin, I would support the fact that I think it is--a cost efficacy analysis in an area where there is questionable safety and potency effect is very worthwhile.

DR. CAPLAN: Karen?

MS. LIPTON: And I think, you know, that might be an appropriate debate. My concern is if we go away from here and we make no statement about reimbursement, we talk about a 300--let's say it's $300 million. But a lot of those costs have already been incurred. If Ron is leuko-depleting already and it's $20, either that's already been passed on to your hospital--so it's not that we're standing on the edge of a precipice waiting to jump off. A lot of people have already jumped, and that downstream is hitting the hospitals as we speak.

So my concern is we can, you know, save the debate and have a debate, but this country is already going in that direction, and there are already real costs that we need to contend with out there. That's my concern.

DR. GILCHER: There are really two issues here, Karen. One is cost and one is reimbursement. And they need to be looked at, I believe, separately.

On the cost side, even though there's an increased cost to leukoreduction, there are decreased costs--I can speak widely on this because I'm so intimately involved. For example, the removal of micro-aggregate filters at many of the hospitals where they were universally used is going to practically negate the cost of leukoreduction. The consensus among our neonatologists and among our oncologists to do away with CMV testing and now accept leukoreduction is going to take another cost out.

I think that has to be part of the cost modeling, is what can we take out that we're currently--that are currently costs to the hospital. Reimbursement is a separate issue.

DR. CAPLAN: Well, why don't we do maybe two more comments on this? Then I'm going to ask Steve to be thinking what the Secretary would like advice on with respect to leukocyte reduction, because what I hear so far is the FDA, from what I gathered from the presentation, is moving toward a two-year slow implementation path. I also hear that a lot of places are leukocyte reducing and incurring costs that concern them. And then the question becomes: Does the committee want to venture anything today about those realities?

So why don't we do four more? Jane, then Paul.

DR. PILIAVIN: Notwithstanding the fact that the FDA committee decision overtly was not involved in the CJD issue or the nvCJD issue, I would like to have some statement put in here that if this total leukoreduction goes in, that this committee and the BPAC reconsider immediately the deferral issue of donors who have been in the U.K.

DR. McCURDY: I'd like to make one addition to what Ron said that's not only a cost and a reimbursement, but something very closely related to reimbursement, and that's price. And price and cost are not necessarily the same.

DR. CAPLAN: Rick and then Jim.

DR. DAVEY: Just perhaps to reiterate an earlier comment, I think that it would help to give us in the profession and industry a timetable, a framework, would be to have the FDA issue guidance expeditiously. That would allow hospitals to say, okay, within two years or three years this is something we have to work into our budgets. It gives us a target. It gives us some structure. And I think the committee should seriously consider making a formal recommendation to the agency to really expeditiously move a statement forward.

DR. AuBUCHON: I recognize that this committee is not a congressional oversight committee looking at how the department or the FDA makes decisions. And I would expect that if this committee did spend time to look at universal leukocyte reduction and try to pull together all the different aspects of it, we would not be able to reach a consensus on the scientific benefits. It's very complex. And many of us, as Mike said, are conflicted as to what it may really offer. However, we are being pushed by the manufacturers of blood products, by the FDA through back-door routes, to make this happen.

The real impediment is cost. There do not appear to be too many downsides, although there are some real downsides. There are some reactions that we are seeing in some recipients of leukocyte-reduced components. We also know that leukocyte reduction removes about 15 percent of the red cells from each unit. At a time when we're looking at a constricted supply, that 15 percent may make a difference for multiply transfused patients.

But the most glaring problem that faces us is indeed cost. We will have to pay for it, and the situation in the hospital is very akin to the DOD situation. Once we go from leukocyte-reducing about 30 percent of our red cells, which is what we do now at our institution for those patients who we are quite certain will benefit from it, to 100 percent leukocyte reduction, we will have to find $220,000 in a fixed budget in order to pay for it based on what the Red Cross is charging us for pre-storage leukocyte reduction.

Where is that going to come from? It is going to come from some other program that actually may provide equivalent or greater health benefit for the community.

Therefore, I think it is entirely within the purview of this committee to make some overall assessments as to whether or not it's worth it. However, I do certainly agree with Karen Lipton that we need to do something now for hospitals who either have made the decision or who are being forced by their blood supplier to convert to universal leukocyte reduction and are incurring huge increases in costs.

DR. CAPLAN: Keith, one agonizing comments, and then, Steven, I'll--

DR. HOOTS: No, it's kind of a question I want to ask that arose from one of the bullet points of Dr. Lee's presentation, which had to do with the timing of the FDA recommendation two years down the line as it relates to availability of adequate supply of filters.

I think one of the pieces of information we ought to at least have access to is where do we stand on that curve and what is a potential shortage of filters going to do to the price of filters if it becomes universally mandated instantaneously at X period of time if there's not an adequate supply. And, obviously, they've thought about that or else they wouldn't have delayed it for two years. But I think the impact of the timing may be really critical to how we approach this.

DR. CAPLAN: Steve, do you want to take a stab at my question?

DR. NIGHTINGALE: Yes, I sure do. Before I take a stab at yours, I will try to take a stab at Dr. Piliavin's comment as well, because I think it requires a response.

The department has made a commitment to periodic review of the British donor referral policy, just as it has implicitly made a commitment to review of all blood or, for that matter, health policies that have complex interactions within the health care system. There is a meeting of the TSE Advisory Committee scheduled for June 1 and 2. There is regular review within FDA that is communicated to the highest level of the department, certainly including the Blood Safety Director, Dr. Satcher.

We continue to monitor the situation closely and hope that new scientific evidence will guide amelioration of the policy. Our past statements hold on that issue. I think Jane's question is an important one and deserved an answer.

DR. PILIAVIN: You do understand the connection. I mean, if this is supposed to be in Europe a fix for their problems, then why isn't it a fix for ours? And this business about it's going to take out 20 percent of the red cells just makes it more urgent that we have more donors.

DR. NIGHTINGALE: Okay. I think I'd probably not, in the interest of time, want to pursue that aspect, although Jane and I will have a private conversation on it.

I think that towards the larger issue of leukoreduction and the health care benefit fact, the total health care budget for the United States Government is fixed by Congress. It is not decided by the Department of Health and Human Services. The way the business works is that the department has a certain latitude, limited by many things, including the Office of Management and Budget, on how it spends the money appropriated by Congress.

What you have seen with the outpatient prospective payment is a decision that was made by the department to separate the costs of blood and blood products from prospective payment in the outpatient arena. As a practical matter, we don't think we've got current law to do that for the inpatient. We don't think that we've got broad consensus to do it for the inpatient. But that's why we're having the meeting here, is to see where that consensus might be.

Were we to say to the hospitals you're going to have to pay so much for a unit of blood so you're not--you can't fire that sixth technician here, you're going to have to fire a technician somewhere else, or fire the flower girl, would be a step in a certain direction that might be right and might be wrong. There is a sentiment on both sides of the aisle of the political parties that the less micromanagement that we do of our country, the better off we are. There's also an understanding that we have to do some micromanagement sometimes to ensure an equitable society.

We are actively listening, but we want to listen to the full spectrum of concerns. I understand Ms. Lipton's concern. I suspect that there will be others. We want to hear them all. And when we hear them, I would ask you and beg of you, make them as brief and to the point as you possibly can.

DR. CAPLAN: I'm going to offer two comments and maybe we'll get some further clarity about what we might or might not want to say about going into the public comment, further public comment beyond the FDA. But this may put the Chair in somewhat opposition to the staff on this one, but it does seem to me important and what we're being asked for is further information by at least some members of the committee to look at the cost/benefit on leukocyte down the road. That does seem to me to be something we have to take seriously in terms of since we've been presented with the issue, responsibly we have to in some ways monitor the issue.

So I would like to see that one come back if the committee agrees, at least for maybe some more inspection at the next meeting. I'm looking for some nods here. Okay. That doesn't mean we have to settle it, but we've got to monitor it. But no one in a way asked us. I was listening to what Mike said about the British blood deferral issue, and I always understood that sometimes decisions are going to move faster than the committee can move, and sometimes not every decision will get reviewed or vetted. I will betray my view known to most of you that I'm not fond of the decision that was made about the British blood thing, but, okay, so be it.

On the other hand, it is one of those areas where we have a monitoring responsibility since, in a way, it's been raised to us. So my comment, before we go into the public comment period here, is that I think it would be useful if we could organize many of our recommendations across the board and then perhaps reflect upon which ones we need to revisit in terms of just updating.

I do appreciate that the agency is in constant motion to evaluate every decision it makes around the clock, as Steve said. However, it would be our responsibility to take a look-see, too, and push to make sure that happens. So if we want to keep an eye on the British blood deferral thing, with all respect to the staff, we want to know. So some of that is just organizing our recommendations around a framework that lets us see, we said this, we said this, we said this, where are we, where are we, where are we. That's been something that I've been urging that we get done both on the website and just on paper. We're far enough down that road that we got to do it.

Leukocyte reduction and British blood donor deferral are both there, probably deserving of a report, and I'll come back to my other favorite, it's our old lookback thing. We probably have to keep pounding the lookback issue just to see where is it, what's happening, how fast, with what speed and so on.

So the bottom line here is I want to be able, if I can get more nods out of the community, to get us--have a framework on the Web and on paper that will let us do that. Okay.

Then why don't we go to the public comment. Can I get a little show of hands about how many people wish to comment at this point? A lot. Well, the Chair will tolerate three minutes of public comment. If you have written statements, you don't have to read them because we will read them. But let's see if we can have some dialogue.

Let's go for people with the last name from A to D. Who's got that?

DR. NIGHTINGALE: Art?

DR. CAPLAN: Do you have a list?

DR. NIGHTINGALE: I don't have a list, but I realize that some of the public commenters have planes to catch. We're going to ask for truly participatory democracy out here and ask that the commenters present themselves along--

DR. CAPLAN: All right. Come up to the podium with your airline ticket.

DR. NIGHTINGALE: Yes. Those who need to make a plane, if you would present yourself to the committee first--

[Laughter.]

DR. NIGHTINGALE: Dr. Marengo-Rowe, would you identify yourself fully into the microphone, please?

DR. MARENGO-ROWE: Thank you. I am Alan Marengo-Rowe. I am director of special hematology and transfusion medicine at Baylor Medical Center. And I am simple hematologist, so I have to use slides. If I could just have the first slide, I'd like to tell you what's happened in our institution.

That's fine. Thank you very much, indeed. Thank you.

In the interest of time, I'm going to omit the first two slides and just show you where I work. This is Baylor Medical Center in Dallas up here, and this is Dallas. Sometimes our administrators get it confused. But we are a 1,000-bed institutions. We have 43 operating rooms. We deliver 4,000 people a year, 40,000 admissions. In short, we use an awful lot of blood--to be precise, exactly 1,000 units a week in order to keep us running.

As you can see, since 1994 going up to last year, there has been an increasing demand for blood, and there's been a 13 percent increase in the demand for blood in the last year.

If you look at our cost structure, this is where my administrators complain to me intensely about it. In the last year, we've had a 25 percent increase in cost structure. The increased costs can be accounted for by NAT testing for HIV and HCV, the fact that we are not self-sufficient in blood components--as in many metropolitan areas, we have to import about 20 percent of our blood supply--lookback and universal leukoreduction. We're currently running at about 50 percent, just under 50 percent.

And if you look at our inflation assumptions in comparison to the overall inflation, you can pick up two particular areas, blood and drugs, are in the region of somewhere between 15 and 16 percent. Those are the major costings that my administrators, our administrators on behalf of the American Hospital Association have asked me to point out to you.

We can shut the slides off and put the lights on again, please.

So, consequently, the rising cost of blood, as has been mentioned by many members of this committee, is a critical problem for hospitals. And it's a major factor, and we are--the AHA is working both in the long and the short term to solve this crisis. With nearly 60 percent of hospitals admitted to have negative margin Medicare by the year 2004, the AHA is seeking a temporary cost of carrier judgment to the Medicare inpatient update, and two bills have been introduced to try and increase the annual inpatient update for blood payments in hospitals.

A longer-term solution that the AHA is exploring would be for HCFA to develop a specific inflation index to measure the change in blood pricing over time. This blood price index would be used to determine a more accurate inpatient update for hospitals on an ongoing basis. We urge the committee to continue to sensitize HCFA to the cost of improving the safety of our national blood supply, and we would also ask that you recommend that the Secretary make resolutions of these concerns a top priority in our department.

One final comment. My dear friend Ron Gilcher can supply blood, can supply filtered blood at an extra cost of $20. We have to pay $35 for ours.

Thank you.

DR. NIGHTINGALE: Doctor, I believe since you are here representing the American Hospital Association, I need to ask you this question. A proposal has been discussed to separate the cost of blood and blood products from the overall reimbursement for inpatient hospital expenditures. What is the American Hospital Association's current position on this proposal?

DR. MARENGO-ROWE: The feeling that I have is that we would like to see that being done.

DR. NIGHTINGALE: Thank you. Is that a statement for the record?

MS. SAVORY: I think we probably--

DR. NIGHTINGALE: Please approach the microphone.

MS. SAVORY: We'd probably like to see the specific proposal you are speaking about. We have been meeting with some of the blood groups in terms of looking at a separate blood proposal. But what we are advocating for Congress right to date is an overall update for Medicare. So that would include blood and blood products. That's a very prominent part of the campaign to get the full Medicare update

DR. NIGHTINGALE: The American Hospital Association is not unaware of this issue, I believe. The more--

MS. SAVORY: We're not familiar--I apologize. We're not familiar with what you're speaking about. We had a meeting with the American Red Cross, AABB, ABC, talking about a specific blood proposal, and if that's what you're speaking about--I'm not clear whether that's what you're speaking about or something separate.

DR. NIGHTINGALE: It would benefit all parties, certainly the government, if the AHA would make its position on that question crystal clear at its earliest convenience.

MS. SAVORY: Okay. Again, we would need to see the proposal you're speaking about before we could answer the question, and I apologize, I don't know--if you could be more specific.

MS. LIPTON: I think the issue is we've been working with AHA, and I think we have agreement. I think the way you characterized it is causing us all a little bit of heartburn. So could I just say I think that we're generally in agreement. The AABB and Red Cross and America's Blood Centers all support AHA's overall bill. We have a separate thing we've been working on with them. But when you said separate, I think everyone's going, now, what exactly do you mean?

Am I right as to the confusion? I see everybody--so I think the answer is that we would all come forward with a unanimous consensus.

DR. NIGHTINGALE: When it's in writing, my address is 200 Independence Avenue, S.W., Room 736-E, 20201.

DR. McCURDY: This committee on at least one and I think more than one occasion has heard from HCFA that there are funds sufficient in the reimbursement at the present time to pay additional amounts for blood. I found the statement as written and presented was long on allegations and short on data, and I think that for me, anyhow, to be sure of where things actually stand on this, I'd like to see some data.

DR. CAPLAN: All right. One more from Karen, and then whoever has got the next airplane ticket, please come up.

MS. LIPTON: I want to clarify that we have been in dialogue with HCFA, and HCFA has told us that they are not planning to do anything with respect to increasing inpatient DRGs as a result of blood. So there may be money in there, but they feel constrained by the Balanced Budget Act not to do anything with that vis-a-vis us. So, I mean, and that's directly from HCFA. Correct, Steve?

DR. NIGHTINGALE: You got it right.

DR. McCURDY: That's not what I meant. I meant that current reimbursements were sufficient. It's up to the hospital to move money from one place to another. And if that's not accurate, then it's time somebody brought that out in a committee like this or other sources.

DR. NIGHTINGALE: That is why I provided the public with my address.

DR. CAPLAN: All right. Let's try people in that last name A to D group. If you'll just line up at the podium, that will be great.

MR. AXELROD: Mr. Chairman--

DR. CAPLAN: I'd ask you to identify yourself for the record.

DR. AXELROD: Rick Axelrod, Pall Corporation. Mr. Chairman and members of the Advisory Committee, I must officially now say good afternoon. I'm Dr. Rick Axelrod, senior vice president, Medical Affairs, for Pall Corporation. Pall is a global company with over 50 years of experience solving complex and critical contamination problems for a diverse customer base. At least one-half of our business is with customers serving the medical community, where the requirements for product quality are among the most rigorous in the world.

I want to use these few minutes to share with the committee Pall Corporation's current status for manufacturing capacity to support implementation of universal leukocyte reduction in the United States and to confirm our mission of providing the highest quality and most reliable leukocyte reduction filter technology throughout the world.

We are pleased to see the U.S. join the growing number of countries around the world that have committed to or have already completed implementation of universal leukocyte reduction. We have worked closely with many of these nations to help ensure efficient and timely implementation, including our Western European partners, such as the United Kingdom, France, Austria, and our northern neighbor, Canada.

The issue and motivation for this removal of white blood cells is improved blood safety for the 4 million patients receiving blood each year in the United States.

In anticipation of universal leukocyte reduction in the United States, the question of product availability has been raised by blood processors and the United States Food and Drug Administration. Pall has escalated filter production and added an additional state-of-the-art manufacturing facility in Italy so that Europe, previously served by United States manufacturing facilities, may be served locally. As a result, we have accomplished our goal for increased local manufacturing capacity in order to meet the needs required to support the rapid expansion rate for routine, high-volume blood filtration in the United States in the year 2000 as stated at the December 10, 1999, FDA Universal Leukocyte Reduction Implementation Review. The FDA expects greater than 50 percent leukoreduction by December 2000. We are both confident and fully committed to having the capacity to meet these needs with a variety of the highest-quality products for leukocyte reduction of whole blood, platelets, red cells, and also for plasma, as is being done in several European countries which have recognized the benefits of filtering plasma. We partner with our hospital and blood center customers to tailor filtration systems for applications at the blood bank or patient's bedside.

Two points that I do feel obligated to address since Dr. AuBuchon suggested patient reactions and red cell loss as a potential downside to universal leukocyte reduction. One, at Pall Corporation, of 11 million pre-storage leukoreduced products transfused to patients using Pall filters, only 6 NDRs have been reported; and, two, our sterile system vent technology allows for almost complete recovery of red cells from the system and is not consistent with the reported 15 percent that we heard today.

Thank you.

DR. CAPLAN: Question, Keith?

DR. HOOTS: Just to follow up on a question from before, you're projecting 50 percent implementation by the end of this year, and I presume then 100 percent implementation by the end of 2001. Will you meet that guideline or that goal? And if you don't, what--I mean, where would--how would you prioritize who got the filters and then what would happen price-wise since there obviously is a pre-market economy?

DR. AXELROD: I can tell you that we are prepared to meet all the requirements for leukoreduction at the end of 2001. Understand, of course, we are not the only filter manufacturer, and so--but we are prepared to meet the entire needs.

DR. CAPLAN: Jim?

DR. AuBUCHON: Rick, you mentioned the filter that allows greater recovery of red cells through the filtration process. Is that the filter that's currently on the market and available?

DR. AXELROD: Yes, that's why I was wondering whether a different system was used during your study potentially.

DR. AuBUCHON: I was not referring to my study. I was referring to the published literature. Is there literature--are there studies published using that filter?

DR. AXELROD: Yes.

DR. CAPLAN: Okay. Thank you.

Next in the A to D zone?

MR. BARRETT: Mine is B. Let me be the second--

DR. CAPLAN: Let me ask you to identify yourself just for the tape.

MR. BARRETT: Sure. My name is Bob Barrett. I am vice president of Chiron Blood Testing, a division of Chiron Corporation, in Emeryville, California.

So as not to let leukoreduction have all the fun this morning, my comments are related to nucleic acid testing, and specifically Chiron's Transcription Mediated Amplification technology for the simultaneous detection of HIV and HCV, and its potential for improving the safety of the nation's blood supply.

Nucleic acid testing represents the next technological advance in ensuring the safety of the nation's blood supply. Nucleic acid testing can directly detect small amounts of virus before antibodies or viral proteins such as HIV p24 antigen or newly discussed hepatitis C antigen are at levels detectable by current screening technologies. Today's blood testing methods depend solely on the detection of these antibodies or viral proteins, so newly infected donors may not be detected due to the window period between infection and detection of these serologic markers.

Scientific studies estimate that nucleic acid testing may reduce the window period of potential hepatitis C infection by 70 percent or more and by nearly 50 percent for HIV. Using TMA technology, nucleic acid testing may be performed in parallel with current serology testing methods, and the results are available quickly enough that tested blood can be released without an impact on the availability of an adequate blood supply.

Nucleic acid testing is rapidly becoming the new standard worldwide. It is currently estimated that approximately 98 percent of the Nation's blood supply is currently being tested for HCV and HIV-I viral nucleic acid under FDA-approved clinical protocols.

The Chiron Procelix HIV/HCV combination assay is currently utilized to screen approximately 75 percent of all volunteer blood donations in the U.S. under IND. Organizations from the civilian blood collection sector and most recently the Armed Services Blood Program are now routinely screening blood donations in pools or individual donors, respectively.

In fact, this is the first time that the FDA has permitted widespread implementation prior to licensure.

Outside of the United States, the Chiron nucleic acid assay has been approved for blood screening in France, Germany, Spain, and Australia. The Australian Red Cross is currently completing validation studies and plans to begin routine screening by the middle of May this year. Various European countries are finalizing plans for full implementation of nucleic acid testing, and several blood centers in France are already screening in advance of a national mandate.

Testing under IND in the United States has been focused on evaluating the ability of nucleic acid testing to detect window period donations, among other things. In less than one full year of testing, the Chiron assay alone--and it's not the only assay being used--has identified 28 hepatitis C-infected donations and four HIV-infected donations that were not detected by current antibody or antigen screening methods. This yield is much greater than other protein-based tests, such as the p24 antigen that was introduced in 1996. The detection and subsequent destruction of these donations prevented HCV and HIV transmissions to a multitude of recipients of blood products and represents a significant improvement in public health and blood safety.

The utilization of nucleic acid testing to achieve maximum safety of the blood supply was recognized last year by Surgeon General David Satcher in testimony before the United States Congress.

In his comments, Dr. Satcher stated, and I quote, "nucleic acid tests may help to close the so-called window period between the time an infectious agent appears in the blood and the time that infection can be detected." Dr. Satcher went on to say, "even more sensitive nucleic acid tests performed on single units are under development. These advances should reduce the risk of transfusion-transmitted Hepatitis C and HIV by an order of magnitude, and possibly even more."

Thank you.

DR. CAPLAN: Comment? Question?

[No response.]

DR. CAPLAN: Thank you.

Anybody else in the A to D last name?

MR. DARLINGTON: My name is Alan Darlington. I am the chairman of HemaCare Corporation, and I'm here to address a narrow issue.

We are a mobile therapeutic apheresis services provider. Nationally we contract with approximately 150 hospitals, and as it relates to--as a member of the blood industry, we're very pleased with the improvement in the overall reimbursement for the blood industry and for our services between the original proposed regulations and the proposed APC schedule we see today.

But with respect to APC 0111, which addresses therapeutic plasma exchange or therapeutic apheresis, the rate is $687, and that in our instance, and we believe in the instances of other mobile service providers, will cause us to leave the business.

We believe that more than 75 percent of the hospitals in the United States that offer therapeutic apheresis services do so through contracts with organizations like ours, dialysis companies, and their local blood centers. And as a result, if mobile service providers leave the business because it becomes uneconomic, we believe that there will be a drastic reduction in the availability and the accessibility of therapeutic apheresis services throughout the U.S.

Our principal or our largest operations are In Los Angeles, and just looking at the Los Angeles or Southern California market, we'll describe the problem in its overall dimension. We operate in L.A. County, Orange County, and Ventura County. That's an area with a population of approximately 14 million people. And within those three counties, there are 183 acute-care hospitals, 15 of which have in-house programs that offer therapeutic apheresis services to patients.

Our company serves another 85 hospitals offering that service on an outsourced basis, and other mobile service providers we believe serve somewhere around 20 or 25 hospitals.

In the aggregate, our company performed 2,700 therapeutic apheresis procedures in our service area last year. On average, that is only 32 procedures per hospital, and candidly, the range in our client base is dramatic. It ranges from one procedure at an institution in the course of a year to almost 400 procedures in the course of a year.

We think in this narrow service area that rethinking reimbursement is necessary because, again, companies like ourselves will not be able to operate given the proposed costs, and companies like ourselves truly do have costs that are greater than the in-house costs of large institutions with high-volume programs. Those costs relate to the fact that we move our nurses, we move our machinery, we set up anew to treat one patient, and then we go away. We have additional administrative costs in terms of our own medical direction and overhead costs. And in the context of what we charge in Southern California, we charge our hospitals on balance about $1,000 a procedure. That's our costs. That doesn't include the costs of the hospital associated with providing the room, providing necessary back-up support.

We also, because it's a rare procedure, we have adjustments for complications, and on balance, our charges are roughly $1,200 per procedure excluding fluids.

While only 15 percent of our services are performed on outpatients and about half of that in Medicare outpatients, we believe that with the established rate that HCFA has set in these regulations in the APCs of $687 that we know in California and nationally that will become the standard for all patients and all therapeutic apheresis services, whether done on an outpatient basis or on an inpatient basis. And we respectfully ask that this committee ask HCFA to re-evaluate that.

Thank you.

DR. CAPLAN: John?

DR. PENNER: A question. Maybe Dr. Gilcher could answer this. The difference in cost between the smaller hospital facility use, bed cost, whatever it is, as compared to a larger facility, is there that much distinction so that it would be much cheaper to run the apheresis unit on a mobile operation in a smaller hospital than it would be to go to a big center?

DR. GILCHER: In our service area, we provide the identical type of service that HemaCare provides in the area, and we have a fixed charge to a hospital, whether it's small or large. We're moving equipment, moving nurses, but it's provided by the blood center.

DR. PENNER: But is there a savings on using facilities at a smaller hospital that would have lower costs perhaps--

MR. DARLINGTON: Perhaps I could--

DR. PENNER: --as opposed to a major center?

MR. DARLINGTON: Excuse me. Perhaps I can address that. One of our clients is a large medical institution in the Northeast, in Maine. We perform 700 procedures annually in that institution. It's the largest referral center in its area, and there our costs are substantially lower than what I referred to here. And you can operate at reimbursement rates that are--

DR. PENNER: So there is a benefit on being able to go to smaller hospitals with costs of medical care than to just having everybody go to a large center where they are operationally functioning with the system?

MR. DARLINGTON: Well, let me first of all say that there are definitely cost benefits to doing therapeutic apheresis procedures in high volume at a single institution. What is involved with the smaller hospitals and the sacrifice that's made is the interruption of the patient-physician relationship if the patient has to be transferred to a major medical institution, and the fact that there will be a lot of, in many instances, unnecessary and expensive transportation costs associated with moving the patient to where the service is rather than the reverse.

DR. PENNER: I'm just trying to get at whether if you sent the patient 60 miles down the road that it would be cheaper than having the patient go in the 50-bed or 100-bed hospital that is out in the rural area.

DR. GILCHER: John, I'm trying to understand your question because I can tell you it's very clearly cost-ineffective for a small hospital to be doing this procedure. Number one, they don't have the expertise because they don't do enough procedures to be competent to do it. And it's clearly much more effective to move competent people and equipment. You reduce the total amount of equipment that's needed that way.

DR. NIGHTINGALE: Mr. Darlington, I assume that you have made this representation to HCFA directly.

MR. DARLINGTON: We plan on making these comments in the next few--

DR. NIGHTINGALE: I think that would probably be a more effective as well as more appropriate venue for those. This committee does not, I think, have the expertise or probably the time at this particular juncture to consider this specific area as--to give this area the consideration that would be its due. But I do thank you for being here.

MR. DARLINGTON: Thank you.

DR. CAPLAN: Anybody in the E to H last names?

I can see people checking their last names. All right. How about H to N? Anybody there? And how about--oh, somebody go in there? Okay.

MS. LOWERHAUS-WIEGMANN: I've been recommended to use my maiden name from a week ago.

DR. CAPLAN: That's the spirit.

[Laughter.]

MS. LOWERHAUS-WIEGMANN: My name is Theresa Lowerhaus-Wiegmann, and I am representing the American Association of Blood Banks today, and I'll try to summarize my comments in consideration of the time limits.

Again, we at AABB are generally quite pleased with the outpatient final rule as issued by HCFA. But now we believe it's critical that we build upon this progress and take similar steps to ensure fair reimbursement in the inpatient setting.

As we've discussed, the vast majority of blood products and services are provided to inpatients, and HCFA's important recognition that the safety of the nation's blood supply is a major concern to the department and that they want to encourage appropriate testing and follow-up care clearly must apply to patients being treating in inpatient as well as outpatient settings.

Therefore, the AABB urges the Advisory Committee to make the strongest recommendations possible in support of legislation to provide Medicare inpatient reimbursement payments that fairly account for the costs of providing state-of-the-art blood products and services, including the costs associated with new blood safety measures.

The AABB believes that, at a minimum, legislation should include the two following types of provisions: first, legislation should provide additional payments for inpatient services specifically to account for the increased cost of providing blood stemming from new blood safety measures, including those that have been recommended by the FDA and/or those that have been adopted as the standard of care; secondly, we believe that HCFA should be required to reflect the increased cost of blood products and services, including new safety measures, in its annual updates of the DRGs.

The first of these provisions, the additional payments for the increased cost of blood, will help address the immediate financial burdens placed on blood centers as well as hospital transfusion services in providing both leukocyte reduction and NAT. The introduction of these two safety-related measures alone is expected to add over 40 percent to the cost of every blood component in the United States. And this reimbursement relief is needed, again, at the earliest possibility date.

In addition, systems must be put in place to ensure that HCFA has better data regarding the types of blood products and services provided to Medicare beneficiaries and their related costs. Establishing a separate market basket designation for blood products and services could help ensure this data is collected in future years.

Lastly, on an annual basis, at a minimum, HCFA must adjust inpatient DRG payments to reflect changes in the cost of blood therapies. Such an annual adjustment will be included in the outpatient APC program, and we clearly think that a comparable system is merited in the inpatient setting.

As we all know, blood is an ever-changing biologic, the safety of which can never be 100 percent guaranteed. Nonetheless, the blood banking and transfusion community are dedicated to ensuring patients have the safest possible blood therapies. We can anticipate additional incremental safety improvements, with additional costs, to be introduced in future years. Systems ensuring fair reimbursement must be put in place to ensure that typically not-for-profit blood centers and transfusion services have the necessary financial support to provide such enhancements in blood safety and patient care on a timely basis.

Just to touch briefly upon the discussions with AHA, what I spoke to today about a legislative solution, AABB has been working closely with Red Cross, America's Blood Centers, and others in the blood community to try to develop a specific legislative proposal to move forward this year, and we have spoken to AHA and I believe have a general agreement that something is needed to add these additional dollars to the Medicare system to account for the increased costs of blood. And as we move forward with more particular legislative language, we'd be happy to share it with the committee and continue to look forward to working with you, Congress, and the administration in addressing this critical issue.

DR. CAPLAN: Comments, questions?

[No response.]

DR. CAPLAN: All right. Anyone else in that pre-N category?

MS. LANE: Yes, will Lane do after Lowerhaus?

[Laughter.]

MS. LANE: Good morning, Mr. Chairman and members of the committee. I'm Jan Lane. I'm vice president of Government Relations for the American Red Cross. I extend the regrets of Jacqui Fredrick, our senior vice president for Biomedical Services, who wanted to be here this morning. She wants me to share with you the absolute importance that the American Red Cross attaches to the issue of reimbursement, and especially from the standpoint on the impact of ensuring continued patient access to state-of-the-art blood products and services.

On behalf of the American Red Cross, first let me thank this committee for their leadership in addressing the issues related to the outpatient rule. Today's discussion focuses most appropriately on ensuring continued patient access to state-of-the-art blood products. The Medicare program, as many of you know, typically represents between 35 and 45 percent of a hospital's total patients, and more than 5,000 hospitals fall under HCFA's PPS, or Prospective Payment System. Reimbursement policies adopted by HCFA can often become the norm for third-party payers. Therefore, it's important that improvements in safety of blood products and services are recognized expeditiously by HCFA and adequately reimbursed through Medicare. Improvements such as newer versions of existing tests, universal leukoreduction, and nucleic acid testing need to be reimbursed quickly in order that patients can benefit as soon as possible from that therapy.

Blood safety has been declared a national public health priority. There is universal agreement that improvements that ensure the safest possible blood supply should be adopted immediately. Consensus on this public health issue was articulated in 1995 through the Institute of Medicine study which recommended, and I quote, "where uncertainties or countervailing public health concerns preclude completely eliminating potential risks, the FDA should encourage, and where necessary require, the blood industry to implement partial solutions that have little risk of causing harm."

Since 1994, the American Red Cross has invested more than $300 million in technologies and systems to improve the safety of the blood supply. We have been at the forefront of many of the improvements, including NAT. We have invested $17 million in research and development alone to make that test a reality. We estimate that NAT will add approximately $9.50 to the cost of each unit. The Red Cross has also made a commitment to universal leukoreduction of our blood components at an estimated cost of what you have all heard here this morning several times of $30 to $40 per unit.

In the near future, the Red Cross also anticipates the introduction of pathogen inactivation for transfusible components. This is truly an exciting way to move. Although still in the research phase, this cutting-edge technology for virus inactivation could work on all blood products and viruses.

Let me just cut down a little bit. Unfortunately, the annual adjustments performed by HCFA to the DRG payment system do not adequately reflect the increasing costs of blood products and services. The market basket index and other indices used by HCFA to adjust the DRG payment system do not specifically take into account the increased cost of blood products, and as you all well know, blood centers are not-for-profit centers. Hospitals are in a similar bind, especially so since passage of the Balanced Budget Act of 1997.

Therefore, we'd like to offer several steps that we believe would ensure adequate reimbursement in the inpatient setting.

First, the Department of Health and Human Services should institute a formal mechanism to ensure that new blood safety measures recommended by the FDA or adopted as the standard of care in transfusion medicine are expeditiously reflected in annual adjustments promulgated by HCFA in both the inpatient and outpatient setting.

Following release of the IOM report, HHS moved to address the issue of blood safety and availability in a high-level, coordinated fashion by establishing the Blood Safety Council and this very committee. It's imperative that HCFA reimbursement policies are consistent with the priorities of these bodies.

Second, we suggest a request that there be an additional payment to the Medicare trust fund for federal fiscal year 2001, starting in October 2000, to cover the added costs to blood products resulting from NAT and leukoreduction. These funds should be directed in a manner which ensures adequate reimbursement of all inpatient services utilizing blood products and services.

Third, blood utilization data must be collected and analyzed in order to incorporate the increased costs of blood products and services and human-derived plasma derivatives into the DRG payment system. This should be done in order to allow for more timely adjustments for blood safety measures that are federally recommended or occur through changes in standard of care.

The Red Cross has heard from our hospital customers and you heard from AHA itself this morning that they are finding it increasingly difficult to pay for these advances. We all agree that safety of the nation's blood supply is the number one priority. Providing these life-sustaining and life-enhancing products to patients nationwide can only occur if hospitals received adequate reimbursement from third-party payers.

Thank you for the time this morning, and I'll take any questions there might be.

DR. HOOTS: Just in terms of the filter costs that you were quoting versus--

MS. LANE: Pardon me?

DR. HOOTS: The cost of leukodepletion, is that cost plus labor, is that the reason we're seeing a differential between what was quoted, say, by Dr. Gilcher--

MS. LANE: I believe that may be, and it may be deviations among our regions, but I will defer to our chief medical officer, who is here today.

DR. DAVEY: I think it's a cost differential between our regions. Actually, I'm not the real expect to comment on costs, either, Jan, but I believe the costs do reflect labor costs, differential costs of the areas that we're in, and there is a range of increase, between $30 and $40 I believe are incremental, Keith.

DR. NIGHTINGALE: Then I would just point out for the record that we have heard other estimates of cost.

DR. CAPLAN: The Chair would like to point out for the record that all leukoreduction should be done in Oklahoma.

[Laughter.]

MS. LANE: We'll try to move there, but I don't think that that would make Dr. Gilcher too happy.

DR. CAPLAN: Okay. Who have we got in the N to S category for presentations?

DR. SANDLER: My name is Gerry Sandler. I direct the Blood Bank and Donor Service at Georgetown University Medical Center. I'm a professor of medicine who takes care of patients with leukemia, lymphoma, sickle cell disease. In fact, I almost missed my presentation because I was buzzed to do an exchange in a sickle cell patient.

I want to give you a perspective from the bedside, where the patient is, and from the hospital. I've been transfusing blood for years, since 1968. Something has happened that has gone wrong. The person at the bedside who is the patient advocate, Dr. AuBuchon, is being painted as the person who's not, and a bunch of patronizing bureaucrats are taking the role of we're for the safety and you're not, we know better than you do, and our 13 experts up in Bethesda in some hotel know more than the transfusion medicine expertise.

The whole world's gone topsy-turvy. I want you to know how this has gone down here in the city if you need blood today. My hospital is receiving 200 units of leukocyte-reduced blood every month now that we don't order--I'm an expert on cost--at $40 from the American Red Cross in Baltimore. That's $8,000 a year. We don't want it. Science hasn't proved it. This is a micro-step in safety.

We made big strides when we put in HIV for $1.50, hepatitis C, a whole bunch of things, even NAT testing. But $40 for this micro-step needs more thinking.

Karen Lipton is entirely right. We're getting hit in the hospitals right this minute. Unlike a lot of you people, I have to work within a budget. I'm like the DOD.

We need help now. Someone has pushed me over the precipice because I have to stay within a budget, and the effect of this universal leukocyte reduction has been to decrease safety in my hospital during the last several months. Someone's got to get that message to bureaucrats, because those of us at the bedside don't see it.

Thank you.

DR. CAPLAN: Questions, comments? Jim?

DR. AuBUCHON: In support of Dr. Sandler's statement, I would note the results of a survey that was conducted as part of efficiency testing in the College of American Pathologists in over 5,000 hospitals two years ago. And this was well before universal leukocyte reduction and increased costs for NAT and other changes in the way we provide blood. Eight percent of hospitals at that time noted that they had already been forced to make changes in their operations within the transfusion service because of budget reductions that decreased the safety of what they did. And that was two years ago.

I'm afraid that what Dr. Sandler is seeing in Georgetown is only going to accelerate to more hospitals across the country unless we take some action to provide them the resources that are commensurate with our expectations of increased safety.

DR. CAPLAN: Jay?

DR. EPSTEIN: Well, I would like to get some clarification of what I perceive as a disconnect. Red Cross has told us they're about 50 percent leukoreducing now. They're about 45 percent of the system. That's about 3 million filtered units per annum at an added average cost between $30 and $40, or $35 per unit. So we're talking about an added cost of $105 million, which has to be coming from somewhere.

Now, presumably, Red Cross does what everybody else does, which is they sell their units to hospitals. So on the one hand, I'm hearing many entities state hospitals can't reimburse us for the added cost of this measure. On the other hand, I'm hearing Red Cross say it's happening.

Can somebody please explain how it can be happening for the Red Cross, happening for Oklahoma Blood Institute, and yet the argument is that it can't happen? So there's something here that doesn't add up, and maybe it has to do with compartmentalization of the system. But I think we need to hear a little bit more about that disconnect.

DR. CAPLAN: All right.

MS. LIPTON: I think what you're seeing is that the costs are being passed along to the hospitals, but in Gerry Sandler's facility, I presume Gerry Sandler is a cost center--and I don't know what your budget is per year, but what happens is it gets passed on. Well, they're not going to give him more money, so what they say to Gerry is you can buy this stuff, that's fine, but that means you have one less person, so you lay off a person.

And my point is what's happening because of the way the DRGs are constructed and we don't get any more when the cost of blood goes up, those aren't adjusted fast enough, particularly in the past year when we saw--we're talking about a 40 percent jump in the cost and the price of blood, not these incremental things. It gets passed on at the hospital level. The hospital says, okay, we're going to leukoreduce, but guess what? Now we're going to share that technologist between the chemistry lab, the blood bank lab, and everything else. So now we're not going to worry about having a specialized person who really knows how to do cross-matching and compatibility testing. Now we're going to pull someone in and cross-train them.

My concern in this is that as we add the cost to the product and we don't push the reimbursement up, it comes out of the process, and the very thing that we were talking about yesterday trying to prevent, which is errors and accidents, we're going to be causing. And so I'm just--you know, my plea here is that it is happening, Jay. People are already doing these things and passing the costs on. But it's having an effect already in the hospitals in terms of safety.

DR. CAPLAN: I understand that Gerry may have something to say about this himself.

DR. SANDLER: Well, I think the disconnect occurs right across my desk. I wake up in the morning, I go out and I look at the patients on leukemia-lymphoma service, I look at the chronically transfused patients, some who get colorectal surgery and a few others of the so-called indications. We use CMV--seronegative tested blood to cover the CMV because it is better than leukocyte reduction for that purpose.

I issue my orders to the blood bank, and I want so many units of blood. I get 200 more per month than my physicians and I want from the Red Cross. I call them up and say, Hey, you know, we can't pay you, we don't want this. When science catches up to this, we will do it. And when the FDA issues a mandate, I guess we're going to have to do it, too. Hopefully, maybe someone will pay for this, but it's not on my list of priorities what I'd do first. I'd do a lot of other things first.

The disconnect comes when I get my budget and this bill comes in from Red Cross forced upon me right this minute, and I've got to pay it. And I say, Hey, I don't want to pay it and I don't want it. That's a big disconnect.

DR. CAPLAN: John?

DR. PENNER: Gerry, I presume your sickle cell patient got leukoreduced, but coming around to the fact that--what percentage of the blood products that you're using do you feel you necessarily have to have leukoreduced? Twenty?

DR. SANDLER: It's between--yeah, it's between 20 and about 25 percent, and the high number is for balancing the inventory. In other words--as I say, I just got a call, it was a B-pos, and I happened to have enough to make it all. So you need to have more in your inventory than you can match.

DR. PENNER: So 25 percent of what you use you would feel would require leukoreduced, and if you could--right now you're getting something like 40 percent or 50 percent?

DR. SANDLER: I can't put the numbers on one foot, but I can tell you it's 200 units a month for the last three months. We wanted 79. We got 279. That's sort of how the numbers work their way out.

On a few of our indications, like in an emergency, we have had excellent in-service for using bedside filters. I wouldn't claim that a bedside filter is going to do a lot of the theoretical things that people claim they can do. But in a pinch, with the unit that has been in-serviced, a bedside filter does a terrific job, and we've got good quality control data to show that. We don't want universal leukoreduction the way it's coming down forced upon us.

DR. PENNER: Do you feel your medical staff are attuned, say, in the emergency room and so on to appropriately use the leukoreduced--

DR. SANDLER: No, we don't use it there. We have the Lombardi Cancer Center. There's tubeless--it's a particular unit. We brought the manufacturer in multiple times. We have very good in-service. And should we, with the 30 percent inventory we have--our supplier is in Baltimore. So, hey, you know, someone comes in in a hurry and we just don't have the right stuff, we are very confident--I go up myself sometimes, jam it in, run it through, make sure that it's done right.

Since 1968, I haven't seen this great big safety problem that all these other guys are telling me. We did an NIH-funded multi-center study--Dr. Busch was one of the heads of that--because leukocyte-reduced blood was shown, was proposed to be better for HIV patients who might get CMV. The results of that study--it's known as the VAT study--published showed, hey, no benefit whatsoever. None. It's the only NIH-funded multi-center study that was out there. No benefit whatsoever.

We shouldn't close the scientific debate. We must not close the scientific debate. The Red Cross is going to get this stuff out. We won't be able to do studies to see if it works or not. Something is terribly wrong here.

DR. DAVEY: If I could comment on the position of the Red Cross on how we're moving this forward. And, Gerry, I think you'd better go back and look at your system, because the Red Cross will only fulfill orders as they come in for leukocyte-reduced products. The only caveat that our regions have are that when the region gets to a certain percentage--and this is the way it is at Chesapeake and Potomac, Gerry--when the region gets to a certain percentage of leukocyte-reduced requests, it becomes inefficient and cost ineffective for the region to maintain two more inventories when it gets above a certain percentage, so some of our regions have moved to 100 percent, some have not. And we will work with customers within the region as we move this implementation forward, and again, I hope that the FDA can issue some guidance so that we can all work under some regulatory framework.

DR. SANDLER: The record should reflect that Georgetown University Hospital is supplied with leukocyte-reduced blood by the American Red Cross, and we do not order it, and we are forced to pay for it.

DR. CAPLAN: One of the benefits of the Committee is that it allows parties to sometimes communicate their consumer gripes. Who else was in after "S?" Who have we got left here? One, two, three. Give me your hands again. All right. We can do this and then go to lunch. Okay.

MS. SMITH: Hi. Thank you. My name is Kristin Smith, and I'm the associate director of legislative and public affairs at America's Blood Centers. You all have our written statement, so I'm just going to be very brief and summarize.

America's Blood Centers is the association of 73 independent not-for-profit blood centers, supplying about--or collecting about 50 percent of the US blood supply.

First, we would like to thank HHS, Congress, and particularly this Committee, for their great efforts to insure adequate reimbursement in the outpatient setting for blood products and services. Our hope is that the positive steps that have been taken in the outpatient setting will now be replicated in the inpatient setting, where the vast majority of blood transfusions take place. It's critical that adequate reimbursement and quality of care be consistent in both settings, as has been echoed by some other groups.

Our written statement outlines basically our proposed solutions, which again are consistent with the solutions proposed by AABB and Red Cross and AHA in their statements, but basically, it's in the short term that we would ask that Congress increase Medicare funding in amounts equal to the cost of new blood safety measures that have been recommended by the FDA and/or adopted as the standard of care in transfusion medicine, NAT and leukoreduction being the main ones there.

In the long term we ask that Congress direct HCFA to reflect the rising cost of blood therapies in its annual DRG adjustments, and we urge the Committee to make recommendations to support these recommended proposals.

DR. NIGHTINGALE: Sorry. Ms. Smith, a question for you and your colleagues.

MS. SMITH: Uh-huh.

DR. NIGHTINGALE: Should the Congress provide additional funding to the hospital industry to reflect the increased cost of blood, what if any guarantee does the blood industry that those costs will be passed on to you?

MS. SMITH: I don't know that there could be a specific guarantee. I think our sense is that we want to make sure that there's more money in the system to pay for these blood safety measures. I don't think, not being reimbursed directly, that we can guarantee that that would automatically go to the blood centers, but it's in our--

DR. NIGHTINGALE: Well, let me emphasize that I did not mean to put you specifically on the spot. This is a question that we have struggled with for over a year. We have called this "the DRG tweak" in the past. It has been very extensively discussed, and the concern of the department has been just the one that I expressed, and we haven't got an answer to it yet, and we would love to have one.

MS. SMITH: I would welcome anyone else's comments, but my thought is that our concern is not--our concern is that those costs be covered and that the hospitals get additional money, and frankly, that's our main part of the concern. Whether it gets to the blood centers or not, we really can't control.

DR. CAPLAN: Jim?

DR. AuBUCHON: I would like to provide assurance to the executive secretary and the department that the collections departments of blood centers are extremely effective, and also the threat of not delivering blood if we don't pay our bills is very effective. Therefore, money that is passed to hospitals to pay the bills from blood centers does indeed ultimately find its way to blood centers.

DR. NIGHTINGALE: To clarify, the last sentence was lost. I didn't--you feel confident that any money that went to the hospitals--Dr. AuBuchon feels confident that any money that was allocated to hospitals for an increased price of blood would be transferred substantially to the blood centers?

DR. AuBUCHON: I think that's entirely predictable, unless in the unlikely event that the government should reimburse more money than the blood actually costs.

[Laughter.]

DR. NIGHTINGALE: Okay.

MS. LANE: Dr. Nightingale, I can't speak to how hospitals allocate their costs internally, I can only speak to the intent behind the legislative solution, that AABB and ABC and the American Red Cross are all working towards. And I can tell you we've joked about this. This is probably the first time that ABC and Red Cross have been on the same page in a long, long time, and we do have consensus here.

All we can do is advocate on behalf of our hospital customers and press their concerns, and try to identify within the course of the discussion the fact that blood safety is key. There's universal agreement. Let's find a mechanism within HCFA and a mechanism within HHS, where there's a coordinated fashion. And we recognize that a hospital is fully able to direct those costs wherever they would like, but certainly it is a message that we will share with our hospital customers about the time and resources that we've devoted in order to increase that reimbursement.

DR. CAPLAN: Okay. Dave?

MS. SMITH: I'm sorry. Just to add on that too. In the interest of time I didn't go into detail on our longer-term solution, but part of the long-term solution that ABC has sort of supported and that's also been supported by the other groups, is to--for within the DRGs in the market basket update--because there's currently no mechanism that measures the cost of blood over time--if that type of mechanism were added to the market basket, that would specifically go to blood costs and would address that issue.

DR. CAPLAN: Okay. Let's have the next presentation. I ask you to identify for the tape.

MS. REARDON: Hi. My name is Susan Zagami Reardon, and by marriage I come into this category, I guess.

I'd like to make sure that just for the record, that all of the written statements will be fully entered into the record, Dr. Nightingale?

DR. NIGHTINGALE: Yes, they will be.

MS. REARDON: And I'll try to summarize my comments. I'm the director of reimbursement and health policy for Ortho-Clinical Diagnostics, a Johnson & Johnson Company. We have a rich history and a deep commitment to blood research and technological advances to insure blood safety. Led by pioneers such as Dr. Philip Levine, one of the discovers of the Rh factor in blood, the company has long provided the medical community and patients with important technology used in transfusion medicine.

Our products span the transfusion medicine continuum from the infectious disease testing at the donor screening centers, to the blood typing and cross-matching products used in hospitals. For the record, we do not make any NAT or leukoreduction devices. We are constantly striving to improve our technology and to develop new technology to respond to patient needs.

In addition, we have a very strong corporate commitment to blood, and in fact, have been number one in company corporate blood drives over the past four years, and collected actually 40,000 units of blood from employees last year.

We are here to add our voice of support to the recommendations made by AABB, ABC and ARC with regard to an immediate legislative fix to this problem of reimbursement, and we urge you to adopt a resolution today that would send a clear message to Congress that this Committee supports this action.

I do have a limited amount of time to speak, and I'd like to just clarify a few of the key elements of this legislative proposal that have been under discussion. One of them is that this proposal would be one for new money in the system. It would not be a budget neutral proposal. The proposal would probably ask for anywhere from 300 to $500 million per year for this add-on. The add-on would be to each DRG in the system that actually uses a pint of blood, with some kind of an average unit determination being the preferred way to go for administrative simplicity.

A price would be determined, an amount of the add-on would be determined that would reflect safety measures generically. It would include leuko and NAT reduction. So each time a hospital submits a bill for that particular DRG, it would receive an add-on that would reflect these costs of safety measures, whether it be $20, $40, whatever, that amount has not been determined.

Another point I would like to make is that there have already been preliminary discussions with members of Congress. We've gone to see some very key people on the Hill, and have met with a fair amount of sympathy with our plight. So again, your voice added to this whole debate, would, I think, carry a great deal of weight.

I'm not going to go through each of the bullets on my list here in the interest of time, but I would point out that HHS, in its own 5-point plan, states that the economic and competitive pressures of health care today make it nearly impossible for blood banks to recover the cost of new innovations, even when such measures are required, and that these economic limitations are a strong disincentive for change.

I'd also like to mention that Blood Centers of California report that their members have lost $33 million over the past three years, and that not only is it the BBA of '97, but also managed care cost-cutting measures that have really put a financial squeeze on hospitals that really exacerbate this problem.

Inadequate reimbursement has an adverse impact on all parts of the blood continuum, including us, those in the manufacturing sector. Economic affecters, believe it or not, affect our decisions to develop new technology that may improve patient care, reduce error, and insure the safest possible blood products and services. And new threats to the blood supply will require new technology. Without adequate reimbursement, the health and vitality of those of us who are the innovators in this industry, who develop the new technology to make blood safer, is jeopardized.

I'd also like to point out that new blood safety technology is routinely adopted in foreign countries. A variety of reasons for this: regulatory reimbursement environments contribute to that phenomenon; leukoreduction is mandated in at least 9 countries. Another example is our own company's HCV core antigen test. This is a test that we believe is substantially equivalent in performance to current NAT testing. It's available outside the US for a single-unit testing, rather than as a pool test, and an ELISA microwell plate format that is an established technology, well understood by even the smallest of donor centers. We intend to submit an application to the FDA for US approval of this test.

Again, as others have said, the current Medicare payment system does not easily lend itself to traditional types of increases to the DRG weights. Blood is a relatively tiny portion of the total $230-billion per year price tag that Medicare currently has, and since blood costs are spread throughout the system, we believe that this is a reason for particularly separating it out and adding this amount.

And again, the administration has recognized the importance of blood and the importance of safety in blood, and has adopted a system that we believe should be the basis and justification for an inpatient fix.

So in conclusion, we do support this appropriate add-on, and also support the fixes for the future related to the annual update and the market-basket approach. Thank you very much for the opportunity to comment. Any questions?

DR. CAPLAN: Questions, comment? Okay, thank you.

MS. REARDON: Thank you.

MR. JACKMAN: Good afternoon. My name is Dennis Jackman, and I'm in this alphabetic slot by right of my mother's maiden name, which begins with a "P", so thank you very much.

[Laughter.]

MR. JACKMAN: I'm going to limit my comments to the outpatient PPS. PPTA, the Plasma Protein Therapeutic Association, would like to commend HCFA's rule on Medicare outpatient prospective system in regard to plasma protein therapeutics. Very positive, and a major improvement over the initial proposed rule. This Committee's resolution, I think, was very, very key in the outcome of that final rule.

The final rule does provide separate APCs for each separate plasma protein therapeutic category by J-code. This provides very appropriate differentiation and allows for reasonable market-based rates of reimbursement to reflect the resource-based value of these therapies. Investments in safety are also recognized. In short, patient access and proper medical care, using today's therapies, will be advanced, and investment in new technologies and therapies and also in production capacity, will not be discouraged. All of this will impact supply.

But the job is not quite done. The rule states that this policy will be examined two to three years after initiation for data, after-data, so data can be collected. What needs to be assured is that the providers understand the rule and code appropriately.

Let me go back to the early 1990s when there was a temporary transition pass-through, transitional pass-through for clotting factor in hospital settings. The transitional role was not utilized. It was not widely advertised. People were not educated in it, and it actually expired, and it took an act of Congress ultimately to put it back into place, and now it's a permanent law.

What would prevent that from happening? Data collection should take place after some implementation period, and that should be updated regularly as well. So HHS and HCFA can be very helpful in assuring that this happens.

We would urge HCFA to promptly issue a guidance to entities covered by this rule that explains how to utilize the provisions of the rule for plasma-related therapeutics, and for that matter, for blood. We also look forward to HCFA working with all interested parties, consumers, manufacturers and others, to develop and implement a comprehensive data-collection program.

It is critical that correct utilization data and data are present in two to three years when the rule is revisited. A good policy should be continued, and we would hate to see a lack of data prevent that from happening. Thank you very much.

DR. CAPLAN: Questions, comments?

[No response.]

DR. CAPLAN: One final public comment. Then we'll do lunch, and come back and discuss any recommendations that we decide we want to make.

DR. WEINSTEIN: Good afternoon. My name is Robert Weinstein. I'm a hematologist, and it seems to be my fate to be the last speaker.

[Laughter.]

DR. WEINSTEIN: I don't have a prepared statement to read. I intend to stammer through a semi-extemporaneous one, however. I have three points that I would like to make before the Committee.

As a preamble, I would like to add my voice to those who have expressed their gratification and in fact their admiration for the work of this Committee in achieving what for the most part is a tremendous victory in the final rule of the outpatient prospective payment system. I think for the most part, it demonstrates that the system works if people participate in it.

There is one slight imperfection in the final rule that I would like to call your attention to, because I suspect it was an oversight, and as such, if attention isn't called to it, an entire new field of therapeutic apheresis, a small but very important one, is threatened, because without reimbursement, it will not survive in the American marketplace.

In January of this year a new CPT code was brought online. It's 36521. And the narrative for that is--it's "extracorporeal absorption apheresis with plasma reinfusion." Now, this was brought online to cover new technologies that have come to the marketplace after the base year of 1996. These include the Lipabsorba [ph] and the Health device to treat stat and refractory familial hypercholesterolemia. It includes the new indication for the Presorba [ph] device for treating medically refractory rheumatoid arthritis, and it includes the Excoram [ph] device, which was approved in, I believe, the spring of 1998 by the FDA, for the treatment of hemophiliacs with high-titer inhibitors to Factor VIII, who because of their inhibitors do not respond to Factor VIII infusions. These devices are all covered by the CPT code 36521.

In January of 1999 the American Society for Apheresis submitted comments to HCFA in response to the original prospective payment system proposal that came out in September '98, and recommended that as part of the anticipated reorganization of APCs to be in compliance with the statute, that the new CPT code anticipated for these new therapies, be bundled together with the code for photopheresis, which is similar in concept to these new therapies and similar in cost.

Again, in May of 1999, ASFA [ph] made the same recommendation to HCFA. The American Society of Hematology, in its comments in June, specifically endorsed those recommendations by ASFA.

In January of this year, a very famous and influential document was issued by the AABB-led coalition of societies. This was the one which recommended that the old APC Number 369 be divided into four new APCs, and in fact, this document has been heavily referred to in the comments in the final rule that HCFA put on their website and in the Federal Register.

In this document, it was recommended that the new CPT code be bundled with photopheresis, for the reason that I already stated, because it follows the requirements of the Balanced Budget Reconciliation Act of '99, or whatever it was called, for clinical coherence and similarity in cost. Somehow, despite at least four recommendations to bundle this new CPT code appropriately, it didn't happen. So instead, these procedures, which ASFA's data submitted to HCFA indicate cost hospitals between 2,000 and $2,500 to perform, have been bundled with therapeutic plasma exchange, which as we heard earlier today, is paid $687.

Now, I think this was a mistake. It has to have been an error, but I think if this Committee doesn't call attention to that, it's just going to go unrectified, and within a year I think these devices will disappear from the marketplace, and little boys with hemophilia, who are refractory to Factor VIII infusions, will bleed to death, and young people with hypercholesterolemia, who have already had their first myocardial infarction before age 35, but who don't respond to ATOR [ph] VA-stat will die, and certainly patients with rheumatoid arthritis will wind up paying more for Embril [ph] and Remicaid [ph], than they might pay for Presorba treatments. So I would ask you to consider calling attention to this, because I think a simple repositioning of a CPT code in a proper APC will solve that problem.

The second comment I want to make is one that came to me while listening to Mr. Darlington's comments. He referred to the fact that the reimbursement for plasma exchange is lower than can be sustained by the providers of that service. It seems like a narrow issue at first blush. However, again, twice, in January and then May of '99, the American Society for Apheresis made recommendations to HCFA, based on its own research into the cost of providing these services. Specifically, ASFA submitted to HCFA that when a hospital-based program does plasma exchange or a red-cell exchange with therapeutic apheresis, apart from the cost of the biological fluid, which has been separated now, the apheresis cost for an in-hospital program was $750 in 1998, mind you. When a mobile apheresis provider provides those services, the cost data submitted by ASFA to HCFA was $854.

In November, at the AABB meeting--Dr. Nightingale may remember the discussion we had about the remedies that were forthcoming, and we anticipated a favorable structure, restructuring of the APCs. But the question was asked, what numbers will be used? Now, the numbers for the blood products I think we're all pretty happy with, and the plasma products and derivatives, but for the life of me, I can't fathom where this $687 number came from, and we had asked HCFA to work with the societies to come up with a number that really represents the costs of doing these things, and I'm hoping that that possibility is not dead, because if it's true that in certain parts of the country 75 percent of therapeutic apheresis is done by mobile providers, and they can no longer do it, I ask who will? This will be an unintended consequence of the final rule, to create a barrier to these therapies for Medicare patients.

The final point I want to make, I guess relates to the DRG tweak, a term I just learned this afternoon. I don't know whether, as has been said, there already is enough money in the budget, in the inpatient budget to accommodate increases in the cost of blood products, or whether another $300 million is necessary. I don't know that. But I do know--and it may be true, but again, I don't know. But I do know that we've learned from the outpatient reimbursement business that inflation in blood products and derivatives is very different from medical inflation in general. It's faster, it's higher, and it's mostly because of mandated safety measures and because of the heightened public scrutiny of blood that requires that we implement safety measures, even if their justification is imperfect.

DR. WEINSTEIN: In blood safety, the old adage "The good should not be the enemy of the perfect" applies, well, I suppose I think for some of them, and maybe it isn't the thing. However, we do know that the cost of using blood products is rising faster than the costs of many other things. If readjustments annually to the cost of inpatient care is based on the market basket of inpatient services, given the fact that blood products are such a small part of that budget, they are not going to be noticed and they won't be adjusted at an appropriate rate compared to their increase in cost.

So it seems to me that whatever the final proposal or legislative fix looks like, it should take into account looking separately at blood inflation, even on the inpatient side. Now, that could mean redirecting dollars or appropriating new dollars, but it should be looked at separately, because it happens differently.

Finally, along those lines, there is a potential fix to this DRG tweak that may make some sense. As many of you know, there is a major effort afoot to assign Level 2 codes to all blood products and derivatives, particularly the new ones and the expensive ones, leukoreduced red cells, (inaudible) plasma, delay release plasma. How often these things are used, how much they cost, how much hospitals depend on them, it's hard to capture just now.

But if this coding initiative, which has been supported by AABB, ASFA, and ASH in writing to HCFA, if this is also supported by this committee and it does--it seems that they are favorably inclined toward it, but a decision still has to be made--if this goes through and we have codes for everything, and if hospitals are made to use those codes, we will know what hospitals are spending on blood products, and in fact if more money is appropriated, we will know if it is being spent on blood products it is meant to purchase. And I think this committee, at least I would like to see this committee support that initiative, which I think will help solve another major problem.

I have probably stolen more than the 3 minutes I was allotted, but I thank you for your attention, and if you have questions I will be happy to answer them.

DR. CAPLAN: Questions?

[No response.]

(Inaudible.) All right. Why don't we take our lunch break for roughly one hour, be back here at 2:05, we will say, and then we will see if there are any recommendations in the reimbursement compensation area.

[Whereupon, at 1:15 p.m., the committee adjourned, to reconvene at 2:05 p.m.]

AFTERNOON SESSION

2:24 p.m.

DR. CAPLAN: While I am calling the meeting back to order, Steve Nightingale promises me that he will be doing nothing but finding 12-year-olds to collaborate with him on the web site for the Advisory Committee to update folks on what we have done and to include a variety of documents that are of interest to the committee. (Inaudible) the web site (inaudible), can we change it to make it user-friendly?

DR. NIGHTINGALE: Yes, the web site is changed. I do not say it is more user-friendly at this point, and I will simply comment that some things have been easier to do here than others. If I were to prepare a (inaudible) lookback at the web site, I would find whether they were (inaudible) each other.

It is my hope that by the end of the day, that I will have addressed the resolution of (inaudible) on this web side, as well as all the recommendations of the Advisory Committee in the (inaudible). I want to address (inaudible) as the most (inaudible) web master.

DR. CAPLAN: We're thinking of a one-day meeting in August, around the 25th, I believe it is a Thursday. Is that right, Steve?

DR. NIGHTINGALE: Thursday.

DR. CAPLAN: Thursday, and what we have in mind for the agenda for that, at least talking among ourselves at lunch, is going through and updating and following up on some of the resolutions that we passed to (inaudible) to the Secretary and other agencies and Congress, and basically checking in to see what is going on, how things are going with respect to things that we have asked to happen. I think we will probably also try to create, that Wednesday night, some sort of a social event, and so you should come prepared to party. And we will give you instructions about that, but it's probably going to be a Wednesday night, Thursday meeting, so that makes it the 24th, Wednesday, 25th, Thursday.

VOICE: (Inaudible.)

DR. CAPLAN: 23rd, Wednesday, 24th Thursday, (inaudible).

VOICE: Just one day?

DR. CAPLAN: Just one day. Well, one day of--okay. Now, the plan for the rest of today is to ask for resolutions that are forthcoming from the Advisory Committee. We're going to hear a little bit from Dr. Emmanuel from WHO about international collaboration on safety and availability of blood, what's going on worldwide. We'll probably have time for a couple of updates on blood availability and plasma availability. That should do it. So let's try and be expeditious in our resolution work, and let me see, would anyone care to put forward--

DR. PILIAVIN: Jim and I have a proposed resolution. We are plugging in the laptop. We may need to make the font bigger.

DR. CAPLAN: I'm for that.

DR. NIGHTINGALE: Is this the most recent thing for what used to be IPPIA?

[Laughter.]

DR. AuBUCHAN: Shall I read it for the record?

DR. CAPLAN: Yes, go ahead.

DR. AuBUCHAN: The committee recommends that the Department of Health and Human Services ensure allocation of Medicare funds sufficient to reimburse blood establishments and transfusion services for improvements in transfusion safety that are mandated by the Federal Government or that have been adopted as standards of practice. In the absence of such assurances, this committee is unwilling to endorse the implementation of universal leukoreduction (inaudible) further information or discussion.

DR. CAPLAN: Do you want to break those into two, if I might suggest?

DR. PILIAVIN: I want it as a unit. I'm not sure Jim is as strong on that as I am.

DR. AuBUCHAN: I will stand with my (inaudible).

DR. CAPLAN: All right. Let's discuss this proposed resolution.

MS. LIPTON: Well, I don't want (inaudible). I mean, I think I really would like to address the reimbursement issue, but that if there's a problem with leukoreduction, I don't think it's our role to say we're not going to--we're not being asked to do one thing or the other on leukoreduction today.

Just in terms of the language, I would suggest that what we're really talking is not an allocation of Medicare funds, because there are Medicare funds, and an allocation just means that we're asking to divide it up differently. What we really want is additional funds. We want incremental funds. We want it specifically for inpatients. So I think what we want to talk about is language like "fair Medicare reimbursement for inpatient blood-related therapies."

Those are my comments.

DR. AuBUCHAN: The choice of the word "allocation" was one that was conscious, because we are of the understanding that the Federal Government also only has so much money to spend on health care. There is only so much money in the Medicare reimbursement system.

However, Jay and I believe that if the Federal Government feels that something is so important that it needs to be mandated, that it should be so important that the Federal Government is willing to pay for it.

MS. LIPTON: But it all comes out of the Medicare Trust Fund. It comes out of already allocated dollars. I'm just talking to you from a technical standpoint. The use of the word "allocation" is self-defeating. If we want additional money, we don't allocate Medicare funds. All we're doing then is taking the same pie and cutting the pieces differently to the hospital.

DR. AuBUCHAN: I understand that. This was a different issue than assuring adequate funding for blood services. This is directed at increasing funds commensurate with increased benefits.

MS. LIPTON: And again my point is that the use of the word "allocation" will not do that. I mean, there are others out here who are more familiar with Medicare reimbursement, but this is this discussion we have been having over the past few weeks about using allocation of funds as opposed to additional monies.

DR. CAPLAN: Jim, did you and your conspirator intend this to be a statement about leukocyte reduction in particular, or do you want to try for a statement about funding, "Don't establish any unfunded mandates," in the language of (inaudible)--

DR. AuBUCHAN: I would say it was driven by our concerns about leukocyte reduction, but that isn't the only mandate for improvement in transmission safety, but it is (inaudible) now.

DR. PILIAVIN: Well, my feeling is that it provides us with a certain amount of leverage, since it's new, it hasn't been put into operation yet. There was considerable doubt expressed in this group about the value of it in a cost-benefit context. So we're trying to put the cost-benefit concerns in here, and I personally am totally unwilling to lend my voice in support of doing this unless there is an assurance that it's not going to be, as you cited, an unfunded mandate (inaudible).

DR. CAPLAN: See, I mean one where you can change the (inaudible) resolution and say, "We believe that we should not have unfunded mandates about safety without an assurance of increased funding," which (inaudible) to endorse leukocytes. I mean, just do it as a statement (inaudible) the issue of inpatient reimbursements for safety generally. What I think you're trying to drive at, and I think you're both (inaudible), is without assurance of increased funding for this particular policy, we're unwilling to endorse it.

DR. GILCHER: Are we unwilling to endorse it for Nucleic Acid Testing? I don't think it belongs here. I'm opposed to that statement, the second sentence.

MS. LIPTON: And we already have costs associated with leukocyte reduction that we need to worry about. Like I said, it isn't that we're about ready to do something. We are in the middle of something, like it or not.

DR. AuBUCHAN: May I ask how you would like this changed, so we can see if we can kill more than one bird with the stone?

MS. LIPTON: Well, I was just going to suggest that, again, instead of ensuring allocation of Medicare funds, that we just have--we say recommend that the Secretary and Congress support legislation--because it has to be legislation. We can't go back to the Department on this. We're not going to get any additional dollars. So that they support legislation to ensure fair Medicare reimbursement for inpatient blood-related products and services, or you can say for improvements in transfusion safety that are mandated or (inaudible). I don't care.

I think that specific language that says "We recognize that you have told us we can't do anything further without a legislative fix, and this committee says we support a legislative fix here, " if that's what it takes, that's what we're interested in. And I don't think we tie it specifically to leukoreduction or NAT or anything. What we just say is, when new things come along, we have to be prepared to reimburse those things.

DR. EPSTEIN: I would just like to read a motion from a September '99 meeting of this committee which was passed unanimously, which stated that "The committee recommends that the Secretary work with Congress to seek additional resources to support the introduction and maintenance of mandated blood safety measures." I think that that statement already captured most of this point, and that the key here today is what needs to be done beyond that.

And what I picked up is that we need to say something about the fact that the failure to have accomplished designation of funding to reimburse blood safety measures for inpatient (inaudible) needs to be addressed, and something along the lines that given the disparity in the rate of cost inflation of blood products compared to medical services in general, the committee recommends that HCFA consider inflationary indexing for this category of products and services.

MS. LIPTON: Annual updates of these inpatient (inaudible)--

DR. EPSTEIN: Right.

MS. LIPTON: --so that you can make sure that every year they have to go back and take a look at the cost of blood and fold that then into the DRG figures.

DR. CAPLAN: Let me just interrupt, because I want to make sure, we haven't discussed this, one type of resolution, when I asked about splitting it, is to form a resolution about reimbursement for inpatient indexing and (inaudible). That's not this. You can try to edit it into that, but it isn't that. If you want to do that, (inaudible).

If you want to say something about leukocyte reduction as an unfunded mandate right now, which is what that looks like to me, that's probably something we could edit, fight over whether it's NAT testing or anything else besides that.

But my suggestion would be, if you want to go first with the reimbursement thing as (inaudible) this one, then we should look for another resolution instead of trying to edit that one, because that isn't what that's going to do. Okay? Okay.

Anybody got a resolution? Let's pull that one down for a second, Jim, and we'll come back. Anybody got a resolution they want to put forward on reimbursement?

DR. PENNER: Can we reaffirm the previous motion, that the--

DR. CAPLAN: Reaffirm the resolution?

DR. PENNER: Reaffirm that motion, and then add on this? Because that gets us the underpinning for what (inaudible).

DR. HOOTS: Yes, I think that's a good preamble, because it says that we haven't changed our mind one bit, and yet this is evidence that what we recommended has not been implemented in this context and we would like to see it implemented in this context.

DR. CAPLAN: Well, how about we try something like this: The committee remains committed to its September 1999 view concerning the necessity for adequate reimbursement for increased measures in safety," (inaudible). "We recognize that some steps have been taken by (inaudible) outpatient. We would like now--we would urge that the problem of inpatient be addressed." And that would give us--I mean, we can do it that way as a preamble.

DR. NIGHTINGALE: May I make a bureaucratic suggestion? The bureaucratic suggestion is, you've got a lot of traffic heading for the Secretary's office already, very dangerous traffic, very sensitive traffic. Don't put too many cars on this train, unless you really want them there. A resolution reaffirming a prior resolution is an empty boxcar. You can do it if you want to. It is not going to accomplish anything.

MS. LIPTON: What I was going to suggest, I mean, still you have to deal with the first two paragraphs. They are introductory, but the heart of it is in the bottom paragraph.

DR. NIGHTINGALE: I'm sorry. My comments were not addressed to the number of resolutions. A couple of years ago we put 20 resolutions up and 19 of them got acted upon. The comment isn't about the number of resolutions, the number of separate ideas. As somebody pointed out earlier, I think it was Dr. Gilcher, there are people who'll read the first sentence of the resolution and go from there, and they are busy people, and you're going to be having to get the attention of busy people.

DR. CAPLAN: So, Karen, why don't you read what you would like us to (inaudible) here?

MS. LIPTON: As I said, that being the case, if we could skip the top two paragraphs. "So, whereas the Advisory Committee on Blood Safety and Availability is dedicated to ensuring patient access to safe blood products and services, whereas the committee recognizes that fair and timely Medicare reimbursement for blood-related therapy is critical to ensuring patient access, the Advisory Committee on Blood Safety and Availability recommends that the Secretary and Congress support legislation to ensure fair Medicare reimbursement for inpatient blood-related products and services. Such legislation should provide sufficient funding to account for increased blood-related costs, including those associated with new blood safety measures, and require that these costs be reflected in annual updates of inpatient diagnosis related groups. We urge the diagnostic (inaudible)."

DR. NIGHTINGALE: That wording will get a careful hearing.

DR. CAPLAN: Discussion? Karen, does that language at the end there reflect any updates? Does that capture indexing?

MS. LIPTON: It can be accomplished through indexing. That's one mechanism of doing it. They could do it another way, but it forces a look at blood separate from, you know, just the general DRGs. We've pointed out we're so tiny sometimes in comparison, that when we go up, what happens is we get stuck into those other costs, and we want people to focus on the cost of blood that's going up and what we're doing, no matter what it's related to.

DR. HAAS: Art?

DR. CAPLAN: Yes?

DR. HAAS: I think the part that's really important in Karen's statement up there is that she's referring specifically to new blood safety measures, and I think we want to be careful to stay away from an indexing concept, because the indexing is relating to a much broader area, and what's happening in the blood area are the mandates. And the review needs to be around the new mandates, not because the general inflationary index went up a little bit. So that language is, I think, very important.

DR. CAPLAN: Larry?

MR. ALLEN: I think what some of the speakers before lunch were saying is that some of these therapies are miscategorized, and part of our language in one of the earlier recommendations acknowledges that fact, and I would like to see some of that added in, so that we can get more specific on some of those treatments. I think that's what's happened in the past. The recommendation did go through, but there were certain categories, or certain treatments, rather, that didn't end up in the right categories.

DR. CAPLAN: Yeah, Steve?

DR. NIGHTINGALE: Art, along those lines, I would like to ask Dr. Weinstein, who I see is still in the audience, to comment if he wishes on whether or not this, the language on the screen up here addresses the concerns that he so eloquently raised a few moments ago.

DR. WEINSTEIN: Thank you, Dr. Nightingale.

I believe that the language on the screen speaks to one of the three major points that I made earlier. It doesn't go as far as to propose something related to level two coding or another mechanism of capturing the data to insure that the funds are truly allocated for blood products. But I mean--and maybe it shouldn't.

DR. NIGHTINGALE: I would actually like you to stay here, because this helps me address a point that is important to me, which is that this Advisory Committee is not structured in such a way within the government that it can immediately address issues that are dealt with at the agency level. For example, this Committee is not structured to address scientific issues that go to Food and Drug, and we intentionally do not discuss them. This Committee is not situated to address very specific reimbursement issues that would go to HCFA. And I made this comment in response to a comment of a previous speaker. I wanted to take this opportunity to emphasize that while we are not unaware, or in some cases at least, by no means unsympathetic to the concerns that are raised, but it is not the role of this Committee to micro manage every aspect, the details of HCFA's reimbursement, and there are some issues that need to go to HCFA directly and some that are of broader interest, and I realize that this addresses one and not the other too. So I guess I brought you up to say what you just said.

DR. CAPLAN: Maybe we can add the word, on the second paragraph, "Whereas the Committee recognizes that fair, accurate and timely Medicare reimbursement." That at least opens the door for revisiting what you're talking about in a way that says "let's make sure we're coding correctly."

DR. WEINSTEIN: I wouldn't presume to dictate to the Committee what the language should be. And I must say, I don't really know what is the Committee's tradition with respect to appropriate wording of resolutions. I mean, I think I made one remedy suggestion.

DR. NIGHTINGALE: No, that's very good, because that gives me an opportunity to try to explain it to the public at large. It is inappropriate for a committee that directly advises the Secretary to deal with matters that are of such technical complexity that one would not expect the Secretary and her immediate staff to have sufficient expertise to advise the Secretary. There's a reason why we delegate things, and it's not because we want to go home early; it's because there's only so much you can do accurately at one level. So what we're trying to do is express, as I said, the sympathy for the issues that you raised, and at the same time point out that this is not the most appropriate place to raise all three of them, but perhaps was the place to raise one of them.

DR. WEINSTEIN: I appreciate that.

DR. CAPLAN: One other issue that I see here that's a problem. Way back before the lunch break--it's nice to get Medicare reimbursement, but I think we heard a complaint about this issue from DOD. There are other funders in the loop here about adequate reimbursement, so we'll probably need a support legislation to include fair reimbursement including Medicare or not limited to Medicare or something.

DR. BUSCH: Thank you. I can't ask for that directly.

[Laughter.]

MS. LIPTON: Well, we can ask for it on your behalf. That's a nice amendment.

DR. CAPLAN: But I mean some language there that I'll try, "To insure fair reimbursement, including Medicare." Are you making that--do we have a little squeegee pen there?

CAPT. McMURTRY: Didn't bring one.

DR. CAPLAN: Oh, you didn't bring one. Well, this resolution will have to pass. Can't edit it.

[Laughter.]

DR. CAPLAN: All right. Steve, if you just make notes on these small--we added the word "accurate", I believe, in the second paragraph, and added the word, "insure fair reimbursement including Medicare", or "such as Medicare." I don't care which.

Other comments?

MR. ALLEN: I just wanted to read something from one of our other recommendations. "Whereas the proposed reimbursement payment for therapies under APC 369, which involve the administration of human derived biologicals including blood components, is inadequate and miscategorized, and therefore threaten patient access to quality and potential life-saving blood-related therapies." Is there some way that we can tie a little bit of that into this statement? I know that we can't--as Steve said, we can't be very specific about the therapies, but at least we can, maybe at some point add on, or allow others to add on specific therapies.

DR. CAPLAN: One way to do that is a simple one-sentence thing in the third paragraph that says, "And consistent with our earlier recommendations", so that will give a tie-back to. You can just point at it, because it's there, what Jay quoted, what you've got. Those are all targeting that way. You know what I mean? Just add that "And consistent with."

DR. NIGHTINGALE: The Advisory Committee may pass whatever recommendation it wishes. I'm trying to inform the Advisory Committee that there are limited tools available to me to go to the Secretary and get the Secretary to say down here on page 741, "I want you to change this 1 to a 2." That is not the best way to run a department the size of Health and Human Services.

DR. CAPLAN: Okay. So if you were on the other side and looking to get some of these therapies--the reimbursement figures changed, how do you go about that?

DR. NIGHTINGALE: I think by getting a general statement at the top on fair and accurate reimbursement, and by noting in the record that the concerns of the public commenters were recognized, and that they were advised in turn to present their concerns directly to the agency responsible for implementing the decisions about where they have concerns.

DR. BUSCH: Yes, two comments. One, if you're going to remove "Medicare" from that third paragraph, you probably want to remove it also from the second where it says, "timely Medicare reimbursement." And, Steve, whether it will be of any political value to you for the Committee to formally state that we recognize the great progress that's been achieved with respect to outpatient reimbursement through your efforts, kind of adding a third paragraph.

DR. NIGHTINGALE: Actually, no. I think in--I very much appreciate that comment, but the best response I can give you is that there is a very widespread feeling within the Department of Health and Human Services, once the debate over outpatient prospective payment was concluded, that we did the right thing. There are people with very different economic views are pleased with that document.

DR. CAPLAN: All right. How about a motion on this one?

DR. AuBUCHON: So moved.

DR. CAPLAN: Second?

DR. PENNER: Second.

DR. CAPLAN: Discussion?

[No response.]

DR. CAPLAN: All right. All in favor of what Steve is about to read, if he can do it with those little inserts--you can just say "inserted", "inserted."

DR. NIGHTINGALE: "Whereas the Advisory Committee on Blood Safety and Availability is dedicated to insuring patient access to safe blood products and services, and whereas the Committee recognizes that fair and timely reimbursement, including Medicare, for blood-related therapies is critical to insuring patient access to the safest possible blood, the Advisory Committee on Blood Safety and Availability recommends that the Secretary and Congress support legislation to insure fair and accurate reimbursement for inpatient blood-related products and services. Such legislation should provide sufficient funding to account for increased blood-related costs, including those associated with new blood-safety measures that require that these costs be reflected in annual updates of inpatient diagnosis related groups."

DR. EPSTEIN: I thought we had agreed to say "fair, accurate and timely", and that we had agreed the statement, "and consistent with its previous recommendations, the Advisory Committee recommends."

DR. NIGHTINGALE: I'm sorry if that was the sense that that was what I intended to say.

[Laughter.]

DR. CAPLAN: You just said it very quickly.

DR. EPSTEIN: Glad you said it.

DR. CAPLAN: All right. So the word "accurate" will be in there. There is a sentence that says "consistent with earlier recommendations."

DR. PILIAVIN: Art, wasn't Steve trying to suggest that a couple of preamble paragraphs would be less effective than without them, and I was wondering whether we wanted to get rid of those preamble paragraphs?

DR. NIGHTINGALE: No. I'm sorry. Steve was not suggesting--that was not what Steve wished to suggest. What I wished to suggest was three substantive resolutions and one fluffy resolution are not as good as three substantive resolutions.

DR. PILIAVIN: Okay. Well, is it your opinion that those two preamble paragraphs make it seem fluffier?

DR. NIGHTINGALE: No. I think a fluffy resolution would be we reiterate a resolution we made 6 months ago, appearances perhaps to the contrary, we have not forgotten those.

DR. CAPLAN: Reiterating resolutions that are made 6 months ago is not fluffy; it's being apparent.

Okay. All in favor with these emendations?

[Show of hands.]

DR. CAPLAN: Opposed?

[No response.]

DR. CAPLAN: Abstained?

[No response.]

DR. NIGHTINGALE: I see no opposition, and as in the previous votes, I see no abstentions. Unanimously passed.

DR. CAPLAN: All right. We have one resolution that's still out there. We can return to that. Are there others, by the way, before I head back there? John, why don't you tell us what you've got, and then we can see the order here.

MR. WALSH: I've got one related to the outpatient prospective payment follow-up, and I have it on a disk if we can just put it--can't do it? Okay. It's the only file in Word.

And I already deleted the preamble based on Dr. Nightingale's insightful remarks.

[Laughter.]

MR. WALSH: But I don't think it's--it's probably not necessary, whereas, we thank HCFA for doing the right thing. At any rate, Mack will put it up, but I'll read it, and Danny Kuhn and I collaborated on this, and there's a precedent set where, Danny, you might want to just give a little background on, that we think necessitates the Committee asking HCFA to take--asking HCFA to take specific follow-up action and notify all HCFA outlets of the change in the regs, and actually collect the data to be able to support the utilization and the pricing or billing information relevant to plasma derivatives.

In better words it's as follows--are you able to get it, Mack?

"The Committee recommends that HCFA promptly distribute guidelines for coding and billing of blood and plasma products to all entities covered by the outpatient prospective payment rule. Furthermore, the Committee urges HCFA to work with stakeholders, including consumers, outpatient departments and manufacturers to devise and implement a data collection system to capture actual utilization and billing data to be used to establish a permanent payment system for blood derivatives administered in outpatient settings."

And without collecting that data, we don't have permanent pricing.

DR. KUHN: I'll second it.

But I'll also make a comment on it. I think Mr. Jackman referred to it before. In the past, the hemophilia population had a temporary cost pass-through bordering on a final rule, and there was not enough information, so we've had to go back to Congress and try to lobby again for it. I think this would prevent that from happening, and also be able to probably establish a good precedent in being able to collect that data, needing to know exactly how to reimburse things.

[Pause.]

DR. CAPLAN: It kind of makes you long for the days of the overhead, doesn't it?

[Laughter.]

DR. CAPLAN: Very good. All right, comment?

[No response.]

DR. CAPLAN: Motions? I need a motion here.

MR. WALSH: Sorry. I move it.

DR. KUHN: Second.

DR. CAPLAN: Kuhn seconds. All right, discussion.

[No response.]

DR. CAPLAN: All in favor?

[Show of hands.]

DR. CAPLAN: Opposed?

[No response.]

DR. CAPLAN: Abstained?

[No response.]

DR. NIGHTINGALE: I see no votes against. I see no abstentions. As previously, the motion approved unanimously.

DR. CAPLAN: Are there any other resolutions besides the Piliavin/AuBuchon still out there? Okay. Let's go back to Jim's then.

DR. AuBUCHON: Some may think that the resolution that Jane and I proposed is now unnecessary, given what we have passed in terms of looking for increased reimbursement through legislation. While I certainly supported that previous motion, I don't think it addresses the entire problem, and I think that our motion dealing specifically with allocation will still be helpful.

Obviously, if more money is put into the system, then the--our motion is moot. However, even if no money is--additional money is put into the system, and the Medicare pie is simply split up differently, this will still aid hospital transfusion services in being able to go to their hospital administration and saying that, "We want to leukocyte reduce", and the federal government is recognizing that this costs more money.

In addition, private payers do indeed pay a lot of attention to how Medicare structures their payments, and if Medicare states that they will support a greater allocation of funds to blood components to recognize safety improvements, then for the half of blood that is paid for outside the Medicare system, the hospitals should be seeing greater reimbursement.

Therefore, I think our previous motion is still germane.

DR. CHAMBERLAND: Is it possible to see it again?

DR. CAPLAN: Mack, are you coming out there now so he can put his back up?

CAPT. McMURTRY: Jane wanted to copy it down.

DR. CAPLAN: Oh, you're still writing it down, okay. That's all right. We can get that. It's okay. You can shut off and put Jim's back up, Jim and Jane.

[Pause.]

DR. CAPLAN: All right.

DR. GILCHER: Dr. Caplan?

DR. CAPLAN: Ron?

DR. GILCHER: I am not in agreement with the second sentence. I feel that it is holding legislators hostage. I think it's tying it to one issue. In the absence of such assurances, this Committee is unwilling to endorse additional safety measures, but not tied to one measure. I can't support it in its present form.

DR. CAPLAN: Keith?

DR. HOOTS: I wonder if you could rephrase this in a positive rather than a negative sort of leverage statement, like, "The Committee would like to discuss more completely the impact", or something to that effect, "of leukodepletion on overall blood safety in relationship to cost before endorsing" or whatever, rather than--I see where Ron's coming from, and I'm concerned about that too. I don't think we've ever passed a recommendation that is quite so "if then", and particularly where we were the ones in the then statement that would then act, and I don't know that we want to get into that box.

DR. GILCHER: And we have another safety measure, namely nucleic acid testing, which is also really part of this as well.

DR. CAPLAN: Jay?

DR. EPSTEIN: I am very sympathetic to the movement toward seeking to review costly measures in terms of their cost effectiveness. I think that that is a larger issue than leukoreduction. Leukoreduction presents itself in that way, but you know, so do policies on precautionary measures for CJD and new-variant CJD, and so may many things coming down the road. And I would rather see no specific comment on leukoreduction, although I accept that it's the sense of the Committee that the Committee would like to hear about the cost effectiveness of universal leukoreduction, that would then be a request to the Secretary to put it on an agenda.

But I think that there's a deeper issue which is within the scope of the Committee, which is to examine the role of cost--economic assessments in decision making related to blood safety and availability. This was among the topics that had been raised very, very early on in the Committee's history as something that should come to light, and I just think that we've come to that point, and I certainly endorse that discussion. I just think that a declarative statement on leukoreduction at this time is problematic because it would be in a way preempting the whole question of whether we're changing our decisional paradigms. You know, we can't reinvent everything at once.

So I just think that if the Committee wishes to state that it would like to hear discussion of the cost effectiveness of universal leukoreduction, I think that would be fine, and I think to express the sense that the whole issue of cost effectiveness needs to come as an agenda topic, would be appropriate.

DR. AuBUCHON: I've tried to capture several comments. If I haven't done that correctly, please let me know.

MS. LIPTON: You know, I just don't want to be so negative. The Committee is unwilling--I mean, I just--I don't speak like that, and I don't want to speak like that as the Committee. I think the issue of saying that, as Jay said, you know, we would like, "The Committee would like to explore the cost benefit of certain things" is fine. This just sounds like you're just throwing down a gauntlet, and I don't think we need to do that. I think we need to be concerned--let me put it a different way. If cost were not an issue, would leukoreduction be an issue? If we had money to do this, would anybody oppose saying, "Let's do it?" And so I think we get ourselves into a bad situation, you know, when we're saying--what we need to be talking about is--

DR. CAPLAN: I might, if it really could do any good. I mean, one of the arguments here is you're throwing resources in, even if you could get them, for something that's not the most effective expenditure. So I might. But I know where you're coming from. It's just--yeah, Jane?

DR. PILIAVIN: As the author of this sentence, I wrote it that way in order to get people's attention, because I am--and I'm glad to hear Art say the same thing--I am extraordinarily concerned at the constant throwing of money at trying to accomplish smaller and smaller reductions in an already extraordinary, safe system, when we spent all of yesterday and part of this morning talking about errors and accidents in a system which--as we all know at this point--account for far more morbidity and mortality than the sorts of things we're talking about here.

And beyond that, I am very concerned about the lack of attention in general to cost benefit issues in the entire health care system. I am very, very worried about the fact that we as a committee have a purview over a very small segment of the medical area, and are able to make recommendations to the Secretary only concerning that small area. And I, for one, think there should be a committee like this looking at the entire health care system and trying to do things that make a lot more sense in terms of the use of our limited resources. I keep thinking about all the kids who don't get immunized against diseases at a fraction of the cost of what we're talking about here for this leukoreduction process that has not really been demonstrated to be universally useful. I mean, it's obviously useful to some patients or it wouldn't be used in 30 percent of the cases now, but I'm just appalled at the way that the decisions about resources are so compartmentalized, and I just wanted to draw attention to this one decision in this context, and try to get people to think a little bit more broadly about what we're spending our money on.

DR. CAPLAN: Do we want a resolution in the spirit of what's been discussed, not to change the one that's up there, but maybe a slightly different one that says something like, "The Committee would like the Secretary to address issues of cost benefit as they apply to new safety measures such as leukoreduction and NAT, and core antibody", and whatever else anybody can think of. I mean, is that where we're sort of stumbling toward, is a request to say, "Let us get into", because they haven't asked us to do it. I mean, she didn't ask us yet. Do we want to ask for that and have that given to us?

DR. DAVEY: I like that last one a little better. I don't even--sure we need the "such as." A period after "measures" may be adequate.

DR. HOOTS: I think it would be good to have at least--I mean, because generally it's kind of a one-way dialog--well, not exactly, because Steve is our communicator to HHS, but essentially we look at issues that are deemed appropriate by the Secretary in HHS, but in the course of doing this, we obviously uncover things like this that we think are very important, and I think we might be remiss if we didn't at least alert the Secretary of the fact, and that we think this should be looked at and that we're willing to look at.

DR. NIGHTINGALE: If I could make a couple of responses to Jane. I think one of the challenges of a public servant is to respond to the substantive content of a public statement, rather than the framework in which it comes. I wouldn't profess that we always do that, but that really is a big part of our responsibility. So I don't think that whether this comes in a gauntlet or otherwise should affect the way that we respond to it. Some of the best suggestions that you get sometimes come from a clenched fist in the street. And if the government is not aware of that possibility, the government's not doing its job right and may suffer as a consequence of it.

I'd also like to suggest that I respectfully disagree with Jane on the subject of the small purview of blood in the health care system. As I count it, there's about 2 billion spent every year on red cells, and the better part of 3 billion spent on plasma derivatives. That's 5 billion, and I count it, about 1 trillion is spent on health. So we're about 5 percent of the health care budget. That is not a trivial sector by any means, and it's also a sector because it's so--I want to say well-controlled, tightly-controlled, and so much in the public eye it provides an opportunity for the public, through its government, to try out new things and see if they work. I think that's in fact what we're doing with the outpatient prospective payment system. I have heard that we don't have time, but I've also heard we don't have data.

The bottom line here is that this is not an insignificant part of the health care system by any means, so I encourage you to look carefully at it, and if you think you can only get somebody's attention with a gauntlet, use the gauntlet. However, you've gotten our attention. You don't need the gauntlet. You need a very strong argument.

DR. CAPLAN: Mike?

DR. BUSCH: I think this discussion is a good complement to the prior proposal because we've said before that we want appropriate reimbursement for safety and issues. Now we're sort of saying we also think that the process, the decision-making process that leads to introduction of safety initiatives, needs to be better defined. And it's sort of saying we're taking--in addition, we'd like to be given--or participate in the responsibility of developing a more appropriate framework for making these safety decisions that will drive these increased costs.

DR. CAPLAN: This is a crude phrasing, but I just want to toss it out there while Jim edits along. What I tried to come up with was--and I know Karen talks this way--"The Committee respectfully requests that we receive more information on the role played by cost benefit information and decisions to introduce new safety measures for our review and comment."

DR. AuBUCHON: Could you please repeat that?

DR. CAPLAN: "The Committee"--I can't even say this without--"The Committee respectfully requests"--I don't talk this way--"that we receive more information on the role played by cost benefit information and decisions to introduce new safety measures for our review and comment."

DR. NIGHTINGALE: Art, what possible response would you anticipate to that question?

DR. CAPLAN: In other words, she starts--then she comes back and says to us, "Here's how we took into account cost and benefit in thinking about leukoreduction or NAT or whatever it is." What do you do with that? How are you doing that? What roles are playing at HHS?

DR. NIGHTINGALE: I think that would be an extremely difficult question to answer, and since I would be the person who would have to draft that answer--

[Laughter.]

DR. NIGHTINGALE: --I haven't a clue as to how I would answer it. I think that the way I would answer it would be to refer the Committee to the published transcripts of this and other meetings. If I wanted to give you a short and real--we try very hard to conduct a democratic process. We also try very hard to respect the views of the minority in a democratic process. We try very hard to identify a solution that will, as the first approximately, serve the greatest good for the greatest number, but we realize that that rather simplistic political philosophy is inadequate at times to address the concerns of the population.

MS. LIPTON: I was going to suggest rather than saying we want to know how the process works, I mean, is it inappropriate to say with respect to new--or just say, "New safety measures, that the Committee would like to receive information on the cost benefit analysis." I mean, either they've done it or haven't. I mean, is that totally out of whack?

DR. NIGHTINGALE: See, actually what's out of whack would be for the Secretary or for the Office of the Secretary to get into a debate on the merits of a particular cost benefit analysis. And we did that with hepatitis-C, and we're not going to do that again.

DR. CAPLAN: Could the Secretary gather or direct the reporting on? I mean, she's not going to do it, but--

DR. NIGHTINGALE: No, I think there is, to me, a fundamental limitation. It's not a flaw. It's a limitation of cost benefit and cost effectiveness analysis. And these are--and the limitation is its strength. These are very effective ways. They are very quantitative ways of describing an argument in favor of a particular position. What they let you do is just say is, "If you accept these premises, you will come to this conclusion." The issue is not the process itself, but whether or not you agree with the premises, and the premises that we have disagreed on in time, are whether or not in particular the market accounts for the true cost of various health care initiatives, and there is a feeling among many--and not just me--that the market does not account for the true cost of many health care initiatives.

I could--I mean, the Secretary might, if she turned her attention to this for a long period of time, say something different, but I think that's a very conventional middle-of-the-road response to the questions being raised. We don't disrespect cost benefit analysis, but we sometimes, when our telephone rings, realize that there are things in some people's analysis but not in others, and it's not our role to get caught in a fight over whether you should include this or that factor.

DR. CAPLAN: I think what's happened is, or what I think the Committee is trying to struggle toward--and I may not have it here--is some understanding that cost benefit information has not been adequately collected--however it's used in driving the policy--on new safety measures. When Gerry Sandler says he gets 200 units for whatever reason of something he doesn't want at high cost, that's bad. So something's wrong here is what I would say.

And so what it seems to me needs some address then is the idea that the Committee is concerned that inadequate attention has been paid to the role of cost and benefit in introducing new safety measures, and seeks more information about that, something like that. And that's where we're heading.

DR. HAAS: Want to back up to the--I guess, Jim, you're working this thing? Back it up a little bit to the one above "The Committee respectfully", after you get finished writing?

DR. AuBUCHON: Yes. So you want to go up?

DR. HAAS: Yes.

DR. AuBUCHON: Tell me when to stop.

DR. HAAS: I thought it was the one right--no, it was--

DR. AuBUCHON: I lost--I must have lost--

DR. HAAS: Oh, you must have lost one I was looking at. There was one that said something about "We should do"--in terms of just hearing more information, which was in the spirit, I think, of what Art was talking about, and I thought we could work right off it, but if we've lost it, we can go back to the one--

DR. AuBUCHON: Tell me where.

DR. CAPLAN: All right. Let's slide back to the inferior versions that we're struggling around with here. This will teach anybody to bring their computer. You wind up being the secretary. Mary.

DR. CHAMBERLAND: My sense of this is, of all of the discussion, is that the Committee is concerned that there has been inadequate opportunity to have a public discussion or dialog about costs and benefits of new blood safety measures.

The FDA--correct me if I'm wrong--but my understanding is that the FDA is really prohibited from consideration of cost, similar to what NIOSH is in--excuse me--OSHA in implementing regulations or requirements. So this really is the venue, as I understand it. I mean, this is the public venue. And essentially we're--I think we're wanting--it's almost not a recommendation. I think the Committee is sort of struggling with we'd like this to be our next major agenda item, and I thought it was a little obtuse the way we got there. One of these recommendations was, "The Committee requests the Secretary to address cost benefit, and as I understood it, somehow the response was going to be that the Secretary would say, "You're right, and we'd like it to be you to address it." Because there are no cost benefit data. I mean, it's not as if the department is sitting on cost benefit analyses or that they do these. I have to assume that for some of these issues there is some literature out there or studies in progress, et cetera, and that those could be, as we do for other agenda items, brought to the attention of the Committee and weighed in in our discussions. I mean, if it was that straightforward, this would have been easy.

And so I think the BPAC, for the example of universal leukoreduction, couldn't consider cost at all, and I remember specifically John Boyle, one of the BPAC Committee members, saying to the effect, "I'm voting for this based on my assessment of the general benefit of leukoreduction." But when you add cost into the equation, that will really, really change the discussion, and that's going to be a really tough one to struggle with. And I get the sense from some of the Committee members, maybe not all, that they feel there hasn't been a venue or an opportunity to dialog that out. That's what I'm hearing, and maybe I didn't get it right.

DR. NIGHTINGALE: Yeah. And I would like to support what Mary just said. I'm trying to figure out how to answer this question. I think the answer that I hope I would have come with was the one that Mary articulated. This is the place where those issues have been addressed. I think the Committee is concerned that they feel they were bypassed on the British donor deferral. I've given the reasons why we agree to disagree, and perhaps on other issues. But nobody is trying to suppress a cost benefit analysis, but I can assure you that cost benefit analyses have not in broad areas of medicine, been the high trump in any game that decided a political outcome.

DR. CHAMBERLAND: I think there was some hope at the time this Committee was conceived, that it would be willing to really deal with the tough, dicey issues, that there wouldn't just be a knee-jerk reflex. If it increases safety, we're going for it, that this Committee was really willing to see that there might have to be some tradeoffs, and that while you might want the--you know, our whole goal is the safest blood supply that we can get, but we have to at some point--as I think it was Jane said--these incremental additions to an already safe blood supply are now coming at extraordinary cost and maybe to the exclusion of other things from the broader public health picture.

So I just--I got the sense from the get-go that that's what this Committee was going to try and be ready to do that.

DR. CAPLAN: Well, let me add in this spirit then. Maybe we don't have to tell the Secretary anything. Maybe we have to tell Steve something.

Steve, the Committee is concerned that there's been adequate discussion of the costs and benefits of new blood safety initiatives. Could we talk about that?

DR. NIGHTINGALE: I think we most certainly could, and I think those of you who had examined the color and the quantity of my hair today and for the day that I arrived on this job, will see some objective data that our discussions have not ducked the big issues.

I would like, though, to introduce an idea that I briefly introduced at the Geneva meeting, and I think it's an important idea.

DR. NIGHTINGALE: There are times when dispassionate discussion of intense political debate is very difficult to hold. When we start--when I showed up the day of the second meeting of this committee, the blood community was rent. The passions were extraordinary over two exceptionally divisive issues; the Hepatitis C lookback and withdrawals for CJD.

This committee has adjudicated those issues, and over the last couple of years there has been a collegiality that has grown among us and in the world that surrounds us, not only in this room around it, that has made much more objective discussions possible, such as the ones we have had over the last couple of days, and I am very proud of that. And I hope that the committee and the public that has contributed to this committee shares that pride. I happen to think that that has been our signal accomplishment over the last couple of years, has to been to create a venue, no matter how hard. It hasn't quite happened yet. I mean, we can still get into a good food fight here. But at the end of the day, it's much easier for us to get together and try again the next day. And last night was an example of that. I'm not sure that we would have been as successful two years ago as we could. I think that it will be much more--it is getting progressively easier for us to accommodate divergence of views, and I hope that that process continues.

DR. HOOTS: At the risk of being verbose, I am just trying to frame how we would justify this, and I will just throw it out very quickly. It's a little wordy. The committee, in assuming its assigned responsibilities, is to recommend strategies for enhancing blood safety, is also concerned about the impact that the cost of implementing such strategies have on the overall system. This is particularly true when the cost of a new strategy, initially or in the long term, may impact full capacity by competing for finite resources to preserve previously accepted strategies to maximize both safety and availability of blood. Therefore, issues of cost benefit are extensions of our responsibilities.

So it's kind of a statement of fact rather than emotion, I mean, why we are here--I mean, why we think we ought to be involved in this.

DR. EPSTEIN: Well, personally, I would like just a statement, but I think it's entirely premature. This committee has not yet reviewed the whole issue of cost benefit in the context of other decision-making paradigms that have operated. We need to look at the zero-risk mandate. We need to look at the safety at any cost mandate. We need to look at the precautionary principle articulated by the IOM. We need to look at the FDA paradigm of product approval based on safety and efficacy without consideration of cost.

We need to look at all of this and then ask ourselves what's the role of cost benefit. And I am just concerned that any kind of conclusory statement made today totally preempts the discussion and critical review that needs to take place about the decision-making process. I think what everybody feels instinctively is that there is a need to restore a certain kind of balance, that we have been pushing the marginal benefits of safety measures at extreme high cost and that there's a problem being created.

I think that all we need to do at this stage is either be silent, which I could accept, which was Art's last suggestion, or say something along the lines that recognizing the significant economic issues currently affecting the blood system, the committee seeks to review the role of cost-benefit considerations in decision making related to new and existing blood safety measures. That way we are just signalling where we want to go without taking a position on it prematurely. Also, it's briefer than Dr. Hoots's statement.

[Laughter.]

DR. PILIAVIN: Jim did you get that down? I thought that was wonderful.

DR. EPSTEIN: I'll read it again, and I'll pass it down.

Recognizing the significant economic issues currently affecting the blood system, the committee seeks to review the role of cost-benefit considerations in decision making related to new and existing blood safety measures.

DR. CAPLAN: Promise me that if we pass this one, Jane, Jim, that we are not going back to yours, are we? Not that I'm linking them.

DR. PILIAVIN: Jim?

DR. AuBUCHON: Well, my concern is that, although I certainly support what Jay has just suggested here, and I haven't yet completed typing, that if this committee looks at this in--when would our next meeting be after August? January?

DR. NIGHTINGALE: Yes, I anticipate--

DR. AuBUCHON: January. Leukocyte reduction will be done. It will have happened. We are very close, at this point, to not being able to change its course any way.

DR. NIGHTINGALE: That is correct.

DR. AuBUCHON: And at that point, it will be done, and the response of the Federal Government may well be, "Well, it's happening already, so why do we have to put any more money into it? You already seem to be tolerating this." And I think we will have short-circuited a very large-ticket item, discussion of a very expensive item.

DR. PILIAVIN: Is it not possible for us to talk about it in August?

DR. AuBUCHON: Well, I understood that we had already decided what we were going to talk about in August.

DR. NIGHTINGALE: Not necessarily.

DR. PILIAVIN: I would be willing not to push on anything after this resolution if we can talk about this in August.

DR. CAPLAN: Sure. So let's plan on revisiting the case of leukocyte reduction in August, and we'll return to Jay's proposal that we would like to seek to have more of a role.

Discussion there?

DR. HAAS: Art, a nitpick. "Cost benefit" carries a very specific meaning in the economic circles. I think maybe a better term there is the cost-effectiveness language.

DR. NIGHTINGALE: Either way we know what you mean.

DR. HAAS: Right. I'm just thinking that once it gets printed, if someone gets a hold of it, it gets interpreted differently.

DR. NIGHTINGALE: Not going to happen.

DR. CAPLAN: All right. Is benefit broader?

DR. HAAS: Well, it's usually interpreted, at least in economic circles, as much more of a financial aspect of it, and it doesn't get to the broader measures of the benefit, especially. It tends to focus more on the cost side--cost benefit.

DR. NIGHTINGALE: Folks, we're at 3:30 in the afternoon, and we know what you mean, and we're going to do it. So let's do it.

DR. CAPLAN: Okay. Any other comment?

DR. EPSTEIN: I think we need to decide clearly whether the phrase inserted parenthetically should be in the present statement because it does imply the Government sort of restraining action until it has been debated.

DR. NIGHTINGALE: It's not clear to me the fact that an Advisory Committee put a phrase in parenthesis that the Government is either obligated or intends to change its current course. It takes a little more than that to shut the Government down, like a snowstorm.

DR. BUSCH: I was just going to comment that I think the broader issue is the decision-making process itself. Cost effectiveness is just one of the issues. The other big one that I think has influenced a lot of recent decisions is the international political movements that have influenced our considerations in the U.S. Not that we need--I just think in terms of what we need to do in terms of the next meeting, it shouldn't be restricted to cost effectiveness. It should be bringing in all of these other elements that are influencing our decision making.

DR. EPSTEIN: I think the parenthesis should be removed.

DR. NIGHTINGALE: Remove the parenthesis and substitute "various" for cost effectiveness and vote and go?

DR. CAPLAN: Anybody strongly committed to holding that in the parenthesis?

[No response.]

DR. CAPLAN: Okay. Let's pull that. And how about a motion?

DR. PILIAVIN: So moved.

DR. CAPLAN: Second?

DR. HOOTS: Second.

DR. CAPLAN: Discussion?

[No response.]

DR. CAPLAN: Good. All in favor?

[Show of hands.]

DR. CAPLAN: Opposed?

[No response.]

DR. CAPLAN: Abstained?

DR. NIGHTINGALE: No opposed, no abstained. Motion passes unanimously. I will get a copy of it, please.

DR. CAPLAN: Now, here is where we're at. We're going to have, talking about xenophobia, we nest have a presentation--is Dr. Emmanuel still out there? Yes--the World Health Organization, who has come complete with black helicopters to impose intergalactic standards upon us all.

I don't know if you have slides going up there. Dr. Emmanuel, why don't you come up and introduce yourself. He actually has played a crucial role in the Blood Policy Division at the much-admired WHO.

DR. EMMANUEL: Thank you very much, Mr. Chairman, Dr. Nightingale and to the Surgeon General, in his absence, for inviting WHO and myself, in particular, and to all of the colleagues and friends who have helped me in my work in WHO, many of whom are around this table.

It gives me great pleasure to be here, obviously, and I think while one comes to share experiences, one probably learns much more than one shares with other people. I have certainly enjoyed the last few days. And it's surprising how the things that have challenged you in this country in finding the lost infection, and doing the precautionary principle and arguing about cost recovery and what needs to be paid for is a similar thing that happens in all countries of the world, but at a different level.

What I'd like to do today is, the time is short, I'm perhaps going to miss some of the slides. I have got a very able assistant. Thank you very much to Mr. McCurty, to help me for that. I thought I'd just set the scene a little to paint a picture as to why we need this Global Collaboration.

The World Health Day has a birthday on the 7th of April each year to commemorate its founding, and this year it was nominated to be Blood Safety Day. We identified this character because of the lack of an internationally, globally recognized logo for safe blood. If we're moving towards the era of characters instead of the written language, then we hope that this will be the sign that everybody will identify.

Some of the wags in the part of the world where I am working at the moment, not from whence I come, suggested that instead of the caliper holding the globe, it looked like a beaujolais glass slipping over on its side.

[Laughter.]

DR. EMMANUEL: I did remind them that perhaps 99 percent of the world didn't live in that particular part of the world and actually didn't know what beaujolais was or ever had the luxury of drinking out of a glass. And I think nobody else has commented on that since.

So WHO has priorities, and the priorities that have been nominated by the Director General for efficiency savings and for additional funding are blood safety at the top, strengthening health systems to which we contribute to a large degree in making pregnancy safer, noncommunicable diseases, obviously, with all of the thalassemias and the hemophilias and so on, investment in change. I hope I haven't contributed to the lack of mental health in the organization or to food safety, but I can't see our association with that.

So our objectives then were to raise the awareness of blood safety globally, to encourage countries to commit and support to national blood programs. The operative word there is "support." I don't think there's a country in the world that wouldn't say it's committed to blood safety, but what they do about it is quite another thing. And to implement a national legal framework, to promote and retain voluntary, nonremunerated blood donors and to renew and strengthen collaborative partnerships with international and national organizations, that is a major part of our work, which is the Global Collaboration.

Our key message is, therefore, that there is a positive health impact in safe blood, the need for more safe blood donations, the effective and appropriate measures to prevent the transmission of blood-borne diseases and to ensure that there's a safe and appropriate clinical use. We've really got no yardstick by which we can measure proper clinical use of blood. Very many countries claim that there's an inadequate supply of blood. But I think that in many countries there's inappropriate use of blood, which leaves inadequate supply for those who really need it. And you will see why in a moment.

So we had an appropriate, and we think an attractive logo. Dr. Nightingale has some lapel pin badges which he has many of, if any of you would like a lapel badge, and please go up and help yourself. There's also some stickers that we had made with the logo, which can be used on bags or car stickers.

And we have sensitized the public. There was an enormous gathering of people around the world to celebrate World Health Day, and it was judged to be the biggest celebration that we've had on World Health Day. Blood is such an emotional thing, an emotive thing, and is recognized by everybody as of great importance.

We hope that this will lead to a political commitment and support. And that certainly will be the case where we'll be pushing for in developing countries. And we will help assist member states who are committed to the programs to build on this momentum.

So the expected outcomes, therefore, are to increase the public awareness and understanding of blood safety. Hopefully, they will see the signs of "Blood Safety Starts With Me," the need for voluntary, nonremunerated blood donation. We'll increase the awareness amongst clinicians on their role for blood safety and how they can increase this activity. We will reinforce ongoing activities to improve blood safety and availability, and we will build on World Health Day stimulus from this year right through for at least the next 6 years.

Now, how did we come to these strategies that we have developed? We used the facts and figures from our WHO global database, which we have been collecting since 1990. But we renewed them each 2 years. It takes us approximately 2 years to gather this data.

And what we have is WHO has 191 member states. It's the biggest of the UN organizations and is responsible for the health of countries. It has 191 member states and two associate member states. We have managed to get information from 167 countries.

We have used the Human Development Index as a category to divide the countries. And this UNDP Human Development Index, HDI, is divided into low, medium and high on life expectancy, educational attainment, and adjusted income.

So what we have ascertained from these figures is that we know that there are many more than 75 million units of blood collected or donated each year. Of this, only 30 million units of the global blood supply is donated in countries with a low or medium HDI; in other words, the lesser developed countries, more economically restricted countries. The blood donation rate per thousand population is 18 times higher in countries with a high HDI, developed countries. In many parts of Europe, there are donations per thousand of well over 50 per thousand. In many developing countries, it is less than one per thousand.

In this pie chart, with the low HDI accounting for 2 percent, the medium for 37 and the high HDI accounting for the rest. What you can see there with the global population in what used to be blue--it looks a bit green now--compared to the blood, is that we have a figure that if you just looked at the developing world, you would have 80 percent of the blood is used by 20 percent of the world's population.

We know, and we all agree, that regular voluntary, nonremunerated blood donors from low-risk populations are the safest donors. This is particularly true in developing countries, where you have high levels of prevalence of infectious agents such as Hepatitis B, C and HIV. In some countries, the HIV in sub-Saharan Africa is 25 percent of the donor population. In some specific groups, like in antenatal groups, as many as 45 percent of the 25-year-old women are HIV positive. Now, when you consider that the next bullet says that family replacement donors have a higher incidence and they do not have organized blood transfusion services where they recruit from low-risk population groups, they are asking the families to supply the blood. Those very families who come in to supply blood are the infected families.

In addition, it is very rare that a family member can bring in three or four units of blood. They usually, the patient is accompanied by one family member or perhaps two. Therefore, the paid donor, hidden paid donor system, is very high. Clearly, the paid donors are extremely risky donors.

Now, if you look at this figure, it shows you that only 16 percent of the blood supply is donated by the low-risk, voluntary, nonremunerated donors, which means in those countries that I have just spoken about, the risk of infection is extremely high.

And when you look on the right-hand side, of the 30 million donated units in the low and medium HDI countries, 43 percent of that blood is not tested, which accounts for 13 million units of blood per year that is not tested and donated. That's 13 million units.

You also should bear in mind that approximately 600,000 women die in child birth each year, of which one-quarter of those die in associated with blood loss, lack of availability of blood. That is not counting the numbers of women who do get blood transfusions, and they are not tested, and the many millions that are infected as a result of those transfusions. So 17 percent of the global population has access to 60 percent of the blood supply; 80 percent has access to only 20 percent of safe blood.

This is just the organogram of our cluster and the department in the center is my Department of Blood Safety and Clinical Technology. The reason for showing you this is that we have the four teams which are involved in the work that we do, and they are quality and safety of plasma derivatives and other related substances, which are associate with the quality--the one on the right--which are the quality assurance and safety of biologicals and together form and are responsible for the expert committee of biological standards.

Together with Blood Safety, we are responsible for the blood safety activities. But in addition, we also have diagnostic imaging to ensure good X-rays and ultrasonography, proper laboratory testing, clinical laboratory testing, devices, and the clinical technology is district surgical services; in other words, making sure that doctors do the operation correctly, minimize blood loss. And within that framework, we also have safety of injection global network to ensure that there's minimizing nosocomial infection.

Our four main objectives then are policy, advocacy, of course, which means that we try and convince governments to commit and support the projects, quality and safety, making sure the blood is accessible and making sure it's used correctly.

So our blood safety then is a continuum from blood transfusion, with the norms and standards, correct laboratory procedures, diagnostic imaging, and the medical devices, injections, waste management and correct surgical procedures in the district hospital.

We have these areas of work, which is advocacy, to ensure government commitment and support. We encourage safe blood donors. We test and put in appropriate testing strategies, and we provide bulk purchasing for test kits through from simple rapid to ELISA testing and other algorithms to ensure that testing is confirmed, and we have guidelines on clinical use of blood and minimizing our unnecessary transfusions.

We are involved in a new project on quality management systems, where we have already started a training program in Africa where we will train two quality officers in each country. And the first course is taking place where ten countries are involved in this quality management training system to cover the whole area of blood safety. Global collaboration then is our next most important area of work.

So the activities that we have for Global Blood Collaboration are associated with the global database for blood safety. Unless we have the information to identify where the problems are and what the needs are, we can't really address the issues of global collaboration. We are particularly concerned because of the low level of voluntary, nonremunerated donors in the developing world about the education, motivation, retention of safe blood donors.

And we produced learning pictures, and the picture on the right is an innovative distance learning material which is now being produced on a worldwide basis in nine major languages, and we have translated this into Chinese, trained three people from every district in China, and this is being developed into a distance learning program, which the Chinese have legislated for 100,000 technicians to be trained. Clearly, without global collaboration of some kind, we can't do this. This has been emulated in Latin America and Argentina. Mike Busch was there and will be involved in this program.

So we're involved in training of blood transfusion staff, training the prescribers of blood to ensure they have policies and plans and they have transfusion committees, national control authorities to ensure that there is regulation, particularly of blood plasma derivatives and quality management systems.

So let's then look at the Global Collaboration for Blood Safety. And from what I've told you, you might think that this is to do just with developing countries. I'd like to remind you that this global collaboration was actually borne out of a need that was experienced in 1993, when two very powerful member states in Europe had a problem with the UB plasma scandal, and we were called to their assistance. And it was clear from all of the evidence that we gathered in having that very quick meeting in Copenhagen at the European office, that there was a complete breakdown in global collaboration. People did not know where plasma was coming from, where it was going to, what regulations were in place or, in fact, even what ministers of health had signed in declarations to ensure the traceability of plasma and to exchange ideas.

As a result of that, unfortunately, at the same time, the Paris AIDS Summit was convened in 1994, and Jay Epstein was very helpful in coming to all of the preliminary meetings and guiding us through a lot of the issues, where we managed to get a declaration on one of the five major topics, and that was blood safety. And global collaboration was the item that was unanimously declared an important issue.

This global collaboration declaration was then carried forward to the World Health Assembly of the following year and was endorsed by all of the member states and the ministers of health of the countries.

This Global Collaboration for Blood Safety then is a collaboration. It's not a coordination. It's not WHO telling anybody what to do. It's a forum or an alliance or a place where people can meet from the groups of patients, of manufacturers, of all of the people that are interested in blood safety, with WHO providing the Secretariat and the infrastructure to hold this meeting.

The objectives were to provide a forum to share information, ideas and experiences, develop a common understanding of challenges facing blood systems nationally and internationally, keeping pace with developments in the field of blood transfusion, and in the field, as they are, in developing countries, identifying and assessing possible solutions and establishing collaborative alliances, where we can coordinate the activities of the different agencies who are trying to assist countries in improving their blood safety.

To develop and maintain this global database and provide information on the status of BTSs, we have the global database for blood safety, which is an ongoing activity of data gathering, which we update every two years. This information is to determine the needs, select priority countries, identify training needs and monitor and evaluate the impact.

So this Global Collaboration, with WHO as the Secretariat, involves all of the partners who need to be present. Jay is always right when he says things like, "This needs to be issues driven." And one of the issues that arose was the meeting that was held in Geneva, at which many of you were present. And this meeting was a consultation to provide a forum for discussing current blood policy issues, sharing the perspectives of different nations, developing strategies to ensure safe and effective blood transfusion practice and to afford communications.

The challenge of the meeting was to see the differences, notably within the developed countries on recent blood safety initiatives, and bridging the gap between blood safety policies and practices developed in developing countries, as opposed to those in developed.

The Global Collaboration, therefore, will build on all existing knowledge. It utilizes existing expertise, promotes dialogue and suggests realistic, effective and practical mechanisms. This is not a way that WHO points to anybody and says, "You should be doing this." This is a meeting of people who come around the table and identify the most logical, and practical and effective organization who should be looking at issues. They could look at WHO and address our weaknesses and tell us where we should be strengthened. We could look at the International Federation or Red Cross organizations and suggest that was an area of work they should be doing or whether it's the European Union or Commission or the Council of Europe, et cetera.

So the GCBS will lead to an improvement in global blood safety by raising international awareness. We will, hopefully, around this table, raise issues that need to be addressed and that the organizations can then take under their wing. We will develop strategies and guidance in general form. They will not interfere with the work of each of the members who attend this collaboration, and they will identify issues of global concern and bring together all countries. This will also, in particular, help developing countries meet with developed countries around a table to see how they can set standards for themselves, address issues, realize what they can and can't do and try and achieve those goals.

So, therefore, within this GCBS, with all of its partners and Secretariat, what I put up there is an example of the sort of work that could be identified by the Secretariat and the Global Collaboration.

At 1 o'clock you had the Decision Formulating and Blood Safety for Policy Makers meeting that was held in Geneva. This addressed a number of issues. One of the things that clearly was a problem for the meeting was it showed the differences between developed and developing countries. It showed the sort of things that you are debating now, which is the area of expertise which is so different from that of a developing country. However, there is still the place to look at that as part of a working group and report back to the major partnership.

There's issues on the selection of safe blood donors. How do developing countries, with a high prevalence of disease, infections, and infectious markers, deal with this issue? The safety and testing of blood and plasma products, the rights of patients working groups, getting thalassemics and hemophiliacs around a table to express their concerns. How should we deal with quality management? And what about the traceability of plasma moving in the international marketplace and many other issues.

In summary, then, the Global Collaboration for Blood Safety will improve safety and adequacy of blood and blood products globally; it will bring together developing and developed countries; it will provide a forum for exchange of information, identification of issues and concerns.

WHO is a unique organization, and in providing this forum that people can meet around a table, identify issues of concerns without obligations to each of those partners, but identifying hopefully to each of the interested parties and the responsible organizations where they need to improve their work, we can go together. We can also hopefully have this network identifying who is doing what and trying to coordinate activities, instead of having three or four organizations in one country all going in different directions.

Thank you, Mr. Chairman.

DR. CAPLAN: Thank you.

I don't think we'll have to ask the committee to formulate a resolution, but I am very grateful for this information, and I am sure that our Surgeon General will want to work closely to coordinate activities with WHO in this area.

I am reminded, too, that this committee has heard both for travelers overseas, terrorist incidents, issues that come up of national security, it is not solely a matter of humanitarian concern, but it is also a matter of concern to Americans what blood policy looks like worldwide.

So thank you.

DR. EMMANUEL: Thank you.

DR. CAPLAN: Did you want to say something?

DR. AuBUCHON: I just wanted to add, if I might, one piece of data that I learned at the Geneva Conference, which was striking. By my rough calculation in my head, the United States spends on the order of $15 per person per year in supplying blood products, blood components, transfusible blood components, to the population, say, at $15 per year per person. And one of the developing African countries--I forget which one--had data presented at the conference that they spend an average of 18 cents per person for all of their health care per annum. They obviously have a lot of challenges ahead of them.

DR. CAPLAN: Thank you.

DR. DAVEY: One quick comment?

DR. CAPLAN: Yes.

DR. DAVEY: Just also in support of Jean, who has done a wonderful job at Who and really good leadership for a number of years now, I think looking at this committee, we are the Advisory Committee on Blood Safety and Availability, and our responsibility is clearly to our own country. But yet, when we hear of these statistics, and many of us have participated in international fora, I think it is clear that when appropriate, from time to time, and I think you have maybe said this, Mr. Chairman, we really need to link very carefully and hear very clearly from our colleagues that are managing this immense problem internationally and just see how we can link what support we can give because we are very fortunate here. And to hear these disparities, it is very important that we have Jean and others over on a regular basis.

DR. CAPLAN: All right. I think we have got two updates to hear from; one on the status of blood availability from Paul and then one on the situation on plasma.

DR. McCURDY: Dr. Satcher and this committee talked in the past about the lack of information about blood supply in the United States. And it was pointed out during those discussions, there have been several attempts in the past to look into this and to collect data--among others, the American Blood Commission in the 1970s, and the NHLBI had a workshop that discussed it, but from which nothing came.

As a result of these two recommendations, however, from the committee and Dr. Satcher, the National Heart, Lung and Blood Institute formed an agreement with the National Blood Data Center of the American Association of Blood Banks to collect monthly data on the collection, distribution and utilization of blood. We opted to do this on a sampling basis, rather than a global basis. We started out with blood centers because we thought this was an easier thing to do, and we could get that up and running a lot more rapidly.

The data that we have to date on the total number of red cell products released, this is our red cells and whole blood. Beginning in October and going through March is shown up here. The top line is the total amount, and we are keeping track of this by blood group, and you see the O-positive and O-negative down below.

The first two months of this I believe had a relatively short number, a small number of banks reporting. Our total sample is meant to be 27 out of somewhat over 100 blood centers. The first couple of months had only 6 I think the first month and maybe 10 or 15 the second month. The last 2 or 3 months, however, we have gotten up to a level of 22 blood banks reporting.

The other thing that we've been doing is collecting inventory information. It's being collected on the first and third Wednesday of each month and submitted to the data center. Those first 2 months, October and November, and I guess the first time in December, again, is a very small sample. And I would not rely upon those figures. However, the last several months I think the figures are more reliable. Because we've had a different number of centers reporting each month and a growing number, I have attempted to normalize this to our current sample of 22; that is, divide by the number reporting and multiply by 22. We expect, as time goes on, that we will become more sophisticated, and I hope extrapolate this to the entire country rather than these 22 or we hope 27 blood centers.

We are making plans now to go to hospital transfusion services, and we expect it will take a little more time to bring those online than the blood centers. But we hope by sometime next fall to begin to collect data from the transfusion services. But one of the things we will be looking at most carefully is the failure to provide blood when a physician requests blood for transfusion or delay in surgical operation. This would indicate that the supply is not good. We will collect this by blood group, so that if it's an A-positive, we're really in trouble; if it's an O-negative, and it doesn't happen all of the time, but it happens from time to time.

DR. CAPLAN: Thanks. That's very exciting to see that underway.

Comments or questions?

[No response.]

DR. CAPLAN: Why don't we move to our last presentation on plasma.

MR. GUIHEEN: Hi. We wanted to make a quick substitution. I am Larry Guiheen. I'm with Baxter, and I have a flight to catch. So Jason has deferred to me. The reason I am here is in reference to our Recombinant Factor 8 supply. In December of last year, we announced that the demand for our product had exceeded our ability to supply. What had happened in the previous 5 months, the demand accelerated, and in fact, in the month of November, it went up from October by 50 percent.

So we announced to the hemophilia community, that would be the treaters as well as the patient organization, that we would be limiting our supply to the patients that we had already put on the product.

So what are we doing about supply? We have just completed or are about to complete our two additional suites in Thousand Oaks. We submitted the license application to the FDA last month for one of those suites, and the other will go in next month. This will be an additional two suites; the one suite we have in Thousand Oaks. That is the end of the process, but there is a process involved; that is, an inspection of those suites, and certainly a review by the FDA of our data and any questions we may have to answer for them.

So that's what's going on in Thousand Oaks. Once those suites are approved, which should be sometime later we hope this year, that should alleviate, I think, a lot of the shortages that we are seeing with our product.

In addition, we have identified another site for a plant. It has actually been under construction for a year, and additional supply will be available within 2 years from there. So we remain, as Baxter, committed to increasing the supply of this product as quickly as we can so that we can meet the needs of not only the U.S., but the rest of the world.

As we look to the community about where is the supply of Recombinant Factor 8, specifically, right now? What we are finding is that the patients that are on it, are able to get some brand. They may not be able to get the brand they want. But that there are some therapies that are not being able to be treated; that is more, in the case of an inhibitor, there are some patients that are not being able to be treated aggressively, and there are the MASAC recommendations concerning prophylaxis, as well as some clinical trials, are not being able to get started. But overall we think that there's a resolution in sight.

That's my comments. If anybody has any questions? Not at this time, okay. Thanks very much.

MR. BABLAK: Good afternoon. I'm Jason Bablak with PPTA. I think I am last on the agenda, so I will be quick and hopefully everyone can get out of here and catch their planes.

As you may recall, PPTA is the trade association representing the major commercial fractionaters. We were formerly known as the International Plasma Products Industry Association. And our members include Alpha Therapeutic Corporation, Aventis Bearing [ph.], Baxter Health Care, Bayer and ZLB.

I am going to speak very briefly about patient access issues that are affected by regulation and product supply. Specifically, I am going to talk about IVIG supply because this has been brought before this committee several times and just sort of as an update. We recognize the importance of all of the therapies we provide, including IVIG. We have taken many steps to ensure continued availability and access to IVIG and the other therapies we produce.

I want to give you a brief update on the supply situation. Our data that we have been collecting now for the last 2 years shows that a general leveling of the U.S. distribution of IVIG with increasing inventories. Our current data shows the inventory levels are at about a 3- to 4-week supply, which is up from approximately a 2- to 3-week supply during 1999.

We have also done other things besides collecting data, including working with other stakeholders, such as the Immune Deficiency Foundation, to ensure that patients have access to an adequate supply of IVIG. Our members individually maintain emergency supplies of IVIG and are also working through the IDF Safety Net Program to help patients locate IVIG if their normal supplier is unable to provide for their needs.

Our industry continues to make major investments in facility upgrades and research and development for IVIG production, as well as other products like Larry just discussed.

We are also working with the FDA to decrease the amount of time it takes to get approval for these new or improved products or processes. An area we are currently focusing on is the development of workshops and/or industry guidances on specific topics that are relevant to our industry. These activities serve to provide a better understanding of current regulatory requirements and help to reduce the time required to fully implement process or facility changes.

Currently, we are working on industry guidelines for process validation, which is a very large issue for our industry and facility supplements. And we anticipate cosponsoring a workshop with the FDA later this year on comparability protocols.

In addition to an improvement in the understanding of regulatory requirements, the volume of submissions and other contacts with the Agency can affect review time, and therefore also needs to be addressed. PPTA supports funding the Agency at a level to allow for adequate staff to review these licenses and other submissions. As you may be aware, our industry pays user fees under the Prescription Drug User Fee Act, and we encourage the FDA to ensure that staffing within the Agency centers is reflective of these payments.

Finally, the FDA recently announced a revised clinical trial protocol for new IVIG products that was developed in cooperation with the IDF. We are supportive of the public-private model used by the FDA, which included input from physicians and patients to develop the protocol that builds on the historical data, requires fewer patients and relies on surrogate markers as end points for the trial, yet maintains confidence in the trial's results. We believe that this consensus position will increase patient access to IVIG by streamlining clinical trials and thereby reducing the amount of time required to license new products and/or processes.

In closing, we support optimal regulation that includes patient access as a critical component. We continue to work with our stakeholders to ensure adequate access to these therapies, while maintaining their safety and quality. We encourage the FDA to continue developing rational regulations, such as this new clinical trial protocol for IVIG and to expand the use of this model to other areas of regulation.

Thank you.

DR. CAPLAN: Thank you.

I have just two questions for you: First, do you think the advisories that the committee asked to be sent out to physicians in terms of prudent use of IVIG have been helpful in building that inventory, getting more supply in place?

MR. BABLAK: I am unable to answer that question. I can only talk about from the manufacturers' point of view, not from a prescribing physician's point of view. Certainly, working with the FDA, they have been able to get some new products approved for this market, get some new production facilities back on line.

DR. CAPLAN: In other words, what I am asking you is it supply increase or more intelligent demand that seems to be building the inventory, if you know? Maybe you don't.

MR. BABLAK: If you look at the numbers, they are down from before the shortage became critical. So one would assume that there is less use of IVIG, while the inventory is going up. So you could assume that, but I can't guarantee it.

DR. CAPLAN: And my other question is do you have any comment on price? That was of concern to the committee sometime back, in terms of per gram?

MR. BABLAK: I have no information on current pricing.

DR. CAPLAN: Other comments?

MR. BABLAK: Except that we haven't heard any complaints recently about it. But that's all I've heard.

DR. HAAS: Could you give us an update like Baxter did on the anti-hemophiliac products, what's happening with the other companies?

MR. BABLAK: I don't have any company-specific information. I can basically review the numbers that we have collected, for example, for all of Recombinant Factor 8, for example. The trend there shows that it is going down slightly, the availability of that product.

DR. CAPLAN: Any other questions, comments?

MR. ALLEN: I know he just asked you about price, but has the price, to your knowledge, stayed the same over the last 4 to 5 months? Has it stayed the same? Has it gone down any? Do you know anything about that?

MR. BABLAK: We do not collect data on pricing for a variety of legal reasons. So I can't really answer that question, except to the extent that we haven't heard the complaints from stakeholders that we had heard earlier, that there was some concern with pricing situations. We haven't heard those complaints, but it may be that because we can't do anything about it we don't hear any more or it may be that they're not there. I just can't answer that.

MR. ALLEN: Is there anyone else that can answer it?

DR. WINKELSTEIN: My understanding is that at least in one company's case there has been a significant increase in the last 6 to 12 months.

DR. CAPLAN: All right. Thank you.

I am sure we will return back in again to the supply situation and cost as well on those products. But it is at least somewhat gratifying to see that we have more data today than we did when we first looked at both plasma and blood product supply situation and whole blood red cell some time ago. So that's actually, on the whole, a good thing I think.

Well, I think we got a tremendous amount done at this meeting. I think that we will return, as promised, to some significant part of the next meeting on leukocyte. We will certainly update at that next meeting. I think we got what the agenda items are for that. We will get you information about the August 23rd-24th. It will be here. It will be here, same place, some station.

Mr. Healy wishes to speak.

MR. HEALY: Yes. I know it's late in the day, so I will be very brief.

My name is Chris Healy, and I'm with ABRA. ABRA is the trade association--

DR. CAPLAN: Oh, I'm sorry. I didn't realize you were here.

MR. HEALY: That's okay. --trade association and standard-setting organization for the source plasma collection industry. I wanted to just briefly touch on plasma supply. At the time when issues regarding IVIG supply were most salient, there appeared to be an abundance of source plasma. However, as the IVIG issues have stabilized somewhat, the supply of source plasma has declined. Data available from the ABRA National Donor Deferral Registry show a 7-percent decline in new plasma donors from '97 to '99.

Other data appear to show a decline in total liters of plasma collected from 11 million liters in 1998 to 10 million liters in 1999, a fairly significant decline. It doesn't appear that this decline has had any impact on the availability of plasma derivatives, however. Nonetheless, it is concerning, and I will continue to monitor these data and support other industry efforts to increase donor recruitment and retention efforts.

That is it.

DR. CAPLAN: Any speculation on what's the decline--

MR. HEALY: On the factors. I think it is a lot of the same factors that we have seen in the whole blood sector. I think the economy has shortened people's days, if you will, and made it tougher to find time to make the hour-and-a-half commitment weekly, a couple of times a month, to make source plasma donations.

DR. WINKELSTEIN: What's the delay between plasma collection and the actual packaging of IVIG--six months?

MR. HEALY: In other words, the time period to manufacture the product? I am not completely qualified. I would venture a guess to say about 250 days, something like that.

DR. WINKELSTEIN: And when did your shortage show up?

MR. HEALY: We've noticed these data just within the last few months, and we've heard people out in the marketplace saying that plasma, the markets are somewhat tighter.

DR. CAPLAN: So that's something to flag then. It could be a time line coming.

MS. LIPTON: I was going to ask exactly the same question. It seems to me that if we're seeing a decline, that we are going to be experiencing this issue again.

MR. WALSH: Mr. Chairman, in fact, related to this there is a shortage of 401 paste for the production of A1PI with the single manufacturer. And if we can get some assistance on that, it would be appreciated. It will affect supply in moving forward.

DR. CAPLAN: I think what we have to do there is make sure, and I know that the Secretary is aware that this committee wants vigilance with respect to the supply area, one of things we'll do then is perhaps even go back as an update to the issue in August, in terms of maybe even getting some comments about attempts to work with the industry to get facilities through manufacturing capacity online, steps to increase the plasma supply. And this is clearly one of our chronic headaches. And I don't know that we're going to make it go away. But we can't back off from it.

Thank you.

MR. HEALY: Thank you.

DR. CAPLAN: John?

MR. WALSH: One housekeeping point with respect to shortages. There is a five points of life ride creating donor awareness for blood, apheresis tissue, organ, and something else--five points at any rate. And we'll be circulating, I'll ask our executive secretary to circulate information about that. It is going through 13 states from Maine to Florida, and we would certainly suggest that any committee member in those states try to participate as much as they can to create additional awareness.

Our esteemed executive secretary is going to make the ride for at least the beginning day or so. And I think this is a very important--it's a good opportunity for us to create more awareness about blood and plasma, specifically.

DR. CAPLAN: Okay. Thank you. We will conclude.

[Whereupon, at 4:19 p.m., the proceedings were adjourned.]