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
P A R T I C I P A N T S
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.
C O N T E N T S
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
P R O C E E D I N G S
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
Federal Register. 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.]
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