Mr. Chairman, Members of the Committee:
Thank you for your invitation to discuss the safety and availability of the blood
I. RECENT ADVANCES IN BLOOD SAFETY
Our nation's blood supply is safer than it
ever has been, and it is getting safer even as we speak. Dr. AuBuchon, who testified
before you last month, reported just two years ago that the risk of hepatitis C from blood
transfusion was about one in one hundred thousand transfusions, or about 120 cases per
year. He also reported that the risk of HIV was about one in five hundred thousand
transfusions, or about 24 cases per year.
Since then, nucleic acid tests for hepatitis C and HIV have been developed. Most older
tests, which produced the results Dr. AuBuchon described, detect antibodies to these
viruses. In contrast, nucleic acid tests detect the viruses themselves. For this reason,
nucleic acid tests may help us close the so-called "window
period"between the time an infectious agent
appears in the blood and the time that infection can be detected.
Currently, nucleic acid tests are investigational, and are being evaluated widely using
pools of donated blood. Even more sensitive nucleic acid tests performed on single units
are under development. Collectively, these advances should reduce the risk of
transfusion-transmitted hepatitis C and HIV by an order of magnitude, and possibly even
II. DEPARTMENT ACCOMPLISHMENTS IN BLOOD SAFETY
A. PRIORITIZATION OF BLOOD SAFETY WITHIN THE DEPARTMENT
Our efforts to increase the safety of the blood supply began in the earliest days of
this Administration. In July 1993, Secretary Shalala commissioned the Institute of
Medicine to prepare a report on the introduction of HIV into the blood supply. She did
this, as she said at the time, "... to insure
the safety of the Nation's blood supply against
new challenges in the future."
The Institute of Medicine released its report in July 1995. On the day it was released,
the Secretary directed Dr. Lee, then the Assistant Secretary for Health, and senior
members of the Department to review it. In October of that year, in testimony before
Congress, the Secretary announced her concurrence with the report. She also announced that
she had designated the Assistant Secretary for Health to be the Blood Safety Director for
the Department. In addition, she announced the establishment of two high-level Committees
on blood safety and availability, one internal and one external to the Department. The
Secretary concluded her testimony by saying
Blood and blood products will always be capable of transmitting disease, and their use
will never be completely free of risk. But for everyone who relies on blood and blood
products to sustain life, the Federal Government will do everything in its power to reduce
risk and assure availability.
We are implementing the Secretary's policy
- Timely introduction of new technologies, such as nucleic acid testing;
- Proactive response to the threats posed by emerging and re-emerging infectious diseases,
such as transmissible spongiform encephalopathies; and
- Increased attention to quality assurance, such as emphasis on current Good Manufacturing
We believe that these three initiatives will permit us to achieve the degree of blood
safety that the American people demand without compromising the blood availability that
the American people need.
B. BLOOD ACTION PLAN
I feel it is important to stress, in light of earlier discussion by this Committee, the
commitment of the Food and Drug Administration to increasing the effectiveness of
regulation of the blood industry. The concrete expression of this commitment is the Blood
Action Plan, which was initiated in July 1997. The Blood Action Plan is one of FDA's responses to the 1995 Institute of Medicine report,
and also to several congressional oversight reports since that time.
The Blood Action Plan established a comprehensive review of all blood-related
regulation. Most of this review has been completed. In May and August of this year, FDA
published Direct Final Rules, and companion Proposed Final Rules, that updated technical
standards for blood and blood products. In August of this year, FDA proposed two
additional rules, one on testing blood donors for infectious diseases, and another on
donor notification. Also, in August 1999, FDA proposed an important consumer safety
initiative: a tracking system for plasma derivatives from manufacturer through the
distribution network to the end user that will insure prompt notification when indicated.
Progress has also been made in improving responsiveness of the Agency. Under the Blood
Action Plan, FDA is harmonizing its new Biologics License Application for blood products
with its New Drug Application. In addition, FDA, CDC, and NIH representatives have formed
an Emerging Infectious Diseases Committee that has developed plans for responding to
emerging infections that threaten the blood supply. FDA has also reorganized its field
inspection programs. As a result, there has been considerable progress towards bringing
the blood and blood products industries into full compliance with current Good
Manufacturing Practice requirements.
C. HEPATITIS C LOOKBACK
In the interest of time, I will only briefly mention the Department's initiative to notify all individuals who may have
been inadvertently exposed to hepatitis C through blood transfusion. FDA has moved rapidly
to issue Guidances on this initiative, and it is moving rapidly as well to issue a Final
Rule on this matter. As part of this effort, CDC is coordinating a Hepatitis C Public
Information Campaign and, in collaboration with FDA and the Agency for Health Care Policy
Research, will carefully evaluate both the targeted and the general notification efforts.
D. DEFENSE AGAINST TRANSMISSIBLE SPONGIFORM ENCEPHALOPATHIES
Over the past four years, the Department has substantially updated its policies
regarding the transmissible spongiform encephalopathies. These include Creutzfeldt-Jakob
disease, or CJD, and new variant CJD, the human form of "mad
cow" disease. Animal experiments indicate that
both are potentially transmissible by blood transfusion, but no actual case of this has so
far been observed in humans.
Extensive epidemiologic data has permitted us to revise some precautions against CJD.
However, new and disturbing information about new variant CJD has recently become
available. We have learned that the prion of new variant CJD is different from the prion
of classic CJD. This may explain why the clinical manifestations of the two diseases are
so different. We have learned that the prion of new variant CJD forms deposits in
peripheral tissues, and there is some preliminary evidence in transgenic mice that
suggests a role for certain blood cells in the pathogenesis of this disease. We have
learned that blood from rodents experimentally infected with Mad Cow Disease can, under
some circumstances, transmit it to other rodents. We have learned that the risk of new
variant CJD is uniformly distributed throughout the United Kingdom. In fact, the only
definable risks of new variant CJD at present are long-term residence in the United
Kingdom, and methionine homozygosity at codon 129 of the PRNP gene."
The first critical piece of information we currently lack is the length of the
incubation period between exposure to the agent of new variant CJD and the onset of
symptoms. In cows it is five years. If it is also five years in people, there may be no
more than 500 victims of this disease. However, if the incubation period is up to forty
years, there could be more than 500,000 victims of new variant CJD.
The other critical piece of information we currently lack is whether new variant CJD is
in fact transmitted by blood transfusion in man. No such instance has yet been identified,
but the possibility has not been excluded. Some have suggested that we take no action
until the first case of transfusion-transmitted new variant CJD is documented. However, by
that time, how many others might be irreversibly infected with a uniformly fatal disease
that destroys the brains of its victims in the prime of their lives? We have chosen to
reduce this risk by the limited methods now available to us, and not wait until the answer
In formulating our policies, we have repeatedly invited comment from scientists, the
blood industry, and the public on their perception of the risk to blood safety posed by
new variant CJD to blood safety, the impact of any measures we might take to reduce that
risk, and what we could do to minimize that impact. As we expected, the advice varied. For
this reason, we followed the process established by Secretary Shalala in 1995, which was
to bring this matter promptly to the highest level of the Department. The Food and Drug
Administration's Transmissible Spongiform
Encephalopathy Advisory Committee made their final recommendation on June 2 of this year
regarding donor deferral based on a period of residence or travel in the United Kingdom;
the Blood Safety Committee, which I chair, met on June 8. The Blood Safety Committee's recommendation was unanimous in favor of this
precautionary measure. FDA announced this recommendation at its Blood Products Advisory
Committee meeting on June 17, and FDA's Guidance
to Industry was issued on August 16.
III.DEPARTMENT ACTIONS TO ASSURE THE AVAILABILITY OF BLOOD
At the June 8 meeting of the Blood Safety Committee, I appointed an Interagency Working
Group to identify strategies to increase the blood supply. I received their report on
August 10, and discussed it at the August 26 meeting of the Advisory Committee on Blood
Safety and Availability. I concur with each of their recommendations, both for the short
and the long term, and I have directed the appropriate agencies to implement these
recommendations as soon as possible. Much of this implementation has already occurred.
A. MONITOR THE BLOOD SUPPLY
The first recommendation of the Interagency Working Group was to institute prospective
monitoring of the blood supply. The NIH has committed up to $300,000 in the current fiscal
year to purchase this data from the National Blood Data Resource Center, beginning as soon
as possible, and perhaps even this month. Plans for long-term management of this activity
are currently under review.
B. ENCOURAGE THE PUBLIC TO DONATE (AND TO CONTINUE DONATING)
The second recommendation was to increase suitable donations. As the General Accounting
Office report to your Committee pointed out, there are plenty of eligible donors; we
simply need to find more effective ways to encourage them to donate. The NIH has committed
$1.8 million dollars in the current fiscal year to research in this area.
C. IMPROVE RELATIONS BETWEEN DONORS AND BLOOD ESTABLISHMENTS
The third recommendation was to improve relationships between donors and blood centers.
While much of this task is the industry's, the
Department will consider steps that it can take. For example, there may be ways to make
required donor questionnaires less burdensome without making them less effective.
D. REMOVE UNNECESSARY RESTRICTIONS TO DONATION
The fourth recommendation was to review current donor deferral policies, particularly
in light of emerging technologies such as nucleic acid testing. FDA's decision to reassess donations by individuals with
hemochromatosis that was announced on August 10 by Commissioner Henney is only one of
several responses under active consideration by FDA.
E. ADDRESS ECONOMIC CONCERNS OF THE BLOOD INDUSTRY
The fifth and final recommendation was to address economic pressures on the blood
industry that may limit its performance. The Advisory Committee on Blood Safety and
Availability reviewed this issue at its August 27 meeting. The Committee's recommendations are currently under review within
IV.OTHER DEPARTMENTAL INITIATIVES IN BLOOD SAFETY
Another issue that both we and you have under active review is accident and error
associated with blood administration. FDA has long recognized the importance of reporting,
investigation, and correction of errors and accidents. FDA has already used information
learned from error and accident reports to identify areas where clinical practice and
government regulation could be improved. FDA did issue a Proposed Rule in 1997 to require
unlicensed as well as licensed institutions to file these reports, and to quantify the
time period in which they should be filed. However, a Final Rule on these matters has not
yet been issued.
It is important to emphasize that the error and accident reporting required under this
rule serves not to alert the FDA to emergencies, but to provide an additional layer of
data for quality assurance, longer-term saftey monitoring, audits, and to help target
inspections. The major proactive approach to assuring high quality and compliance with
regulations in the blood industry is FDA's
regular inspection process. In fact, the vigorous FDA inspection program has led, in
recent years, to a number of major enforcement actions which have helped assure blood
Mr. Chairman, I would like to join your fellow Committee members who have thanked you
for holding this hearing, because you have raised awareness of an essential layer of the
blood safety system. I want to assure you that none of us are waiting for the time
required to issue a final rule to address this important matter. As you know, on August 13
of this year I had directed the Advisory Committee on Blood Safety and Availability to
place this matter on their agenda. This meeting is scheduled for late January 2000, and
preparations for it are already under way. One of the invited speakers will be Dr. Harold
Kaplan, the Professor of Transfusion Medicine at Columbia University, who has been awarded
a research grant by the National Heart, Lung, and Blood Institute to study transfusion
error and how it might be reduced. The Department will promptly consider any
recommendations that arise from this meeting, and I would be delighted to inform you of
our response to these recommendations as soon as it is formulated.
I would be happy to answer any questions you may have.