[Senate Hearing 111-309]
[From the U.S. Government Publishing Office]
S. Hrg. 111-309
BIOLOGICAL THREATS: IS THE CURRENT U.S. VACCINE PRODUCTION SYSTEM
PREPARED?
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HEARING
before a
SUBCOMMITTEE OF THE
COMMITTEE ON APPROPRIATIONS UNITED STATES SENATE
ONE HUNDRED ELEVENTH CONGRESS
FIRST SESSION
__________
SPECIAL HEARING
AUGUST 21, 2009--PITTSBURGH, PA
__________
Printed for the use of the Committee on Appropriations
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COMMITTEE ON APPROPRIATIONS
DANIEL K. INOUYE, Hawaii, Chairman
ROBERT C. BYRD, West Virginia THAD COCHRAN, Mississippi
PATRICK J. LEAHY, Vermont CHRISTOPHER S. BOND, Missouri
TOM HARKIN, Iowa MITCH McCONNELL, Kentucky
BARBARA A. MIKULSKI, Maryland RICHARD C. SHELBY, Alabama
HERB KOHL, Wisconsin JUDD GREGG, New Hampshire
PATTY MURRAY, Washington ROBERT F. BENNETT, Utah
BYRON L. DORGAN, North Dakota KAY BAILEY HUTCHISON, Texas
DIANNE FEINSTEIN, California SAM BROWNBACK, Kansas
RICHARD J. DURBIN, Illinois LAMAR ALEXANDER, Tennessee
TIM JOHNSON, South Dakota SUSAN COLLINS, Maine
MARY L. LANDRIEU, Louisiana GEORGE V. VOINOVICH, Ohio
JACK REED, Rhode Island LISA MURKOWSKI, Alaska
FRANK R. LAUTENBERG, New Jersey
BEN NELSON, Nebraska
MARK PRYOR, Arkansas
JON TESTER, Montana
ARLEN SPECTER, Pennsylvania
Charles J. Houy, Staff Director
Bruce Evans, Minority Staff Director
------
Subcommittee on Departments of Labor, Health and Human Services, and
Education, and Related Agencies
TOM HARKIN, Iowa, Chairman
DANIEL K. INOUYE, Hawaii THAD COCHRAN, Mississippi
HERB KOHL, Wisconsin JUDD GREGG, New Hampshire
PATTY MURRAY, Washington KAY BAILEY HUTCHISON, Texas
MARY L. LANDRIEU, Louisiana RICHARD C. SHELBY, Alabama
RICHARD J. DURBIN, Illinois LAMAR ALEXANDER, Tennessee
JACK REED, Rhode Island
MARK PRYOR, Arkansas
ARLEN SPECTER, Pennsylvania
Professional Staff
Ellen Murray
Erik Fatemi
Mark Laisch
Adrienne Hallett
Lisa Bernhardt
Bettilou Taylor (Minority)
Dale Cabaniss (Minority)
Sara Love Swaney (Minority)
Administrative Support
Teri Curtin
Jeff Kratz (Minority)
C O N T E N T S
----------
Page
Opening Statement of Senator Arlen Specter....................... 1
Statement of Hon. Jason Altmire, U.S. Representative From
Pennsylvania................................................... 2
Statement of Bruce G. Gellin, M.D., M.P.H., Director, National
Vaccine Program Office, Department of Health and Human Services 4
Prepared Statement........................................... 5
Statement of Jeffrey A. Romoff, President and Chief Operating
Officer, University of Pittsburgh Medical Center, Pittsburgh,
Pennsylvania................................................... 12
Prepared Statement........................................... 14
Statement of Philip K. Russell, M.D., Retired Major General, Army
Medical Corps; Board of Trustees and Senior Scientific Advisor,
Sabin Vaccine Institute, Washington, DC........................ 16
Prepared Statement........................................... 18
Statement of Philip Gomez, Ph.D., Director, Biodefense and Public
Health Practice, PRTM Management Consultants, Washington, DC... 20
Prepared Statement........................................... 22
Statement of Donald S. Burke, M.D., Dean, Graduate School of
Public Health; Associate Senior Vice Chancellor for Global
Health; Director, Center for Vaccine Research, University of
Pittsburgh, Pittsburgh, Pennsylvania........................... 24
Prepared Statement........................................... 26
Statement of Nigel Darby, Ph.D., Vice President, Biotechnologies,
Life Sciences, GE Healthcare Bio-Sciences AB, Uppsala, Sweden.. 27
Prepared Statement........................................... 29
BIOLOGICAL THREATS: IS THE CURRENT U.S. VACCINE PRODUCTION SYSTEM
PREPARED?
----------
FRIDAY, AUGUST 21, 2009
U.S. Senate,
Subcommittee on Labor, Health and Human
Services, and Education, and Related Agencies,
Committee on Appropriations,
Pittsburgh, PA.
The subcommittee met at 10:30 a.m., in Courtroom 6A,
Pittsburgh Federal Courthouse, 700 Grant Street, Hon. Arlen
Specter presiding.
Present: Senator Specter.
Also present: Representative Jason Altmire.
opening statement of senator arlen specter
Senator Specter. Good morning, ladies and gentlemen. It is
10:30 a.m., time to proceed with the hearing of the
Appropriations Subcommittee on Labor, Health and Human
Services, and Education to take up the subject of 21 CB, 21st
century biodefense.
The subcommittee is pleased to welcome Congressman Jason
Altmire to join us in this hearing. Congressman Altmire's
district encompasses some of UPMC.
Our hearing today will focus on a very serious problem
facing the United States and facing the world and that is the
problem of swine flu and the problem of biodefense against
other items where we need vaccines, some of which implicates
the threat of terrorist attack.
As to swine flu, for which we are looking for a vaccine of
H1N1, as of August 13, a week ago yesterday, in the United
States 7,511 people have been hospitalized, 477 deaths in the
United States from swine flu; worldwide, 1,799 deaths. And we
have been looking for a vaccine of H1N1 where the expectation
had been to have some 120 million doses as of October 15, and
the Department of Health and Human Services (HHS) announced
last Monday that instead of the 120 million doses previously
forecast, we only have 45 million doses.
A number of problems have been created, which we will hear
detailed in our hearing. One company in Australia was taking
care of Australia first, not taking care of the United States
first. We would expect UPMC not to ignore Australia, but look
at the United States first.
Another company had to take care of their regular
accommodations.
I do not know what happened to the other three companies.
The witness, the distinguished Dr. Bruce Gellin, Director of
the National Vaccine Program Office, can tell us about what
those problems were there. But it is obvious that we have a
significant problem in meeting a problem which could be
enormously serious--enormously serious.
We are looking at the limitations of availability of
vaccines which have quite a number of other potential fronts
such as smallpox, anthrax, ebola virus, botulism, and another
long list, some of which are susceptible to terrorist attacks,
for example, if the smallpox virus were unleashed in the United
States. What we want to do is to avoid having the Government
come up short on something like what happened with Katrina
where we are unprepared for the eventuality.
Since 2004, when I chaired this subcommittee, with the
joinder of Senator Harkin, who is now the chair, we
appropriated $6.4 billion to deal with the vaccine problem, and
in the supplemental recently we added $5.8 billion for the
discretion of the President to call up the funding. So you can
see that we are talking about very substantial funds to meet a
very substantial problem.
What we are looking for on the project, which has been
worked on by UPMC, is an $830 million project, $580 million
from the United States and $250 million from a public/private
partnership, which is directed to give the United States
control, not to have it in the hands of Australia or companies
which may or may not be reliable.
UPMC has come forth with a very important program where
their CEO, Jeff Romoff, has played an active part. We have had
a series of top level meetings, one in the office of Vice
President Biden on March 31, an old train pal of mine. Early in
his work as Vice President, I said to Joe we need a meeting
with some people here to tackle an important problem, and he
promptly opened his office and brought in key people. He is in
charge of the funding on the stimulus package, $787 billion,
and on May 20, asked Secretary Sebelius to be present at a
meeting which was in my office. Again, CEO Romoff came, and on
June 15 when Secretary of Homeland Security Janet Napolitano
was in Philadelphia on another matter, we had a third meeting
to acquaint the key people who are involved in this important
matter.
One witness today from General Electric (GE) will testify
about GE's involvement on some matters and technology which
they have exclusive control. They have the patents. Smaller
companies, to change all the production of vaccines, have to go
to different equipment and clean steel which takes a long time.
GE has a disposable plastic apparatus.
So you can see a lot of thought has been given to this
issue, and the general approach is to recognize the primacy of
competitive bidding. John Myers, my able deputy, is now
drafting authorizing legislation to move ahead on this project
so we do not get caught like we got caught on Katrina, do not
get caught with a problem with swine flu or any of these other
kinds of problems.
Now I am pleased to yield to my distinguished colleague,
Congressman Jason Altmire. Jason.
statement of hon. jason altmire, u.s. representative from pennsylvania
Mr. Altmire. Thank you, Senator, and thank you for allowing
me to participate as part of this very important----
Senator Specter. Jason, you may want to wait just a minute.
The TV cameras are being set up.
Mr. Altmire. I think they are probably here to see you,
Senator.
Senator Specter. I am not going to begin my presentation at
the start, although I probably should, but I will rely on PCN,
Pennsylvania Cable Network, to carry it.
Mr. Altmire. Well, I would just say very briefly thank you,
Dr. Gellin, for being here. There are few, if anyone, in the
country who knows more about this and has a level of expertise
about this subject matter than you, and we very much appreciate
you being here today.
This is an issue that is very important to many in the
Congress, both in the House and the Senate. We feel that we
need to have some advice from you on whether or not we are
prepared as a Nation because it appears often--we saw it with
H1N1--that we are in a reactionary mode rather than a
preparedness mode. We are always reacting to what we see. And I
know you cannot predict the future. You cannot predict what is
coming down the road, but we believe that the Nation would
benefit from having a better prepared system.
We strongly believe here in western Pennsylvania that we
have assets that we can bring to the table that would be of
great national interest. We have strength of organization here
that is unmatched anywhere else with regard to this issue. We
believe we have the expertise and the planning to put this
together in a way that is unmatched anywhere in the country. We
have the strengths of infrastructure and in financing that we
feel like are unmatched anywhere in the country. Regardless of
what the conclusion is on that, we believe that the first step
has to be an acknowledgement that we could do better with
regard to preparedness with our vaccination program.
So we greatly appreciate you being here and look forward to
your remarks on this issue, and we look forward to hearing the
second panel as well. Thank you.
Senator Specter. Thank you, Congressman Altmire.
Just one additional note. With the kind of a proposal for
UPMC, it would create 1,000 jobs, 6,000 indirect jobs, and
there is a major concern in Washington, DC, about stimulating
the economy. We are not going to spend any money which is not
really necessary as a matter of public policy and public
welfare, but in an area which has been hard hit by an economic
decline, this is something which the President has in mind and
I know the Vice President does as do the Secretaries of HHS and
Homeland Security.
Dr. Gellin is the Director of the National Vaccine Program
Office within HHS. He has held positions at the National
Institutes of Health (NIH), the Centers for Disease Control and
Prevention (CDC), the two premier Federal agencies dealing with
health, also the Rockefeller Foundation and the Johns Hopkins
University School of Public Health, quite a prestigious
background.
Under our subcommittee procedures, Dr. Gellin, you have
testified many times. So you know it is 5 minutes. To stay
within the limit, to the extent you can, would be appreciated.
Please proceed.
STATEMENT OF BRUCE G. GELLIN, M.D., M.P.H., DIRECTOR,
NATIONAL VACCINE PROGRAM OFFICE, DEPARTMENT
OF HEALTH AND HUMAN SERVICES
Dr. Gellin. Thank you very much, Senator Specter. It is a
pleasure to be with you here today to discuss this important
topic, particularly what you have highlighted in terms of our
ongoing challenges related to the H1N1 influenza outbreak and
in terms of lessons that we have learned in our preparedness
planning for an influenza pandemic. Vaccines are an important
part of our public health system and our medical countermeasure
armamentarium particularly against biological weapons. But we
also need to recognize--and my role at HHS is in vaccines at
the National Vaccine Program Office, but what we are talking is
the range of countermeasures of which vaccines are one. So it
is in addition to the preventive aspects or some of the
treatment aspects for chemical, biological, radiological, and
nuclear threats.
In the interest of time, I am going to focus my brief
remarks this morning specifically on medical countermeasure
advanced development, recognizing it is just one step in the
larger process.
In July 2008, Dr. Bob Kadlec, then the Special Assistant to
the President for Biodefense and Senior Director for Biodefense
at the Homeland Security Council, requested that HHS and the
Department of Defense (DOD) conduct an analysis of alternatives
to identify the optimal facilities and operating models to
address the gaps in the production and manufacturing of medical
countermeasures against weapons of mass destruction threats in
a manner that provides the best long-term value to the
Government. Again, that highlights what Congressman Altmire was
talking about, just the relative degree to which we are looking
at preparedness rather than just reacting to things and just
recognizing that we need to be moving forward on this.
To accomplish this analysis, HHS and DOD commissioned an
independent third-party study of manufacturing facility
alternatives that should be considered. Because the commercial
market for these products is small and the pathways long and
the complexities are many--I think that is evidenced with what
you discussed, Senator Specter, about the complexities of just
the reliability of producing the influenza vaccine by the
manufacturers that make vaccine.
For these reasons, the industry as a whole has not been
particularly interested in these kind of products, and there is
a perception that large gaps exist in the manufacturing and
production facilities. The recent history shows that both HHS
and DOD have successfully contracted with biotechnology
innovators and contract manufacturers for advanced development
and procurement of medical countermeasures. Moreover, some of
these contractors are investing heavily in production
facilities, and the majority in the United States, to further
address these gaps.
The independent third-party analysis, which I mentioned,
suggests that the Government should increase our capabilities
to oversee the advanced manufacturing process development and
supply programs to ensure that the needed medical
countermeasures achieve FDA approval, there is an ongoing
supply of the product, and there is an ability to surge
production should a need be there.
Three alternative scenarios for the development, approval,
manufacturing, and the sustained replenishment of large
molecule medical countermeasures were examined in this analysis
using both quantitative and qualitative criteria.
The first of the three examined the continuation of the
existing process of contracting the development and manufacture
of medical countermeasures.
The second alternative examined was continuing the existing
process of contracting for the development but, in addition to
that, strengthening the technical, quality/regulatory, and
sourcing and supply capabilities in addition to contracting
with additional manufacturers for bulk ingredients, as well as
additional manufacturers for final formulation and filling.
This approach also provides for enhanced access to process
development and manufacturing capabilities.
The third alternative is one that has been proposed in
previous studies, and it calls for the Government to
manufacture all needed medical countermeasures. This approach
includes establishing a new public/private partnership that
would include the need for a fully dedicated manufacturing
facility for all medical countermeasures under control of the
U.S. Government.
HHS is committed to protecting the health and safety of
American citizens from both CBRN biodefense threats and
emerging infectious diseases and, as we talked about, assuring
the Nation's preparedness. Along with our colleagues at DOD,
HHS is committed to a full examination and discussion of all
viable options for the manufacture of vaccines and other
medical countermeasures against these identified threats.
prepared statement
There have been numerous conversations at the technical
level about the needs that have been identified and this gap
that is being discussed, as well as the range of possible
solutions. Given the importance of this topic, all options are
on the table right now. And HHS and DOD leadership will be
meeting soon to discuss the findings of this report and
determine the path forward.
We appreciate your support on this very important topic and
your continuing interest in this area. I am happy to answer any
questions you have.
[The statement follows:]
Prepared Statement of Bruce Gellin
introduction
Good morning, Senator Specter and Representative Altmire. I am Dr.
Bruce Gellin, Director of the National Vaccine Program Office within
the Department of Health and Human Services (HHS). I am honored to be
here today to discuss this important topic, particularly in light of
our ongoing challenges related to the 2009-H1N1 influenza outbreak.
Vaccines are an important piece of our public health system and our
medical countermeasure armamentarium, particularly against biological
weapons. This morning I will provide a brief overview of HHS
responsibilities for medical countermeasures development for chemical,
biological, radiological, and nuclear threats and then will focus more
specifically on the topic of medical countermeasure manufacturing.
medical countermeasures--development and acquisition
Our progress in securing medical countermeasures begins with and
depends on effective planning. The central framework for medical
countermeasures planning and implementation in the Federal Government
is the HHS Public Health Emergency Medical Countermeasures Enterprise
(PHEMCE), established in July 2006. This coordinated interagency group
is led by the Assistant Secretary for Preparedness and Response (ASPR),
and includes my office, the Centers for Disease Control and Prevention
(CDC), the Food and Drug Administration (FDA), and the National
Institutes of Health (NIH) as well as our partners from the Department
of Defense (DOD), Department of Homeland Security (DHS), and Department
of Veterans Affairs (VA). Through this Enterprise-wide effort, we are
able to ensure that Federal activities with respect to needed medical
countermeasures are effectively coordinated from research and
development to acquisition and, ultimately, deployment. This supports a
range of programs that I will briefly summarize for developing and
acquiring medical countermeasures for man-made and naturally occurring
public health threats while building domestic manufacturing
infrastructure.
The Biomedical Advanced Research and Development Authority (BARDA)
within HHS's ASPR directs and coordinates the Department's
countermeasure and product advanced research and development
activities. BARDA establishes systems that encourage and facilitate the
development and acquisition of medical countermeasures such as
vaccines, therapeutics, and diagnostics, as well as innovative
approaches to meet the threat of chemical, biological, radiological and
nuclear (CBRN) agents and emerging infectious diseases, including 2009-
H1N1 influenza. BARDA provides an integrated, systematic approach to
the development and purchase of the necessary vaccines, drugs,
therapies and diagnostic tools for public health emergencies. It
directs and coordinates the Department's countermeasure and product
advanced development activities and medical countermeasure domestic
manufacturing infrastructure building, including strategic planning for
medical countermeasure research, development, and procurement. This
coordinated approach is critical to achieving success in the area of
bioterrorism preparedness and has been proven through the recent H1N1
effort.
Specifically with respect to vaccines, HHS has a number of efforts
underway. These efforts supported the first U.S. licensure of an avian
influenza-based H5N1 vaccine in April 2007, which was highlighted by
Time Magazine as the number one medical breakthrough of 2007. By the
end of 2007, HHS had stockpiled 12 million courses of pre-pandemic H5N1
vaccine. However, maintaining a domestic production capability for
these priority countermeasures is also an essential component of the
pandemic influenza preparedness strategy. In May 2006, HHS awarded five
contracts for more than $1 billion to GlaxoSmithKline, MedImmune,
Novartis (formerly Chiron), Solvay, and Dynport (with Baxter) for
support of advanced development of cell-based influenza vaccines toward
U.S. licensure and expanded domestic vaccine manufacturing surge
capacity. In June 2007, we awarded two contracts for the retrofitting
of existing domestic biological manufacturing facilities to produce
egg-based influenza vaccines and included warm base operations for up
to 5 years. Additionally, contract awards were made in 2008 for the
construction of new domestic facilities for manufacturing cell-based
influenza vaccines that is expected to quadruple the domestic pandemic
vaccine manufacturing surge capacity by 2012.
A robust and groundbreaking advanced development program has led to
the rapid maturation of modernized cell-based influenza vaccine
production and antigen-sparing technologies. New combinations of
adjuvants and products provided by multiple manufacturers are currently
supported by performance-driven milestone contracts. More rapid vaccine
production may be afforded by the development of next generation
recombinant influenza vaccines, which HHS will support.
These investments enhanced our current capabilities to respond to
the urgent needs for development and manufacturing of vaccine for use
against 2009 H1N1 influenza. Currently, HHS has contracted with five
companies that are now producing and conducting clinical trials or 2009
H1N1 vaccine for U.S. supply (GSK, Sanofi, Novartis, CSL, and
MedImmune).
medical countermeasure analysis of alternatives
In July 2008, Dr. Robert Kadlec, the then-Special Assistant to the
President for Biodefense and Senior Director for Biodefense at the
Homeland Security Council, requested that HHS and DOD conduct an
Analysis of Alternatives:
``to identify the optimal facilities and operating model for
addressing the gap in production and manufacturing of medical
countermeasures against WMD [Weapons of Mass Destruction]
threats in a manner that provides the best long-term value to
the U.S. Government.''
It is important to highlight that the inspiration behind the
development of such a biofacility is consistent with many of the broad
goals articulated in the draft National Vaccine Plan (November 2008),
especially regarding the objectives of
--Fostering advanced research and development toward new and/or
improved vaccines that prevent diseases, including those that
protect against emerging, re-emerging, and important
Biodefense-related pathogens, and
--Improving access to appropriately designed pilot lot manufacturing
facilities that produce clinical grade material for promising
vaccine candidates.
As Dr. Kadlec noted, the timely availability of sufficient
quantities of medical countermeasures ``is essential for saving the
lives of civilians and warfighters following a WMD attack.''
A principal mission of HHS and DOD is to provide medical
countermeasures--drugs, vaccines, and therapeutics--to protect civilian
and military populations, respectively, from attack with CBRN agents.
Developing sustainable medical countermeasures that are effective and
readily available is an enormously complex task from a technical and
organizational perspective.
Such medical countermeasures require:
--Discovery and early research;
--Development and testing in surrogate animal models;
--Advanced development through clinical trials;
--FDA regulatory approval;
--Production and manufacturing;
--Stockpile supply management;
--Distribution and dispensing strategies; and
--Stockpile replenishment.
To accomplish the requested analysis, HHS and DOD commissioned an
independent, third-party study of vaccine manufacturing facility
alternatives that should be considered. We will soon meet with our
colleagues at DOD to discuss the findings and determine any
recommendations that will be made in response to the request. But
permit me to briefly share some general findings with you.
The focus of the analysis was on the advanced development, FDA
approval and sustainment phases of the medical countermeasure
lifecycle. Within the advanced development phase, the focus was only on
advanced manufacturing process development. Sustainment refers to the
storage, maintenance and replenishment of the active pharmaceutical
ingredients and the dosage forms of medical countermeasures, as well as
procurement, storage, and maintenance of required ancillary supplies
needed to administer the countermeasure.
To date, the United States Government (USG) has successfully
procured small molecule medical countermeasures (e.g., drugs) from
established contractors, and the analysis determined that there is
excess industry capacity available for manufacturing these types of
products. Thus, the current process of contracting with industry to
produce `small molecule' medical countermeasures appears to be viable
now and in the future.
Together, HHS and DOD have stated requirements for a collective
portfolio of 23 large molecule CBRN medical countermeasures that are
biologically based products. Past analyses have recommended that the
USG own and manage a production facility for the manufacture of large
molecule products (e.g., vaccines, monoclonal antibodies) to increase
control of the approval and supply processes in order to minimize risk
of supply disruption.
Since the biopharmaceutical industry, as a whole, has not
traditionally developed medical countermeasures for the USG, there is a
perception that large gaps exist in manufacturing and production
facilities. However, recent history shows that both HHS and DOD have
successfully contracted with emerging biotechnology innovators and
contract manufacturers for advanced development and procurement of
medical countermeasures. Moreover, some USG contractors are investing
heavily in production facilities, the majority in the United States,
further addressing the facilities gap.
The analysis suggests that the USG should increase its capabilities
to oversee the advanced manufacturing process development and supply
programs to ensure that,
--Needed medical countermeasures achieve FDA approval, and
--There is an ongoing supply of the product.
alternative scenarios examined
Three alternative scenarios for the development, approval,
manufacturing, and sustained replenishment of large molecule medical
countermeasures were examined using both quantitative and qualitative
criteria.
--The first alternative examined was continuing the existing process
of contracting the development and manufacture of medical
countermeasures.
--The second alternative examined was continuing the existing process
while strengthening technical, quality/regulatory, and
sourcing/supply capabilities; and contracting with additional
manufacturers of bulk active product ingredients and
formulation, filling, and finishing. This alternative also
provides for enhanced access to process development and
manufacturing capabilities.
--The third alternative is one that has been proposed in previous
studies. It calls for the Government to manufacture all needed
medical countermeasures. This approach, which includes
establishing a new public-private partnership, anticipates a
fully dedicated manufacturing facility for all medical
countermeasures under control of the USG.
The first alternative only satisfies the need of the USG to a
limited degree because, although the USG has been successful in
developing and manufacturing some medical countermeasures, it does not
provide the USG with the most effective and efficient processes for
managing the potential growing number of highly complex medical
countermeasures. While this alternative is the least costly of the
three alternatives, it provides the fewest capabilities and carries the
risk of less than optimal oversight to ensure the manufacturing
capability for the growing medical countermeasure supply chain.
The second alternative builds on the successful current USG medical
countermeasure contractual approach and enables flexible decision
making for advanced manufacturing process development, stockpiling,
backup, and surge. It expands current capabilities to meet the
complexity and urgency of medical countermeasures yet to be developed
and scaled to manufacturing requirements. This alternative offers the
lowest operational risk to achieve current requirements of all the
alternatives by creating a collaboration of USG and a network of
incentivized, highly specialized, and knowledgeable industry suppliers.
It enables future operations to be enhanced most efficiently by
incorporating dedicated technology, quality and regulatory compliance,
and sourcing and supply functions. In addition, contract manufacturing
is less costly and timelier than constructing and operating a dedicated
facility.
Of all alternatives examined, the third alternative potentially has
the highest risk of supply chain failure and, compared to the two other
alternatives, carries the highest cost. In addition, the time required
to develop reliable systems for long-term, full-scale manufacture of
biologics (large molecules) could impede progress toward needed FDA
approval. This alternative also does not provide sufficient surge or
backup capabilities. Finally, only a few, if any, biopharmaceutical
companies have clustered the production of so many products in a single
facility. An adverse regulatory decision or a catastrophic event could
shut down the single facility. A single facility with many products is
more complex to manage, and is more likely to trigger an adverse
regulatory decision than a network of specialized manufacturers.
conclusion
HHS is committed to protecting the health and safety of American
citizens from CBRN threats. Along with our colleagues at DOD, HHS is
committed to a full examination and discussion of all viable options
for the manufacture of vaccines and other medical countermeasures
against identified threats.
We appreciate your support and continuing interests in this
important area. I am happy to answer any questions.
Senator Specter. Well, thank you, Dr. Gellin, for that
opening statement.
Dr. Gellin, in looking at the statistics as of 1 week ago
yesterday with 7,511 hospitalized cases of swine flu in the
United States and 477 deaths in the United States and 1,799
deaths worldwide and the prospect of having serious problems of
additional cases of swine flu, I was very concerned to see the
report on Monday of this week that the companies were only able
to produce 45 million doses of vaccine for swine flu when the
expectation had been for 120 million vaccine doses.
Now, is there not really a very substantial public risk
when there has been that shortfall on the number of vaccines
available for this very serious problem?
Dr. Gellin. You are right. We were dismayed to learn that
as well. We have been working with the manufacturers to try to
determine what the expected number of doses would be. This to
me, as I said, highlights how complicated this kind of
manufacturing is.
Senator Specter. And are there not major problems in
looking for manufacturers because the big companies do not want
to undertake manufacturing these kinds of vaccines because
compared to their other business where they have enormous sales
and very substantial sums of money, that these are relatively
small and not attractive for other big companies?
Dr. Gellin. Exactly. I think we should put separately the
influenza discussion because those vaccines are being made by
the same manufacturers that make influenza vaccine around the
world. What we are focusing on here is exactly as you say,
Senator, looking at the manufacturing for products that do not
have a large commercial market.
Senator Specter. So what are we talking about is smallpox,
anthrax, ebola virus, botulism, and others where there could be
a very serious public health problem if terrorists, for
example, were to unleash smallpox germs on the community.
Dr. Gellin. It is exactly the same. There is a list of
those threats and there is a need to make sure that we can have
the products that we need should we have to face a threat like
that.
Senator Specter. Dr. Gellin, when you testify, as you did,
about Federal Government oversight, is that sufficient? If you
do not have a controlling voice in what the company is going to
do and you deal with somebody in Australia, it is
understandable that the Australian company wants to look after
Australia first. I do not like it, but you have to expect that.
Or if you find a company that you have contracted with is going
to take care of their regular business first and their
interests, not in the public interest, I do not like that
either, but that is to be expected.
But does there not have to be something more than
governmental oversight? Does there not really have to be
something like a public/private partnership where the
Government is able to control what the producers are going to
do, looking out after U.S. health interests?
Dr. Gellin. I think you are exactly right. And the
situation you describe really underscores at least our approach
with influenza, as you have mentioned, and the generous support
the Congress has provided to develop domestic-based facilities.
I think that is clearly a part of this. I think there is a
range of possible solutions to this problem, of which the
public/private partnership is clearly one.
Senator Specter. And when you have a need to change
equipment on vaccines and the standard equipment on stainless
steel requires a good bit of cleaning and down time and delay,
isn't GE in a unique position having the patent on the plastic
so that they are in a position to move from one production of
vaccine to another?
Dr. Gellin. I cannot speak to GE specifically, but----
Senator Specter. Well, we will hear from them this morning.
Dr. Gellin. But GE--like many--I think--we are encouraged
that a number of companies are taking this seriously and are
trying to move us into the 21st century with some of these
production techniques.
Senator Specter. Has any entity as prominent as UPMC with
its very prestigious, very effective operation been as
innovative and thoughtful in coming up with a proposal to deal
with this very serious national problem?
Dr. Gellin. I am certainly impressed by the UPMC group
proposal, and again, coming into Pittsburgh, it is hard to not
see the footprint that UPMC has here. What they have put
together, as the Congressman has highlighted, really represents
the range of the strengths that they bring to it.
Senator Specter. Well, my red light is on the red line, but
I will ask you in addition to being impressed, which you
testified to, my question was, has anybody like UPMC come
forward to tackle this problem in this imaginative, innovative
comprehensive way. I want to hear more than whether you are
impressed with them. I want to know if anybody else is doing
what they are doing.
Dr. Gellin. There are a lot of discussions about this from
a procurement standpoint. So I am not aware of other offers
like this. There may be----
Senator Specter. Okay. The answer is none that you are
aware of.
Dr. Gellin. Correct.
Senator Specter. Well, you are the witness.
Dr. Gellin. I am the witness and----
Senator Specter. Congressman Altmire.
Mr. Altmire. Thank you, Senator, and thank you, Dr. Gellin.
I read your written remarks which, of course, in 5 minutes
you cannot go into everything, and you were very thorough. And
I wanted to ask you a couple questions that you address in
there.
You outlined the options in your oral statement right now
that people are talking about, but without picking a favorite
option, what is your opinion about the need to restructure the
process in a way that maybe better adapts to modern technology
and modern threats so that we are not constantly being in that
responsiveness frame of mind that we have talked about?
But when you do have to respond quickly, is it fair to
say--I am not going to put words in your mouth. I am asking for
your opinion--that we need to do a comprehensive restructuring
of the process or that we just need to keep doing what we are
doing and maybe just refine it a little bit?
Dr. Gellin. To pick up where you left off, I think we need
to take a fresh look at the way we are approaching this. What
is comprehensive is, I think, in some ways in the eye of the
beholder, but I think that we have identified that there are
some substantial gaps. We need more flexibility in our
approach, and we need to be more fluid. In my assessment of the
assessment of the variety of alternatives, that is what they
are looking at as a way to be able to close some of these gaps.
Mr. Altmire. With regard to the American role, public/
private partnerships in this process, I think the Senator has
accurately said some things that are of concern to the Congress
generally, that when you have foreign entities involved, even
though they are allies, they are going to look out for
themselves first. So when we talk about the idea of putting
forward large sums of money, $600 million or more, toward this
effort, would it be your opinion that that would be money well
spent if we were to look at the idea of having one leading
center in the country that would have the expertise and all the
assets that we talked about, regardless of where in the country
that is located, versus having--I think you referred to this as
an option--a bunch of co-equal centers around the country that
do the same thing, but maybe the coordination, in my opinion,
may not be what we would like if we had to respond very
quickly?
Dr. Gellin. Yes. I cannot speak to that directly. I think
that people make an argument on both sides of that. And my
experience with the NIH on the research side is they have a
number of Centers of Excellence and they are able to tap into
the expertise of different places on common problems. So again,
I think there are a number of approaches to this, and I think
that that is where the discussions between the leadership of
HHS and DOD I think will be quite helpful in trying to figure
out what is the right balance and what is the right structure.
Mr. Altmire. Thank you. I have no further questions,
Senator. I have a couple of minutes left. I would be happy to
yield to you if you had one more, or just yield back.
Senator Specter. Well, we are not going to let any time go
unused. So thank you.
Dr. Gellin, how do you see the matter progressing? I
believe that there will be considerable support in the Congress
for the authorization and then ultimately for the
appropriation. What next steps do you see in the consideration
of this issue? And I ask that in the context that I want to see
us move ahead promptly. I do not want to see the bureaucracy
slow it down. To what extent will the Office of Management and
Budget be a block to getting the necessary funding?
Dr. Gellin. So I think it is clear, as I mentioned, that
this now--the analysis has been done. The discussions have
occurred at the technical level and it has got to be briefed up
the chain of the respective Secretaries. That then because of
the importance of this contract is going to obviously include
White House conversation in which, at least in my experience,
OMB is always a part of those conversations.
Senator Specter. Can you give us some insights as to what
the report has shown? I realize it has not been made public,
but I know there is tremendous interest in knowing. Describe at
least the parameters of the report and what it has been
designed to seek.
Dr. Gellin. Well, you have actually highlighted the broad
strokes of what that report has shown. I tried to emphasize
some of that in my testimony. Do we continue similarly to what
we are doing now? Do we have a little bit more of a hybrid
approach of doing what we are doing now but having additional
ability to add more control? And then the third possibility is
the possibility of having a place where this goes on. So I
think that is what is out there for discussion. The analysis
has been done. The analysis has to go up to the leadership to
try and figure out the best route forward.
Senator Specter. Does the executive share the sense of
urgency, which the subcommittee feels on this subject? A sense
of urgency to move ahead and get something done?
Dr. Gellin. Merely because we are in the middle of the H1N1
problem does not mean that everything else is not a problem
right now. We recognize that a range of problems exist. The
gaps that have been identified exist, and we need to try to
close those gaps.
Senator Specter. Thank you very much, Dr. Gellin. The red
light is on and we will move now to panel two. We would
appreciate it if you would stand by because there may be some
questions which will arise from the testimony of our
distinguished panel which you could shed some additional light
on.
We now call CEO and President of UPMC, Mr. Jeffrey A.
Romoff, retired Major General Philip K. Russell, Dr. Philip
Gomez, Dr. Donald Burke, and Dr. Nigel Darby.
Thank you very much for coming, gentlemen. As noted a
moment or two ago, in accordance with our general practice, the
subcommittee allocates 5 minutes for opening statements to give
a maximum amount of time for questioning and answers.
We turn now to our first witness, Mr. Jeffrey A. Romoff,
President and CEO of UPMC, one of the leading nonprofit health
systems in the United States. He began his distinguished career
at the University of Pittsburgh in 1973 as Director of the
Office of Education and Regional Programming at Western
Psychiatric Institute and Clinic. In 1992, he was elevated to
the presidency of UPMC, and in 2006 became UPMC's President and
CEO. A master degree in philosophy with a specialty in
political science at Yale University, recipient of the honorary
Doctorate of Public Service from Chatham College, and an
honorary Doctorate of Science and Technology from Carnegie
Mellon University. Welcome, Mr. Romoff, and we look forward to
your testimony.
STATEMENT OF JEFFREY A. ROMOFF, PRESIDENT AND CHIEF
OPERATING OFFICER, UNIVERSITY OF PITTSBURGH
MEDICAL CENTER, PITTSBURGH, PENNSYLVANIA
Mr. Romoff. Thank you very, very much, Senator Specter.
Thank you, Congressman Altmire, for being here.
I have submitted testimony that provides an overview of
UPMC's perspective on this problem, and we have also submitted
a summary of the 21st century biodefense project.
Senator Specter. All of that will be made part of the
record, as will all of the formal statements.
Mr. Romoff. Also, I am privileged here to be a member of a
panel with far more distinguished and knowledgeable scientists
and chem specialists. So I will seek to address issues as you
would address issues rather than get into many of the details.
The first issue that is so absolutely essential here is the
issue of preparedness, which you, Senator, highlighted in the
previous testimony. It is our view and I think it is the view
of most commentators and most observers that when it comes to
the protection of the national security from a bioterrorist
attack, all of the protection of the national security and the
national well-being from various naturally occurring infectious
diseases, this country and virtually all countries in the world
are relatively defenseless. We can talk about why and how, but
this is a very, very grave problem and one that we do not often
realize regrettably until after the fact.
It is not just a problem of finding the right medical
countermeasures or vaccines to address the problem, should
there be a natural accident, as we already saw, or should there
be SARS, as we already saw. It is even a more serious problem
because the public psyche is highly vulnerable. It is always
vulnerable. It was vulnerable after 9/11. It was certainly
vulnerable after Katrina, and now coming out of, hopefully, the
recession, we see that something that happens unexpectedly,
something that shocks like a bioterrorist attack or a lot of
what happened with the subprime causes ripple effects that are
even more profound than the initial occurrence.
The most serious harm for this country that will come from
even a small bioterrorist attack would likely not be the
casualties that occurred initially, but the harm will come in
the school systems, in the workplace, in the economy, in all
the aspects of our daily living because when we do not have the
confidence that our Government or that our corporations or that
our society is well-prepared to deal with something that is
unknown and frightening, we lose confidence completely.
I find it fascinating that yesterday there were statistics
that only, if you will forgive the expression, 15 percent of
the subprime mortgages actually went into foreclosure. Now,
this country reacted extraordinarily to the fact that subprime
mortgages were very, very vulnerable. Now, the facts now come
out that it was a limited damage in itself, but the damage to
the economy, the fact that the credit system came to a
screeching halt is profound.
We are now watching the H1N1 situation, and that is a
situation where I believe, despite this last finding that they
were not making enough vaccine as anticipated, the Government
deserves kudos because as soon as the H1N1 emerged, Secretary
Sebelius, Secretary Napolitano, the CDC got in front of the
public and said we are on top of this or we are learning about
it. This is what we are doing, and it calmed the public and it
raised the expectations. Now, of course, if the expectations
are not completely met, then we have a secondary set of
problems.
PREPARED STATEMENT
If this Government, if there is a bioterrorist attack or
there is a new infectious disease, cannot stand before the
public and say straightforwardly we anticipated this--and it is
anticipated. It was anticipated in the Weapons of Mass
Destruction Commission report which says there is a reasonable
chance that in 5 years--that is, 5 years from a year or 2 ago--
that there will be a bioterrorist attack. If the Government
cannot stand before the people and simply state we anticipated
this, we knew about it, and this is what we did, we caused
these things to happen, not that we caused these studies and
analysis to happen, but that we caused these concrete
approaches, just like we caused new--about H1N1 for now, then I
believe beyond whatever attack occurs, there will be a
significant, significant diminution in the confidence of the
Government and a diminution in the competence of the
Government. This, of course, is what we saw after Katrina.
So in conclusion, I thank you once again for caring about
these very, very important issues, and UPMC stands absolutely
ready to do everything we can if we have the opportunity.
[The statement follows:]
Prepared Statement of Jeffrey A. Romoff
Thank you, Senator Specter, for the opportunity to provide
testimony on the vital issue of improving the Nation's capacity to
develop and manufacture countermeasures critical to national and
homeland security. I'd also like to recognize and thank Congressman
Jason Altmire (and Congressman Mike Doyle).
I am here today in my capacity as president and CEO of the
University of Pittsburgh Medical Center (UPMC), the University of
Pittsburgh Medical Center. UPMC is a unique organization that has, over
the past two decades, evolved from an outstanding academic medical
center into an $8 billion integrated global health enterprise. We have
50,000 employees and are the largest employer in western Pennsylvania.
We act as a major health resource for residents of the western
Pennsylvania, and contribute more than $500 million annually in
community benefits. UPMC is closely affiliated with the University of
Pittsburgh, which ranks fifth in National Institutes of Health (NIH)
research funding.
One of the key roles that UPMC plays is revitalizing the economy of
western Pennsylvania by nurturing new capabilities derived from our
intellectual capital and based on medicine, science, and technology.
UPMC's core expertise in this regard is vividly demonstrated by the
new $622 million, state-of-the-art Children's Hospital of Pittsburgh of
UPMC, where we have deployed a fully integrated electronic health
record, developed in collaboration with and for the doctors, nurses,
pharmacists, and infection control practitioners at the hospital.
Children's is among the only 1.5 percent of the Nation's hospitals that
use a comprehensive electronic record, and the care provided is among
the finest, safest, and most cost-effective available today.
Our expertise and capacity for innovation are also evident in the
Hillman Cancer Center, which acts as the hub of a network of cancer
centers throughout western Pennsylvania. These centers are connected
through sophisticated linkages that enable the most advanced forms of
radiation therapy to be delivered to patients here and abroad.
In a similar vein, one of the telemedicine programs that we have
implemented now provides the expertise of stroke specialty consultants
to physicians and patients in distant community hospital emergency
departments without the risks attendant in spending valuable time in
transit to larger hospitals.
In Italy, a decade ago, in collaboration with two hospitals and the
Region of Sicily, we brought transplantation and other specialty
surgical care to Palermo at a level never before available in the
region. The hospital we built, which is known as ISMETT, has become a
leader in transplantation in Italy. Building on the success of ISMETT,
UPMC is now working with the Italian Government on plans to create a
new biomedical and biotechnology center in Sicily, in collaboration
with the University of Pittsburgh, the Italian Council of Ministers,
the Region of Sicily, and the Italian National Research Council.
Obviously, we are proud of each of these accomplishments, but what
do they have to do today's topic, ``Advanced Development and
Manufacturing for U.S. Biodefense?'' I would submit that these
seemingly unrelated initiatives share a number of common threads.
First, each of these examples demonstrates that extraordinary
achievements can be brought about through out-of the-box creativity and
innovation. This creativity should be applied to the challenge of
protecting this Nation from bioterrorism. My colleagues on this panel
will speak to this issue in greater detail.
Let me just say that UPMC has had a long-standing commitment to
serving the Nation and advancing our readiness in the area of
biosecurity. In 2003, we founded the Center for Biosecurity of UPMC, an
independent, academic think tank dedicated to providing research,
analysis, and policy solutions to address national and international
biosecurity challenges. We have been at the table in a wide array of
regional readiness efforts, and most recently have been conducting
comprehensive analysis, in collaboration with DARPA, to assess the U.S.
Government's biodefense countermeasure requirements and the Nation's
infrastructure in place to meet those requirements.
The second theme that our experience illustrates is that by
departing from traditional paradigms, one can develop new and effective
solutions. This is as true in biodefense as in electronic health
records or telemedicine.
We are already seeing growing recognition that biological weapons
and naturally occurring pandemics represent a grave risk to the health
of the populace and to the continued economic recovery of this Nation.
Over the past decade, we have all seen official Government report
after report cite biological weapons as being among the top national
security threats to the Nation. A recent Weapons of Mass Destruction
Commission report concluded that a biologic or nuclear attack somewhere
in the world is more likely than not in the next 5 years. President
Obama recognized biological threats as a major national security issue
during his campaign and has committed to improving U.S. biosecurity
since taking office. Congress also has worked to improve the state of
U.S. biosecurity.
In particular, the central importance of countermeasures--medicines
and vaccines--to U.S. biosecurity has been recognized. The Obama
administration has expressly committed to ``accelerate the development
of new medicines, vaccines, and production capabilities.''
It is also recognized that the traditional platforms for developing
solutions have not yielded the biologics, vaccines, and countermeasures
that are required.
It is not because the Nation lacks great scientific knowledge or
new ideas--our universities are brimming with new leads and new
directions, but they lack the ability to bring these great ideas to
market.
It is not for lack of superb pharmaceutical companies that have the
top-level industry knowledge and experience to develop, license, and
manufacture biodefense countermeasures. There is no commercial market
for these products outside the Government, there are substantial
opportunity costs for these companies, and they have largely not seen
the Government as a predictable partner in this enterprise.
As a consequence, the current U.S. approach to biodefense medicine
and vaccine development relies almost completely on small biotech
companies. These companies are innovative and focused, but few have
demonstrated the capability to produce licensed vaccines or medicines,
and few have in house the technical expertise and/or regulatory
experience needed to do this work. These companies must raise money to
build dedicated factories to make these products. They must have the
plans and capacity to manufacture a batch of their products annually to
maintain their FDA license, with the result that many of these
manufacturing plants will be idle most of the year, with a very limited
ability to surge production.
It is in this context that I would strongly urge this subcommittee
to initiate a new public-private partnership within HHS BARDA, the
mission of which would be to establish and run flexible, multiproduct
medical countermeasure development and manufacturing facilities that
would address these issues and challenges.
This public-private partnership would make maximal use of flexible
technologies that do not require building highly capital-intensive
facilities. It would be capable of developing and manufacturing
multiple products concurrently in different suites, using disposable
technology that can easily be changed depending on the needs and
requirements of the Government. In time of national crisis, such as
after a substantial bioattack on a U.S. city, all suites of the
multiple-suite vaccine plant could be converted to the manufacture of a
single drug, providing critical surge capacity not now available.
Another key feature of this public-private partnership would be
that it would provide and concentrate ``big pharma'' development
expertise--high-level expertise that is not easy to find for the
biotechnology companies now working to develop these products. This
would not only increase the odds of success substantially, but also
would reduce risk of failure midway through a complex product
development life cycle.
In this partnership, key responsibilities of the Government would
include setting requirements for medicines and vaccines, making
decisions about procurement, and providing part of the funding. The
private sector partner would provide the remainder of funding and bring
pharmaceutical and technological know-how, and would provide workforce
training and education. A range of professional backgrounds and
educational levels would be required to operate this facility, from top
scientific and professional training to high-school-educated workers.
The design, construction, and operation of this facility would provide
thousands of jobs.
An economic analysis undertaken by UPMC demonstrates that this
approach would save the U.S. Government--that is, American taxpayers--
$28 billion over the next 25 years, an estimated 80 percent of the
development costs for making the current DOD/HHS requirements for
vaccines and medicines. In addition, adoption of such an approach to
this challenge would help maintain the U.S. industry in
biomanufacturing, an industry that has provided excellent jobs, but
that has been steadily moving overseas and will continue to do so
unless U.S. policy changes.
Finally, this public-private partnership model can and should be
used to develop noncommercial medicines and vaccines for emerging
infectious diseases--a group of diseases that is of great importance to
the world, but for which many of the same market conditions and risks
have prevented progress.
We fully anticipate that a project of this importance and scope
would be competitively bid. In all likelihood, UPMC and its partners
would participate in this competition. We have a proven track record of
superb execution and success, but we would anticipate top-level
competition from others as well.
In closing, let me again thank you for the subcommittee's
leadership on these issues. These are critical issues for the country.
The nature and seriousness of the biological threats have been clearly
stated for years. The importance of making medicines and vaccines to
counter these threats has been similarly recognized for quite some
time. There are concrete and innovative steps the country can and
should be taking to deal with these vulnerabilities and challenges that
will make us far better prepared for a range of crises. We can make new
medicines and vaccines for these threats more quickly, more reliably,
with less risk and at less cost to the Government. The private sector
is capable of being a much more active and effective partner with BARDA
and the U.S. Government on these efforts. In the last year, there has
been growing discussion in Washington about the vital importance of
establishing new public-private partnerships to solve problems that
neither the Government nor the private sector can solve alone. My
judgment is that countermeasure development and production is the
archetype of such a problem, and that the elements of the public-
private partnership I have described in this testimony are central to
addressing it.
Senator Specter. Thank you, Mr. Romoff.
We turn now to Dr. Philip K. Russell, Trustee and Senior
Scientific Advisor to the Sabin Vaccine Institute in
Washington. He served in the U.S. Army Medical Corps from 1959
to 1990, some extended period of time. Following the anthrax
attacks in 2001, Dr. Russell led an HHS effort to develop and
stockpile vaccines and other medical countermeasures against
bioterrorism agents. Thank you for coming in today, Dr.
Russell, and we look forward to your testimony.
STATEMENT OF PHILIP K. RUSSELL, M.D., RETIRED MAJOR
GENERAL, ARMY MEDICAL CORPS; BOARD OF
TRUSTEES AND SENIOR SCIENTIFIC ADVISOR,
SABIN VACCINE INSTITUTE, WASHINGTON, DC
Dr. Russell. Good morning, Senator, Congressman. Thank you
very much for the opportunity to be here today and provide my
views on the urgent need for a major improvement in the
capability of the U.S. Government to develop and acquire
biomedical countermeasures that are needed to protect our
citizens against bioterrorism.
The experiences that I have more than 40 years of research
and development in infectious diseases, including senior
leadership positions in both the U.S. Army and HHS medical
biodefense programs, has provided me with some insight into the
deficiencies of our current programs and the urgent need for
change.
The successful development and licensure and utilization of
several vaccines by the Walter Reed Army Institute of Research,
including meningitis type C and type A--vaccines, unattenuated
adenovirus type 4 and 7 vaccines, and hepatitis A vaccines,
were made possible by several critical factors. First, a
recognized need and adequate funding. Second, a strong internal
research base including scientists capable of process
development. An associated availability of a pilot-scale GMP
manufacturing capability. Partnerships with large vaccine
manufacturers, and clinical trial capability.
Without these elements, those complex vaccine development
programs would not have succeeded.
Another vaccine, the leading malaria vaccine, now
undergoing a final phase III trial in Africa, and a similar
product--development program in partnership with GSK.
Industrial partnership plus adequate funding from the Gates
Foundation is critical to this important vaccine.
The success of the military in developing vaccines against
recruit camp diseases and natural disease threats has not been
matched by success in developing medical countermeasures
against biologic threat agents, although we have made enormous
investments in basic research, notably in DARPA, and the NIH
program, and the Centers of Excellence. We have not been able
to translate the results of that research into new products
licensed for use. This is in spite of the magnitude of the
threat of bioterrorism and its potential devastating impacts on
our Nation.
The deficiencies of the DOD biodefense program, as well as
regulative changes that have been outlined by several
independent studies and reports over the past 2 decades
beginning with the Tri-Service Task Force report in 1990. It
included an independent study known as the--Report in 2001 and
two Institute of Medicine studies, the last in 2004. The common
themes of the recommendations in the reports were manufacturing
capacity and the need for industrial capability.
In HHS there is a similar problem. The gap between basic
research and industrial development and manufacturing, is only
partly felt by BARDA funding, that is dependent on an
increasingly reluctant and fragile biotechnology industry. The
very impressive results that HHS has had with influenza
vaccines underlines two issues: capability that exists within
the vaccine industry if properly utilized and the fact that
vaccine manufacturing is increasingly moving overseas.
The success of HHS's biodefense field is the very rapid
development and licensure of ACAM 2000 smallpox vaccine. That
was made possible by very solid funding from Congress, a
partnership of the small developer with a large pharmaceutical
manufacturer with bulk manufacturing capability in Europe and a
strong capability in process development and regulatory
affairs. Process development capability, as well as
manufacturing capability and surge capacity, are necessary to
provide the biologic products needed to counter the very real
threat of bioterrorism. The best way to provide that capability
within the context of a biodefense program is a facility
designed and managed to be responsive to Government needs.
PREPARED STATEMENT
A very important additional benefit would be the capability
to develop and manufacture vaccines against emerging infectious
diseases such as Rift Valley Fever, chikungunya, and
hemorrhagic fever. That would be a very valuable contribution
to global health and to medical diplomacy, as well as meet the
response to something like the next SARS epidemic.
Thank you for your time. I will be happy to answer
questions.
[The statement follows:]
Prepared Statement of Philip K. Russell
Good morning Senator Specter, thank you for the opportunity to
appear here today and provide my views on the urgent need for
improvement in the capability of the U.S. Government to develop and
acquire biologic medical countermeasures needed to protect our citizens
against bioterrorism. I am Dr. Philip Russell, a retired Army Medical
Corps Major General. My medical specialty is infectious diseases and I
have been involved in research and development for my entire medical
career. For 2\1/2\ years following September 11, 2001, I served as a
senior advisor to the Department of Health and Human Services (HHS). In
that capacity I was deeply involved in the acquisition of medical
countermeasures against smallpox, botulism, and anthrax, as well as the
experimental H5N1 avian influenza vaccine. As Acting Director of the
Office of Research and Development Coordination within the Office of
the Assistant Secretary for Public Health Emergency Preparedness I was
responsible for coordination of the initial purchases made under
Project BioShield.
During my military service, I served as Director of Walter Reed
Army Institute of Research (WRAIR) and later as Commander of the U.S.
Army Medical Research and Development Command. I was responsible for
the management of the biodefense research and development program from
1985 until 1990. As a research scientist and R&D manger, I was involved
in the successful development of several licensed vaccines including
meningitis, adenovirus and hepatitis A vaccines by the U.S. Army as
well as the development of the ACAM 2000 smallpox vaccine. Through this
experience I learned what is needed to move a potential new medical
product from the laboratory through the development process to
licensure, manufacturing and utilization.
The successful development of several vaccines by WRAIR was made
possible because of several critical factors:
--A recognized need and adequate funding;
--A strong internal research base including scientists capable of
process development;
--Pilot-scale GMP manufacturing capability;
--Partnerships with large vaccine manufacturers; and
--Clinical trial capability.
The success of the military in developing vaccines against recruit
camp diseases and other natural disease threats, however, has not been
matched by success in developing medical countermeasures against
biological threat agents.
The investment in basic science by the Department of Defense (DOD)
through the service laboratories, notably the U.S. Army Medical
Research Institute of Infectious Diseases (USAMRIID), by contractors,
as well as the immense investment by NIH through its university Centers
of Excellence, has produced a wealth of new technical opportunities for
development of new and improved vaccines and therapeutics to counter
the very real threat of bioterrorism. Yet in spite of the magnitude of
the bioterror threat, neither DOD nor HHS has been very successful in
moving potential products from the research laboratory to the field or
to the national stockpile. We still mainly depend on products developed
in the 1950's and 1960's for prevention of casualties while the results
of research efforts by both the basic scientists and the biotechnology
companies go largely undeveloped.
Both DOD and HHS depend on product development through contracts
with contractors that lack the experience, capabilities and resources
of the large vaccine manufacturers. The large manufacturers have shown
little interest in the high-risk, low-volume, and low-profit medical
countermeasures needed to combat bioterrorism.
Several studies of the DOD program have detailed the weaknesses
inherent in the current approach. In 1990, a DOD task force entitled
Project Badger (``Tri-Service Task Force for the Expansion of the
Industrial Base for Production of Biological Defense Vaccines'')
analyzed the shortage of medical countermeasures for anticipated
threats.\1\ Continuing concerns over the lack of a stable pipeline of
medical countermeasures to protect troops led to the creation of
another task force to focus on assessing the need for a Vaccine
Production Facility (VPF). This additional task force was to determine
a solution for DOD biodefense medical countermeasure manufacturing.
---------------------------------------------------------------------------
\1\ Chronology of Project Badger (Long Term). October 24, 1990.
CMAT Control # 1998337-0000036.
---------------------------------------------------------------------------
In 1993, this VPF task force recommended a Government-owned,
contractor-operated (GOCO) facility that could manufacture a variety of
medicines and could surge production in times of crisis.\2\ The task
force recommendation reflected the view that the private sector lacked
the means to provide medical countermeasures to the military on its own
without adequate incentives. The choice of a GOCO model also reflected
a then common DOD acquisition strategy to procure military equipment
(e.g., ammunition, tanks) from GOCO facilities. A high-level conceptual
design of the facility proposed by the task force was also
completed.\3\ At the time of the recommendation DOD concluded that the
VPF concept was too costly to implement.
---------------------------------------------------------------------------
\2\ DOD Vaccine Production Facility Task Force Final Report, U.S.
Department of Defense, 1991 (draft), 1993.
\3\ DOD Vaccine Production Facility. LF-0445.00. Conceptual Design
Submittal. Life Sciences International. November 27, 1993.
---------------------------------------------------------------------------
DOD vaccine acquisition strategy then evolved to a prime systems
contractor approach, one in which a single contractor is dedicated to
the development and licensure of biologic products. This was executed
in anticipation of the biopharmaceutical industry ultimately supporting
DOD production requirements. Over time, however, very little commercial
interest in producing biodefense medical countermeasures emerged, thus
DOD still had no assurance that existing producers would provide
vaccines and novel medical countermeasures.
Consequently, the prime systems contractor approach proved
insufficient. Biopharmaceutical companies were discouraged from medical
countermeasure development by such factors as low profit margins, the
risk of liability for adverse reactions to the products, marginal
Federal funding for medical countermeasure programs, and inconsistent
U.S. Government priorities for product acquisition. Examples of that
troubled process include the loss of availability of Wyeth
Laboratories' adenovirus vaccine in 1996, which caused an increase of
respiratory disease in military trainees; the loss of the Greer
Laboratories' plague vaccine in 1997, which had proven extremely
effective in Vietnam against bubonic plague; and temporary loss of
Bioport's (now Emergent Biosolutions) anthrax vaccine in 1997.
In July 2001, a study by an independent panel of experts provided
the Report on Biological Warfare Defense Vaccine Research and
Development Programs to the Deputy Secretary of Defense.\4\ The report
recommended the overhaul of DOD biodefense program management and the
construction of a GOCO VPF, advising integration with the industry and
the national scientific community. The recommendations in the report
were not implemented.
---------------------------------------------------------------------------
\4\ Report on Biological Warfare Defense Vaccine Research and
Development Programs.
---------------------------------------------------------------------------
In 2004, the Institute of Medicine and National Research Council of
the National Academies completed a report critical of DOD's efforts in
developing drugs and vaccines against biological agents.\5\ This
report, entitled Giving Full Measure to Countermeasures describes the
substantial efforts to develop new drugs, vaccines, and other medical
interventions against biological agents and made recommendations for
major revisions in the structure of the DOD effort including the
creation of a new agency within the Office of the Secretary of DOD.
This subcommittee recognized the problems inherent in developing and
manufacturing medical countermeasures and the need for major changes.
---------------------------------------------------------------------------
\5\ Giving Full Measure to Countermeasures: Addressing Problems in
the DoD Program to Develop Medical Countermeasures Against Biological
Warfare Agents. Authors: Lois M. Joellenbeck, Jane S. Durch, and Leslie
Z. Benet, Editors, Committee on Accelerating the Research, Development,
and Acquisition of Medical Countermeasures Against Biological Warfare
Agents, National Research Council.
---------------------------------------------------------------------------
Major progress has been made in recent years by HHS' Biomedical
Advanced Research and Development Authority (BARDA) through the use of
the special appropriations for Project Bioshield and for influenza
preparedness. The success of the influenza vaccine program managed by
BARDA underlines the critical importance of industrial partners with
vaccine development and manufacturing experience. Influenza, however,
is a special case with significant market incentives due to an
expanding annual market.
It is very clear from reviewing the progress made in the past two
decades and the several pertinent reports from independent reviews that
major changes are needed to provide the protection the country needs
from a bioterrorist attack. It is also very clear that the threat is
very real and the countermeasures currently in the stockpile will be
insufficient to provide the protection that would be made possible by
an effective product development effort making maximum use of recent
and future scientific advances in the field.
A Government-funded capability to carry out process development,
pilot manufacturing as well as surge manufacturing capacity would be
immensely valuable and would remove the present bottleneck in
development of medical countermeasures. Industrial experience in
process development and biomanufacturing must be incorporated into any
proposed facility. In addition, a proposed facility should take
advantage of advances in manufacturing technology to achieve maximum
flexibility and surge production capability.
A Government-controlled capability to develop and manufacture
biologics for defense against bioterrorism could also provide a very
important capacity to make vaccines against emerging infectious
diseases such as Rift Valley Fever, chikungunya and hemorrhagic fevers
for both emergency use and as an important medical diplomacy option.
This could be a very important ancillary role.
I appreciate the invitation to discuss these issues and will be
happy to take questions.
Senator Specter. Thank you very much, Dr. Russell.
Our next witness is Dr. Philip Gomez, the Director of PRTM
Management Consultants and President of the 21st Century
Biodefense Industry. He holds a bachelor degree from Dartmouth,
a master of science and doctor of philosophy from Lehigh, and
an MBA from the University of Maryland. We appreciate your
joining us here today, Dr. Gomez, and we look forward to your
testimony.
STATEMENT OF PHILIP GOMEZ, Ph.D., DIRECTOR, BIODEFENSE
AND PUBLIC HEALTH PRACTICE, PRTM MANAGEMENT
CONSULTANTS, WASHINGTON, DC
Dr. Gomez. Thank you, Senator Specter, and thank you,
Congressman Altmire, for the opportunity to speak here today.
As I described in my written testimony, I had the privilege of
working in the private sector for nearly 10 years and then
working at the NIH on vaccine development for nearly 7 years.
So I hope my perspectives will be helpful in this discussion
today.
I have been working with UPMC for almost 2 years now in
examining this problem and greatly have been impressed and
thank UPMC for taking the leadership on this important issue.
What I will describe today is my support of a study that
was sponsored in a cooperative agreement with the Defense
Advanced Research Projects Agency, DARPA, which UPMC conducted
with a variety of experts on that team to perform that study.
The current U.S. Government procurement model relies on
industry to development biodefense countermeasures through the
Food and Drug Administration (FDA) licensure, followed by
procurement on a product-by-product basis as products become
available. This is an incremental approach, whereby the
Government identifies the highest priority threats and then
issues an RFP to purchase doses. History has shown, however,
that in lieu of the participation of large, well-established
pharmaceutical companies, the advanced development and
manufacture falls on the academic laboratories and innovative
small biotech companies that perform the initial research and
development of the product. Lacking extensive experience,
infrastructure, and resources, these laboratories and smaller
companies face extraordinary challenges moving candidate
medical countermeasures successfully through advanced
development that includes both clinical trials and the complex
time-consuming and costly licensure process. Because of the
limited investment in advanced development, the current
Government approach in biodefense has yielded few successes of
novel drugs or vaccines.
The UPMC study analyzed the feasibility and potential
technical and economic advantages of building capabilities for
advanced development and manufacture of biologics for the
Government. A key finding was that the Government demand does
exist for these countermeasures that have yet to be developed.
Although some countermeasures have substantial requirements
that have already been procured, for example, anthrax vaccine,
therapeutics, and the smallpox vaccine discussed, most of the
countermeasures needed for national security require much
smaller quantities, especially when compared with the demand
needed for commercial drugs or vaccines that you highlighted,
Senator. Because these lower volume countermeasures have no
viable commercial market, unlike flu, as Dr. Gellin reported,
it is left to these smaller biotechnology companies to license
and produce them over the long term.
Although the Government has had a need for many different
biologics, there is only a limited number of core suitable
manufacturing technologies to actually produce them. This now,
for the first time, technologically allows us to incorporate
these core technologies in flexible, multi-product development
and manufacturing capability. And we believe in the study this
would greatly reduce the cost and other constraints, enabling
the manufacture of medical countermeasures for both stockpiling
and, most importantly, surge production.
Implementation of this approach can be pursued via one of
many options for structuring and operating the capability.
These options range from a wholly private sector approach, a
contractor-owned and operated entity, to a wholly public sector
approach; i.e., the Government could own and operate an entity.
Our study concluded that combining the Government and private
sector resources would significantly reduce the long-term costs
and the technical and strategic risks commonly associated with
licensing and producing required medical countermeasures in a
dedicated capability.
The UPMC study included an industry outreach to determine
the factors necessary to encourage industry participation in
medical countermeasure development. The results of this
outreach indicated a preference for operating models that
enhance collaboration among all stakeholders, with the most
support aligned behind those models that include elements of
collocation of advanced development with manufacturing. This is
a very important point. Our study started with manufacturing
but quickly realized that advanced development, the process of
getting ready for FDA licensure, was a very critical aspect
that could not be ignored and only from the analysis.
Successful procurement requires the participation of both
biodefense innovators and experienced biopharmaceutical firms.
Biopharmaceutical firms have the experience, but have avoided
the U.S. Government medical countermeasure market because of
its perceived low profitability and high risk. They also cite a
critical shortage of scientists and engineers to work in this
field and that they cannot afford to assign these valuable
individuals to noncommercial projects. Therefore, a successful
dedicated capability would leverage the development expertise
of experienced biopharma while retaining the innovation of
small biotech companies and other innovators. It must also
focus on training the next generation of scientists and
engineers to develop the new drugs that NIH research so capably
supports. To succeed, it is critical that the U.S. Government
demonstrate a long-term fiscal commitment to medical
countermeasure development and procurement so all industry
partners have the economic incentive to become and remain
engaged.
The major conclusion of the study was that a flexible,
multi-product capability to accomplish advanced development and
production of biologic countermeasures would offer numerous
scientific, technical, economic, and strategic benefits not
provided by the current system. Operated as a public/private
partnership, a capability supporting both advanced development
and manufacturing would provide the Nation with a much-needed
expansion of its domestic industrial base for vaccines and
therapeutics. In addition, it would streamline the
effectiveness of advanced development while simultaneously
reducing technological risk. Analysis suggests that such a
capability would also significantly lower the Government's
costs of acquisition while enabling the Nation's first domestic
flexible surge production capability for both CBRN and
noncommercial public health threats.
PREPARED STATEMENT
Finally, in my view, it is important that the job of
executing this concept be awarded through a competitive
process. This will ensure the best-qualified coalition of
private sector partners is brought to bear in helping the U.S.
Government meet this important challenge.
Thank you for your support in highlighting these national
security and public health needs at this time. I would be
pleased to answer any questions you may have.
[The statement follows:]
Prepared Statement of Philip Gomez
Mr. Chairman, Ranking Member, Senator Specter and other
distinguished members of the subcommittee, I thank you for the
opportunity to discuss the Nation's capabilities in the area of
biologics development and manufacturing. Senator Specter, I appreciate
your leadership and focus on this issue, and hope my testimony will be
informative.
I have been fortunate to work for more than 15 years as a
biochemical engineer bringing drugs and biologics to market, both in
industry and the U.S. Government. I hold a Bachelor of Arts from
Dartmouth College in Engineering Science, a Master of Science and
Doctor of Philosophy in Chemical Engineering from Lehigh University,
and a Master of Business Administration from the Smith School of
Business at the University of Maryland.
I am currently a Director at PRTM Management Consultants, where I
work in the field of drug development, helping clients develop
operational strategies for successfully developing and manufacturing
biologics. Previously, I was employed at the Vaccine Research Center at
NIAID/National Institutes of Health (NIH), where I established the
Vaccine Production Program Laboratory in 2001. My laboratory developed
the Vaccine Pilot Plant for production of vaccines for clinical trials.
During my 6 years at NIH, my laboratory oversaw the manufacturing of
more than 40 bulk pharmaceutical compounds and more than 15 candidate
vaccines utilizing innovative collaborations with industry to advance
the development of vaccines against HIV, Ebola, Marburg, West Nile
Virus, SARS, and influenza. In 2007, I was awarded the NIH Director's
Award for the establishment of the Vaccine Pilot Plant and rapid
production of a pandemic influenza vaccine.
Prior to NIH, I spent nearly a decade in the pharmaceutical
industry at Abbott Laboratories, Sanofi Pasteur, and Baxter Healthcare
in positions of increasing responsibility, leading process/product
development organizations and project teams for multiple biologics.
In industry, I saw first-hand the enormous benefits that can flow
when the public and private sectors collaborate to address the need for
vaccines like meningitis and influenza, and I believe in areas like
biodefense, these partnerships can play a critical role in enabling the
development and production of new vaccines and drugs. I believe this
combination of NIH and industry experience qualifies me to participate
in this hearing and contribute uniquely to this discussion.
For nearly 2 years, I have been working under contract for UPMC as
it has examined the challenge of vaccine and biologics development and
procurement for biodefense, and as it has sought to identify options
for addressing that challenge. Medical countermeasures are needed to
protect military and civilian populations against chemical, biological,
radiological, and nuclear (CBRN) attacks and naturally occurring
emerging infectious diseases. As part of my work, I was assigned to
assist UPMC in conducting a Defense Advanced Research Projects Agency
(DARPA) sponsored study, which examined the U.S. Government's ability
to rapidly develop, license, and manufacture biologics required by the
Department of Health and Human Services and the Department of Defense.
The study was performed under a Cooperative Agreement with UPMC and
DARPA. DARPA is currently reviewing the final report and has therefore
not yet released the full report to the public. However, I am pleased
to provide a high-level overview of the scope and findings of the study
as submitted. Please keep in mind that the study content does not
necessarily represent the position or the policy of the U.S.
Government, and no Government endorsement should be inferred.
The current U.S. Government procurement model relies on industry to
develop biodefense countermeasures through Food and Drug Administration
(FDA) licensure, followed by procurement on a product-by-product basis
as products become available. This is an incremental approach, whereby
the Government identifies the highest priority threats and then issues
a request for proposal to purchase doses. History has shown, however,
that in lieu of the participation of large well-established
pharmaceutical companies, the advanced development and manufacture
falls on the academic laboratories and innovative small biotech
companies that performed the initial research and development of the
product. Lacking extensive experience, infrastructure, and resources,
these laboratories and smaller companies face extraordinary challenges
moving candidate medical countermeasures successfully through advanced
development that includes both clinical trials and the complex, time-
consuming, and costly licensure process. Because of limited investment
in advanced development, the current Government approach in biodefense
has yielded few successes for novel drugs or vaccines.
The UPMC study analyzed the feasibility and potential technical and
economic advantages of building capabilities for advanced development
and manufacture of biologics for the Government. A key finding was that
Government demand exists for CBRN biologic countermeasures that have
yet to be developed. Although some countermeasures have substantial
requirements that have already been procured (e.g., anthrax vaccine and
therapies, smallpox vaccine), most countermeasures needed for national
security require small quantities when compared to quantities needed to
fulfill the demand for a commercial drug or vaccine.
Because these lower volume countermeasures have no viable
commercial market, it is left to the small biotech companies to
develop, license, and produce them over the long term.
Although the Government has a need for many different biologics,
there is only a limited number of core technologies suitable for
manufacturing them. Incorporation of these core technologies in a
flexible, multi-product, development and manufacturing capability would
reduce cost and other constraints, enabling the manufacture of medical
countermeasures for both stockpiling and surge production purposes.
Recent technological advances in disposable manufacturing equipment and
associated changes in the regulatory environment have greatly enhanced
prospects for a multi-product capability by both reducing the overall
capital costs and the time necessary to change over from producing one
biologic to another. It also provides additional production capacity
with reduced technical and strategic risks.
Implementation of this approach can be pursued via one of many
options for structuring and operating the capability. These options
range from a wholly private sector approach (i.e., a contractor-owned
and -operated entity) to a wholly public sector approach (i.e., a
Government-owned and operated entity). The study concluded that
combining Government and private sector resources would significantly
reduce long-term costs and the technical and strategic risks commonly
associated with licensing and producing required medical
countermeasures in a dedicated capability.
The UPMC study included an industry outreach to determine the
factors necessary to encourage industry participation in medical
countermeasure development. The results of this outreach indicated a
preference for operating models that enhance collaboration among all
stakeholders, with the most support aligned behind those models that
include the element of co-location of advanced development with
manufacturing. Successful procurement requires the participation of
both biodefense innovators and experienced biopharmaceutical firms.
Biodefense innovators have researched promising early stage medical
countermeasure candidates, yet they often lack the advanced development
expertise to produce FDA-approved products. Biopharmaceutical firms
have this expertise, but have avoided the U.S. Government medical
countermeasure market because of perceived low profitability and high
risk. They also cite a critical shortage in scientists and engineers to
work in this field, and that they cannot afford to assign these
valuable individuals to noncommercial projects. Therefore, a successful
dedicated capability would leverage the development expertise of
experienced biopharma interests, while retaining the innovation of
small biotech companies and other innovators. It must also focus on
training the next generation of scientists and engineers to develop the
new drugs that NIH research so capably supports. To succeed, it is
critical that the U.S. Government demonstrate a long-term fiscal
commitment to medical countermeasure development and procurement so all
industry partners have the economic incentive to become and remain
engaged.
The major conclusion of the study was that a flexible, multi-
product capability to accomplish advanced development and production of
biologic countermeasures would offer numerous scientific,
technological, economic and strategic benefits not provided by the
current system. Operated as a public-private partnership, a capability
supporting both advanced development and manufacturing would provide
the Nation with a much-needed expansion of its domestic industrial base
for vaccines and therapeutics. In addition, it would streamline the
effectiveness of advanced development while simultaneously reducing
technological risk.
Analysis suggests that such a capability would also significantly
lower the Government's cost of acquisition while establishing the
Nation's first domestic, flexible, surge production capability for both
CBRN and noncommercial public health threats.
Finally, in my view, it is important that the job of executing this
concept be awarded through a competitive process. This will ensure the
best-qualified coalition of private sector partners is brought to bear
in helping the U.S. Government meet this important challenge.
Thank you for your support in highlighting these national security
and public health needs at this time. I would be pleased to answer any
questions you may have.
Senator Specter. Thank you, Dr. Gomez.
We now turn to Dr. Donald S. Burke, Dean of the Graduate
School of Public Health, and Director of the Center for Vaccine
Research, and Associate Vice Chancellor for Global Health at
the University of Pittsburgh. First occupant of the UPMC Jonas
Salk Chair in Global Health. Bachelor degree from Western
Reserve University and M.D. from Harvard Medical School.
Thank you for coming in today, Dr. Burke, and the floor is
yours.
STATEMENT OF DONALD S. BURKE, M.D., DEAN, GRADUATE
SCHOOL OF PUBLIC HEALTH; ASSOCIATE SENIOR
VICE CHANCELLOR FOR GLOBAL HEALTH;
DIRECTOR, CENTER FOR VACCINE RESEARCH,
UNIVERSITY OF PITTSBURGH, PITTSBURGH,
PENNSYLVANIA
Dr. Burke. Senator Specter and Congressman Altmire, thank
you for the opportunity to discuss the pressing needs of the
U.S. Government to find a better way to develop and produce
countermeasures for our country's health security.
And Senator Specter, on behalf of health scientists and
patients here in Pittsburgh and around the Nation, I also thank
you for your extraordinary efforts to provide increased funding
for biomedical research through the NIH.
Senator Specter. Thank you.
Dr. Burke. I currently serve as the UPMC Jonas Salk Chair
on Global Health and work at the Center for Vaccine Research.
In every research setting in which I have worked, I have
witnessed personally the difficulty of translation of advances
in basic scientific research into medical products.
The University of Pittsburgh is committed to joining with
UPMC and other partners in developing a flexible, multi-product
vaccine facility, and indeed, the university could bring
exceptional strengths to this partnership. As you know, the
University of Pittsburgh has a long and proud tradition of
vaccine research, dating back to Jonas Salk's extraordinary
achievements in development of the polio vaccine.
The Center for Vaccine Research that I direct is evidence
of the university's resurgent excellence in the field of
vaccines. The establishment of the center was made possible by
a $10 million contribution by UPMC, a visionary step toward
this process we are engaged in now. It is housed on two floors
of the new, state-of-the-art biomedical science tower. We have
31 full-time and affiliate doctoral-level research academics
and occupy 32,000 square feet of laboratory space. We also have
a regional biocontainment laboratory, a high containment
facility that was constructed with NIH support. This lab is
designed to permit research on vaccine development for avian
influenza and swine influenza, tuberculosis, dengue, tularemia,
and other highly infectious disease threats.
The Pittsburgh Regional Biocontainment Laboratory at our
university also serves as a core laboratory for the NIH-
supported Regional Centers of Excellence for Biodefense. This
laboratory houses the animal model core for the Mid-Atlantic
Regional Center of Excellence and Biosafety Level 3
laboratories in one facility and supports a full spectrum of
investigations from basic microbiological and immunological
manipulations to animal challenge studies.
Another strength here in Pittsburgh is our leadership in
developing and using computational modeling simulations to
optimize vaccine development and deployment strategies.
Recently the university was recognized as one of two NIH
National Centers of Excellence for modeling of infectious
diseases to serve the country.
We also serve as the home to the Vaccine Modeling
Initiative which is supported by the Bill and Melinda Gates
Foundation.
Pitt also has an excellent research and training
collaboration between the School of Medicine and the School of
Engineering, especially as related to the design and production
of medical devices and more recently the development of novel
biomanufacturing processes. These interdisciplinary medical-
engineering programs create a unique environment for academic
consultations and training of biotechnology personnel related
to vaccine design and manufacturing.
Thus, the University of Pittsburgh today demonstrates
excellence in many disciplines that are critical in vaccine
research. However, university professors who conduct early
preclinical and clinical stage research are not able to bring
the candidate product all the way to licensure on their own.
Product development, as opposed to basic discovery, requires a
different set of skills and expertise, and this product
development expertise resides primarily within large
biopharmaceutical companies whose business it is to bring drugs
and vaccine discoveries from the lab to the consumer market.
PREPARED STATEMENT
The formation of a public/private partnership such as UPMC
is proposing is the only real alternative that has the
possibility of success in fixing the critical problem that has
faced our Nation for many years. The University of Pittsburgh
with its outstanding research and training capacities stands
ready to support these public/private partnerships.
Thank you very much.
[The statement follows:]
Prepared Statement of Donald S. Burke
Mr. Chairman, Ranking Member, Senator Specter, and other esteemed
subcommittee members and staff, thank you for the opportunity to
discuss the pressing need for the U.S. Government to find a better way
to develop and produce biologic medical countermeasures for our
country's health security. This endeavor represents a key component in
the larger biodefense and public health framework and will certainly
help ensure a safer and more resilient America. And on behalf of health
scientists here in Pittsburgh and around the Nation, I also thank you
for your extraordinary efforts to provide increased funding for
biomedical research through the National Institutes of Health (NIH).
I am a physician, an infectious disease expert, an epidemiologist,
and vaccine researcher. I have worked on prevention and control of
epidemic infectious diseases for my entire career. Previously, I served
23 years on active duty in the U.S. Army, including service as the
Associate Director for Emerging Threats and Biotechnology at Walter
Reed Institute of Research (WRAIR). I currently serve as the Director
of the University of Pittsburgh's Center for Vaccine Research. In every
research setting, I have witnessed the difficulty in translation of
advances in basic scientific research into medical products to protect
people from serious health threats. I am here today to discuss the
exciting idea of a new facility to solve this pressing national
problem.
As you are aware, the first step in producing any effective drug to
counter a bioweapon or epidemic begins in the research lab. Initial
research and testing, if successful, then requires the crucial step of
applied research. Translational research is a term used to describe the
process by which a drug or vaccine candidate moves from early basic
research, through the various stages of development, and eventually
into a licensed product.
Both the NIH and Department of Defense (DOD) have made tremendous
investments in basic research, with significant progress achieved in
understanding disease threats, and identifying potential drugs and
vaccines to mitigate their impact. Although there have been remarkable
advances in the diagnosis and treatment of many medical conditions,
infectious diseases remain the leading cause of deaths worldwide. Few
discoveries in biomedical research are as important as those that
revolve around vaccines for infectious agents that pose risks to global
public health and global security.
The University of Pittsburgh is committed to joining with UPMC and
other partners in developing a flexible multi-product vaccine facility,
and indeed the University brings exceptional strengths to that
partnership. Out of more than 3,000 institutions nationwide, Pitt now
ranks fifth in NIH funding and fifth in the number of individual grants
received. Last year the University and its affiliates received more
than $450 million in NIH support.
The Center for Vaccine Research (CVR) that I direct is evidence of
the University's growing excellence in the field of vaccines. The
Center is housed on two floors of the new, state-of-the-art biomedical
science tower. The CVR consists of the research teams of 31 full-time
and affiliate doctoral level researchers, and occupies 32,000 square
feet of laboratory space. A key component is the Pittsburgh Regional
Biocontainment Lab, a high-containment facility that was constructed
with NIH support. This lab is designed to permit research on vaccine
development for avian influenza and swine influenza, tuberculosis,
dengue, tularemia, and other highly infectious disease threats.
The Pittsburgh Regional Biocontaiment Lab (RBL) also serves as a
core laboratory for the NIH-supported Regional Centers of Excellence
for Biodefense. The RBL houses the nonhuman primate core of Mid-
Atlantic RCE and Biosafety Level 3 (BSL3) research labs on a single
floor, thus supporting a full spectrum of investigations from basic
microbiological and immunological manipulations to animal challenge
studies.
We have also gained national recognition for our exceptional
collaborations between the School of Medicine and the School of
Engineering, especially as related to the design and production of
medical devices and more recently to the development of novel bio-
manufacturing processes. These internal collaboration have been further
enhanced by inclusion of engineering faculty from Carnegie Mellon
University. We have NIH training grants for the Medical Scientist
Training Program (MD/Ph.D.) and for biotechnology, the latter focused
on vaccine development with the CVR. These interdisciplinary medical-
engineering programs create a unique environment for academic
consultations and training of biotechnology personnel related to
vaccine design and manufacturing.
Those performing early clinical stage research such as is done in
the CVR are typically not able to bring a product candidate all the way
to licensure on their own. Discovery and development require different
expertise. Product development expertise resides primarily with large
biopharmaceutical companies whose business it is to bring drugs and
vaccines all the way from lab to the consumer market. However, because
most biodefense products are noncommercial by nature, there is no
market-based incentive for biopharmaceutical companies to pursue their
development and they have accordingly been reluctant to engage.
As products are developed and brought into early human clinical
testing by the NIH or DOD there currently is not a clear path to
licensure and procurement for the U.S. Government. Despite attempts by
the U.S. Government to attract experienced biopharmaceutical companies
to the process, few have entered.
For this reason, commercial partnering with the U.S. Government is
essential to bringing biologic medical countermeasure candidates to
full licensure. Without such a partnership, only the biopharmaceutical
industry retains the ability and know-how for scaling production levels
of biologics up to required levels. Larger biopharmaceutical companies
possessing this experience and expertise in advanced development must
be incentivized to engage if the U.S. Government wants to fulfill its
biodefense requirements.
It is clear that given the lack of commercial incentives for these
products, we cannot expect industry to enter alone. The formation of a
public-private partnership between industry and the USG is the only
alternative that has the real possibility of success in fixing a
challenges that has been at the DOD and HHS for many years, one that I
have personally encountered. I encourage the subcommittee and the
Government to continue to develop this concept, and ultimately compete
it implementation to ensure the best and most current ideas are
incorporated.
Senator Specter. Thank you very much, Dr. Burke.
Our final witness is Dr. Nigel Darby, Vice President,
Biotechnologies and Chief Technology Officer at GE Life
Sciences. These products are used in the manufacturing of more
than 90 percent of registered biopharmaceutical products. He
has been the Vice President for Chemistry Technology at Astra
Zeneca, 16 years in academic research in medicine and molecular
biology. He has a master's degree in natural science from the
University of Cambridge, UK, in 1981 and a Ph.D. in
biochemistry from the University of Kent in 1985.
Thank you for coming in today, Dr. Darby, and we look
forward to your testimony.
STATEMENT OF NIGEL DARBY, Ph.D., VICE PRESIDENT,
BIOTECHNOLOGIES, LIFE SCIENCES, GE
HEALTHCARE BIO-SCIENCES AB, UPPSALA, SWEDEN
Dr. Darby. Thank you. Senator Specter, Congressman Altmire,
firstly, thank you for the opportunity to testify today.
GE Healthcare provides much of the technology which is used
in the discovery and manufacturing of lifesaving
pharmaceuticals. Relevant to the testimony today, as you just
mentioned, GE provides the technology which is used in the
manufacturing of more than 90 percent of registered
biopharmaceuticals and many vaccines.
Now, we have heard today from our colleagues about the
importance of manufacturing biological countermeasures such as
vaccines in response to biological threats. I am here today to
outline the current state of the technologies used to
manufacture these countermeasures and the importance in
developing a partnership between the public and the private
sector to deploy these technologies to provide an effective
response to future biological threats.
Any manufacturing solution addressing biological threats
needs to address four key issues which relate to the
development of countermeasures: quality, safety, flexibility,
and speed. I will briefly address the primary considerations
about each of these areas.
Traditional manufacturing facilities for biopharmaceuticals
and vaccines are, indeed, expensive. There are often
constructed from ``hard-plumbed,'' permanent, stainless steel
technology and they are dedicated to a single product. They can
cost up to $1 billion to construct and take up to 5 years to
build and validate. Between manufacturing runs in these
facilities, the equipment must be extensively cleaned and
tested to ensure that there is no contamination, a time-
consuming and costly process reducing productivity but ensuring
the safety of the final end product.
So how do you go about achieving some more flexibility?
Clearly, a $1 billion biological countermeasure production
facility dedicated to a single biological threat is not a
viable response to a threat which is as yet not well-defined.
Recent advances in developments in biomanufacturing, however,
mean that we can design more flexible facilities that can
manufacture a number of medicines and still retain the quality
and safety profiles of traditional facilities and, in addition,
achieve this at a lower cost and with improved efficiency.
New developments are allowing us to replace the traditional
stainless steel manufacturing technology with disposable
plastic technologies. These allow us to rapidly reconfigure the
manufacturing process, using off-the-shelf, ready-to-use
components. After a manufacturing run, components are simply
discarded and a new set installed for the next manufacturing
cycle. Expensive cleaning and validation processes required in
traditional facilities are significantly reduced, improving the
facility productivity substantially, but retaining the overall
level of safety in manufacturing.
The use of this disposable technology improves
manufacturing safety. It reduces contamination risk and
increases the outputs of the facility, and that adds the
crucial element of flexibility. It also reduces the capital
expenditure and lowers the start-up costs.
If we turn to speed, in a pandemic or biological attack,
reaction time inevitably must be short. Preparedness,
therefore, should focus on solutions that deliver a
significantly more likely response. Disposable, ready-to-use
manufacturing technologies bring unprecedented speed to both
the development and manufacturing of biopharmaceuticals and
vaccines. In collaboration with the vaccine developer, we have
demonstrated that disposable technologies can reduce the time
it takes to get a flu vaccine into production by up to 60
percent.
In summary, then our four critical manufacturing
challenges--safety, quality, flexibility, and speed--can be
addressed by implementation of the new disposable manufacturing
technologies being developed by GE and our industry peers.
Virtually all manufacturing steps required can be carried out
with disposables.
While currently not capable of achieving the manufacturing
scales of the largest fixed stainless steel installations, the
technology is evolving rapidly enough to deliver significant
volumes of therapeutic agents and vaccines in a safe and timely
manner.
PREPARED STATEMENT
In conclusion, biopharmaceutical and vaccine manufacturing
technology is at an exciting and important point in its
development. What we now need at this point is a commitment
from Government to help bring these technologies to the
forefront at a time when we have the capacity to do so and when
the need has never been greater. We believe this presents a
unique opportunity for the public and the private sector to
come together and ensure that these critical technologies can
be in place to deal with the threats of biological agents which
we face potentially in the near future.
That concludes the testimony that I have. Once more, thank
you for allowing me to participate today, and I welcome any
questions that you have. Thank you.
[The statement follows:]
Prepared Statement of Nigel Darby
Chairman Specter, distinguished guests, thank you for your
attention to the important issue before us today. My name is Dr. Nigel
Darby. I am the Vice President for BioTechnologies of GE Healthcare's
Life Sciences division and have worked extensively in the
pharmaceutical and biotechnology industries, both as an executive and
in research and development. Today I manage a large product portfolio
of tools and technologies that support research, discovery and drug and
biopharmaceutical manufacturing.
Prior to entering industry, I spent 16 years in academic research
in medicine and molecular biology at the National Institute for Medical
Research in London, the M.R.C. Laboratory of Molecular Biology,
Cambridge and at the European Molecular Biology Laboratory in
Heidelberg, Germany. I have an M.A. in Natural Sciences from the
University of Cambridge and a Ph.D. in biochemistry from the University
of Kent in the UK.
GE Healthcare provides much of the technology used in the discovery
and manufacturing of pharmaceuticals. Most relevant today is our
expertise in manufacturing of biopharmaceuticals and vaccines.
Biopharmaceuticals are drugs such as insulin and monoclonal antibodies,
often based on proteins, and are among the most rapidly growing groups
of medicines today. More than 90 percent of registered
biopharmaceuticals and many vaccines rely on GE-developed technologies
or processes for their manufacture.
We have heard a lot today about the risk and the threats biological
weapons and pandemics pose to national security and the necessity to
develop and manufacture biological countermeasures. I am here to
outline the current state of the relevant manufacturing technology that
can be brought to bear on these threats, the key issues that need to be
addressed, and how that technology is evolving.
Any technological solution addressing biological threats, whether
natural outbreaks or terrorist actions, needs to address four key
issues in manufacturing biological countermeasures: quality and safety,
flexibility, and speed.
quality and safety
For reasons of patient safety, quality and consistency in
pharmaceutical manufacturing is critical and this is particularly the
case for biopharmaceuticals and vaccines. These are complex medicines,
usually produced in biological systems, such as cell culture, and they
require a series of purification steps to deliver a safe end product.
The quality and safety of the end product is assured by robust control
and validation of the manufacturing process. The aim is to deliver as
reproducible a process as possible and to exclude all possible sources
of contamination.
Biopharmaceutical and vaccine manufacturing requires established,
validated equipment and highly skilled, fully trained individuals to
perform the procedures. Production must meet standards as set out by
FDA approval guidelines and ISO standards, including complying with
cGMP biocontainment requirements for aseptic production, and biosafety
regulations. These standards must be maintained throughout all stages
of the development process to ensure that the product remains the same
and retains high quality. Development of systems and standard operating
procedures are vital to promote stability, reduce costs, and ensure
quality.
Traditional manufacturing facilities for biopharmaceuticals and
vaccines are expensive, partly, and rightly, because of the time and
effort spent controlling the facility's environment, the cleaning and
maintenance required to avoid contamination of the end product. These
are often `hard-plumbed' stainless steel facilities, dedicated to a
single product, and can cost up to $1 billion and take 5 years to build
and validate. Between manufacturing runs, the equipment must be
extensively cleaned and tested to ensure there is no contamination; a
time-consuming and expensive process.
While reasonable for mainstream commercial pharmaceuticals and
vaccines, where volumes are high and demand is predictable, a billion
dollar biological countermeasure production facility dedicated to a
single biological threat is not a viable response to an as-yet
nonspecific threat. Recent developments in bio-manufacturing mean we
can design flexible facilities that can manufacture a number of
medicines and vaccines and yet retain the quality and safety profiles
of traditional facilities.
flexibility
To achieve this flexibility, manufacturing must move beyond the
dedicated ``hard-plumbed'' stainless steel plants focused on a specific
product. Ideally, we should be able to rapidly manufacture different
products in a single facility, and do this without compromising product
safety.
New developments are allowing us to replace much of this stainless
steel technology with disposable plastic technologies. These allow us
to rapidly reconfigure the manufacturing process, using off-the-shelf
ready-to-use components, for example pre-sterilized where appropriate.
After a manufacturing run, components are simply disposed of, with a
new set brought in to manufacture the next batch, or even a different
medicine or vaccine.
The use of this disposable technology improves manufacturing
safety, reduces contamination risk and increases the throughput of the
facility, and adds that crucial element of flexibility. They also
reduce capital expenditure and lower start-up costs.
When compared with traditional stainless steel production
facilities, the ``ready-to-use'' system combines the advantages of
single-use technology--at its simplest, offering plug-and-play ease of
use--with cost-efficiency and additional safety aspects. In the event
of an emergency, a flexible and modular production approach would
facilitate rapid capacity deployment in a cost-effective and robust
manner.
Additional benefits of ready-to-use and single-use technology are
numerous. By eliminating many of the time-consuming steps from initial
set-up, cleaning, analysis and documentation--downtime can be turned
into uptime and production capacity within such a facility increased.
In cases where rapid facility change-over is demanded, single-use
products will be especially useful eliminating the risk for cross-
contamination and operator exposure, especially important when
manufacturing processes are based on the production of biological
pathogens.
Adaptability is a key advantage of ready-to-use systems. Many of
our customers are switching to systems with these components as a more
flexible option, allowing the user to quickly change the target
molecule, and providing some flexibility in batch volume.
In summary, single-use components offer many benefits beyond speed
and flexibility, including reduced risk of contamination, minimized
downtime for cleaning, sterilization and corresponding validation
procedures, reduced operation costs and minimal maintenance.
speed
In the event of an incident such as an outbreak of an influenza
pandemic or other pathogen, reaction time is inevitably short.
Therefore preparedness efforts should be directed at implementing
solutions that will facilitate a rapid response. Simplifying
development and accelerating manufacture of a biological countermeasure
are critical challenges, and GE Healthcare has allocated significant
investment to lead technological progress in this area.
To effectively protect a target population, a biological
countermeasure must be developed in such a way that it can be produced
and delivered in large volumes. For example, when faced with the threat
of seasonal and pandemic influenza, vaccine manufacturers face the
challenge of scaling up production to deliver large batches of product
in the shortest possible time. Currently, in the event of a pandemic,
existing global manufacturing capacity would provide sufficient
influenza vaccine for only around 4.6 percent of the world's
population.\1\
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\1\ Heuer A., Editor's Note. The Bridge. 2006 Fall: 36(3):3.
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Understanding both the biological and production hurdles, much of
our focus has been on developing more cost-effective, quicker, modular
ready-to-use manufacturing solutions, to both help traditional
manufacturers gain efficiencies, but also to meet the growing need for
flexibility in development, as governments search for solutions that
would enable production capabilities to be implemented at speed and low
cost.
Take the current influenza epidemic as an example: Traditionally
many vaccines such as flu vaccines, rely on an inactivated or weakened
attenuated pathogen that is produced in eggs and purified. With normal
seasonal lead-times, flu vaccine demands can be met, however in a
crisis situation, even if all the current manufacturing plants were to
concentrate on producing a ``pandemic'' vaccine, a serious capacity gap
would still exist.
Responsible health planners around the world are looking to
increase manufacturing speed and capacity and vaccine stockpiles to
counter the threat of a flu pandemic: the global demand for pandemic
influenza vaccine has been reported as possibly approaching 7 billion
doses, and according to the WHO with current world capacity, the
potential vaccine supply would fall several billion doses short of the
amount needed to provide protection to the global population.
Disposable and ready-to-use manufacturing technologies bring
unprecedented speed to both the development and manufacturing of
biopharmaceuticals and vaccines. As an example, working with a vaccine
developer as a partner, we have shown that it is possible, with
disposable technologies, to reduce the time it takes to get a flu
vaccine into production, after the isolation of the relevant viral
strain, by up to 60 percent.
Safety and quality, flexibility, and speed--all three of these
critical elements can be addressed by the advent of new disposable and
ready-to-use manufacturing technologies being developed by GE and our
industry peers. Virtually all manufacturing steps can be carried out
with disposables, from cell culture to purification steps such as
chromatography and filtration.
While currently not capable of achieving the manufacturing scales
of the largest fixed stainless steel installations, the technology is
evolving rapidly enough to be able to deliver significant volumes of
therapeutic agents and vaccines. This is aided as the flexibility of
disposable technology allows the economic and rapid set up of multiple
production lines in parallel.
In conclusion, biopharmaceutical and vaccine manufacturing
technology is at an exciting point of development, increasing speed and
flexibility in manufacturing, while assuring product safety and
quality. Interest in these developments is high, as pharmaceutical
companies, governments, and their national health and security
organizations recognize the new capabilities that this technology can
deliver in the challenging circumstances of an emerging infectious
disease or other biological threat.
Mr. Chairman, thank you for the leadership you've shown and for
calling attention to this important issue.
Senator Specter. Thank you very much, Dr. Darby.
We will now proceed with 5-minute rounds of questions from
the panel.
I begin with a statement made by Dr. Gomez commenting that
they started with manufacturing and then quickly moved to
advanced development, emphasizing the need not only for
manufacturing capabilities but also to keep abreast on
technological development. UPMC, the University of Pittsburgh
Medical Center, has the advantage of having the University of
Pittsburgh research at its side, and the University of
Pittsburgh has the advantage of having UPMC as well. There is a
little mutuality.
Dr. Burke had testified of his appreciation for the
increase in funding by the NIH, and just a comment about that.
When I was elected to the Senate in 1980, the first committee I
chose was Appropriations because of its power, candidly, in
bringing money back to the State. And the first subcommittee
was the Subcommittee on Labor, Health and Human Services, and
Education, which is the subcommittee with this hearing. In my
first year in the Senate, the NIH budget was $3.6 billion, but
when I became chairman, in collaboration with Senator Harkin,
now the chairman--he was then the ranking minority member--the
NIH budget was $12 billion, and we took the lead in increasing
it to $30 billion, some years as much as $3.5 billion added.
And during those years, there was a marked decrease in
fatalities or morbidity due to stroke and many other maladies.
I have taken a look here at the funding for the University
of Pittsburgh from NIH, which has been considerable. In 1998,
it was $169 million, and then with the increases, it went as
high as $386 million. A lot of money. And in the span from 1998
to 2007, it was--I want to be sure these figures are right. It
is $3,020,000,000, which has really been--well, how would you
characterize it, Dr. Burke? What has that done for the
University of Pittsburgh?
Why should I characterize it when I have a witness at hand?
Dr. Burke. I would characterize it as a lot of money, sir.
Senator Specter. Well, what has it enabled you to do?
Dr. Burke. During this time, there has been the explosion
in biotechnology is probably the most important change in----
Senator Specter. And it was not only the University of
Pittsburgh. It happened across the country.
Dr. Burke. That is correct. The entire country has had this
explosion of biotechnology that now allows us to move some of
these vaccine technologies into the advanced development that
would permit this kind of facility to be useful where
previously we relied on the traditional steel technologies, the
larger-scale technologies, and here I think we now have
flexible, fast-moving technologies. So it not only affects
these. It is across the board. There has been an incredible
impact on biomedicine.
Senator Specter. And in recent years, it has been cut back
as a result of budget constraints, no cost-of-living
adjustments, across-the-board cuts so that there was a decline
on NIH in real dollars of $5.2 billion. But then with the
stimulus package, the amendment, which I offered, got into the
bill for $10 billion extra. What has that done in terms of
reawakening a whole generation of research scientists?
Dr. Burke. As you point out, in the last 2 or 3 years, we
have had a flattening and an actual decline, and it has been
very difficult to sustain first-rate biomedical research, and
the additional funding that was provided in the last year, the
additional $10 billion, has allowed us now to take up some of
those projects that otherwise would not have been funded and
carried forward. So it has been an incredible stimulus. The
money is being well-spent and being spent quickly.
Senator Specter. I am going to move through the 5-minute
round, if it is okay with you, Congressman Altmire.
Mr. Altmire. Yes, sir.
Senator Specter. You will have equal time. But I am on a
line of questioning which I want to pursue further with Mr.
Romoff.
Comment on the relationship between the University of
Pittsburgh Medical Center and the University of Pittsburgh. You
are separate but how do you interact?
Mr. Romoff. We are two separate, independent organizations
but intimately joined at the hip. I think using the 21st
century biodefense project as an example, as Dr. Burke stated
before, we contributed $10 billion--$10 million--excuse me----
Senator Specter. You are getting your zeroes mixed up, Mr.
Romoff.
Mr. Romoff. These days it is too easy, I am sorry to say.
We contributed $10 million to develop, under Dr. Burke's
leadership, the Center for Vaccine Research, and Dr. Burke and
his colleagues bring the scientific underpinnings, both the
basic science and political science, to figuring out and
solving----
Senator Specter. So tell me why is it that UPMC undertakes
this kind of a project as opposed to having the University of
Pittsburgh do it?
Mr. Romoff. Because UPMC has the capacity to take science
and translate that science into products and then to, say, a
vaccine factory or to translate into patient care innovations.
The university does a good deal of the science and research. We
take the research and translate it into things that work for
patients, work for society in general.
Senator Specter. Well, that is the unique opportunity which
I wanted Dr. Gellin to hear in detail. May the record show he
saluted and raised his hand and acknowledged the information.
Well, that is what hearings are about. Dr. Gellin is going to
play a key role in what is going to be done here in his
position.
The joinder of the University of Pittsburgh and UPMC is
very unique. I look for a competitive bidding at the end of the
rainbow, and I am going to be pushing hard to land it for
Pittsburgh in the 7,000 jobs, which I commented about earlier,
and also the expertise to really contribute to the national
welfare. There are two edges of the sword. We are not going to
spend the money foolishly, but when you can produce this kind
of a product, you produce the jobs to an area which needs them,
there is a lot to be said for that. And the synergy between the
University of Pittsburgh and UPMC to get the job done I think
is really significant.
Mr. Romoff. Can I just add one point, Senator? In addition
to thanking you once again not only for--or $10 billion in NIH
funding which produced the science, but for your extraordinary
efforts on behalf of this project here.
But to focus in on the economic development aspect, this
project, if done, would bring 1,000 jobs directly and 6,000
jobs indirectly, but that is really just the beginning. As is
clear from testimony here, we are talking about creating an
industry and an industry without significant bounds, and that
is, if we were able to, through competitive bidding, land this
public/private partnership with your assistance here in
Pittsburgh, I have no doubt that we would then be able to
attract around that other private biotechnology companies to
come to Pittsburgh who would set up laboratories----
Senator Specter. Do you have any idea as to what the dollar
figure would be or the employment opportunity figure would be?
Mr. Romoff. It is all speculation, but I think----
Senator Specter. Go ahead. Speculate a little.
Mr. Romoff. Well, I think we are talking about multiplier
effects of at least 10 times what the initial investment is.
Senator Specter. So something like 70,000 jobs and----
Mr. Romoff. I think you can certainly imagine that because
everyone understands that these small biotechnology companies
do not have the resources, do not have a place to turn to
develop--do not have a flexible facility that is able to
produce small batches, not just large batches, of different
kinds of vaccines and other kinds of drugs that they need to go
to clinical trials and to develop things that become
commercially viable. A facility like this serves not only the
Government's needs, but it serves an enormous amount of
proprietary needs, and that is where you get the influx into
the region of this kind of money, energy, people, and jobs.
Senator Specter. Dr. Russell, you said in 1993 that the
task force recommended a Government-owned, contractor-operated
facility for manufacturing medical countermeasures, and that
kind of an organization would avoid the kind of the problem
that we have with Australian manufacturers for vaccines. They
look out for Australia not for the United States. But the DOD
determined that it was too expensive.
Now, I commented earlier about the kind of funding which
has been produced here, that in 2004, when I chaired the
subcommittee, we put up $6.4 billion and the President now has
additional discretion from further appropriations for $5.8
billion. And considering the severity of the problem, would you
continue to recommend, as you did in 1993, that it be a
Government-owned facility or at least a public/private
partnership where the Government could have a determinative
voice in manufacturing for the welfare of the United States in
light of the kind of threat we face now after 9/11, the kind of
money which the Congress has already been willing to put up?
Dr. Russell. At the time that decision was made by the DOD,
I disagreed with it. I was in support of the Government-owned,
contractor-operated. The technology at the time was more
expensive and the methodology required dedicated facilities. It
was not as efficient as we can do today with today's
technology. I would argue very strongly that the decision by
the DOD to move to a prime contractor method of operation
basically failed to produce the product effectively.
Senator Specter. So the old system failed, and you stand by
your approach for Government participation?
Dr. Russell. I think it definitely requires that we have a
facility that the Government has sufficient control over to
meet its needs when it is required and it has the flexibility
to develop new products as they come out of the research base
in the universities.
Senator Specter. Dr. Darby, I have run a little long on
time, but Congressman Altmire will have equal time.
GE has a patent on an operation on plastics which enables
to shift from one vaccine to another very promptly, contrasted
with having stainless steel equipment which takes an amount of
time to clean. May the record show the witness is upward
nodding in the affirmative. That is true, is it not?
Dr. Darby. Yes.
Senator Specter. I am asking you a leading question, but I
want to move forward with some speed here so we can conclude in
a reasonable time.
Senator Specter. But GE does have that patent. Right?
Dr. Darby. Yes. GE has technology which is surrounded by
patents.
Senator Specter. And that enables GE's participation to
move from one vaccine to another with disposable plastic as
opposed to a lengthy cleaning process.
Dr. Darby. Yes, sir.
Senator Specter. And you have been working with UPMC to
bring that technology to bear in the kind of an operation which
they have proposed.
Dr. Darby. Yes. All the studies that UPMC has been putting,
which have been mentioned, we have been involved in those
studies with the technology knowledge that we have today. So I
think we are reasonably satisfied that it can be made to work.
Senator Specter. Congressman Altmire, you are entitled to
equal time, and that is 14 minutes and 16 seconds.
Mr. Altmire. Thank you, Senator, and I do not plan to use
all that time, but I appreciate it.
Very quickly to begin with, Dr. Gomez, you spoke at length
about the study that you conducted, and as a management
consultant at the firm, you obviously have a financial
relationship with people that commissioned the studies. I am
making no implication, but just for the public record, what is
your relationship with UPMC as a consulting organization, and
do you feel that that impacted that study and the results in
any way?
Dr. Gomez. So PRTM was under contract to support that
study, so we certainly provided what we believed was the best
advice and analysis of part of that study.
Mr. Altmire. But do you believe that the outcome and the
conclusions that you testified about today and that the study
showed were in any way impacted by that relationship?
Dr. Gomez. No. I certainly believe that we provided the
best analysis of the industry based on certainly my experience,
having been in industry for a long time, as well as being
within the U.S. Government.
Mr. Altmire. Thank you.
Mr. Romoff, you spoke at length in your initial testimony
about the need for this type of center, a national vaccination
center, and in response to Senator Specter's question, about
the assets that UPMC has to the region with its relationship
with the University of Pittsburgh. And Dr. Burke spoke about
their specific interest in the vaccination center and the long
history going back to Dr. Salk that they have with that issue.
I want to give you the opportunity to close the loop on these
things because you talked about the benefit western
Pennsylvania has, you talked about the need that exists,
University of Pittsburgh's role in it. What are UPMC's assets
specific to the vaccination center, and why is western
Pennsylvania a good location, and why is UPMC a good
organization to do this?
Mr. Romoff. Well, thank you for the softball question.
UPMC, as you know so well, Congressman, is an $8 billion
organization with 50,000 employees and that does not include
the enormous assets at the University of Pittsburgh. We manage
20 hospitals, hundreds of clinics. We do a great deal of
things. We have laboratories and we do international
consultations with hospitals in Ireland and Italy. UPMC has
developed an intellectual managerial power fundamentally in the
health care sector and in the health research sector to get
things done, to take the brilliance of the scientists and the
consultants and convert it into things that happen and grow and
develop, treat patients, come up with new products, and then
they bring economic value. We have been very successful at
doing that, and we have been enormously comfortable with--here
in western Pennsylvania, with the workforce that is here,
with--that is here, and when it came to this project, it was
first and foremost needed in a way that I think was obvious for
everyone.
And secondly, western Pennsylvania just has such
extraordinary natural resources and such a good climate for
productivity and a good workforce that this became a natural
thing for UPMC to do here.
Mr. Altmire. Thank you.
Dr. Darby, I was very impressed by your talking about the
leadership that GE has in this issue and the expertise that you
have. My question is, with regard to this project in specific,
which is more important to GE? Is it the dollars, the Federal
investment of $600 million or more, or is it the commitment
that the Federal Government brings that this is an important
project? And I ask it in the context of the obvious question is
GE a company that historically has done very well. You have
leadership on this issue. You have obviously made a substantial
investment through the company. Why do you need public money?
Is it a Federal commitment that you need that this is
important? Is this something you can do yourself, or is this
something that you really believe the public needs to play a
role in financing?
Dr. Darby. Well, I think one of the major elements of this
is, of course, we bring the manufacturing technology, which is
at the heart of the puzzle. I think we need Federal backing
behind this to bring the different players together, GE,
organizations such as UPMC, to create the whole structure that
is required to make this actually happen. So we bring a part of
the puzzle, but there is no way that we could provide a
countermeasure solution simply by ourselves. We need to work in
partnership with the Government and other players to help to
deliver this to you.
Mr. Altmire. And you feel that the players at this table,
others in the country who are interested in these types of
programs, universities, private companies, that there is not an
ability to make this happen without a substantial Federal
investment?
Dr. Darby. I think the Federal investment puts a certain
level of imperative behind it. One cannot say it will never
happen without the Federal investment, but I think the Federal
investment and sponsorship serves as a rallying point to make
this happen. So it will happen far more quickly.
Mr. Altmire. Thank you.
Lastly, for Dr. Russell, you spoke about the industrial
development in the United States for these types of centers. I
wanted you to talk about the risk that exists if we do not do
this, if we do not move forward. What are we putting
ourselves--and I am not asking you a leading question. Perhaps
you would say there is no risk, but I would suspect you would
say that there is if that is the case. What is the cost of
doing nothing? What is the cost of inaction?
Dr. Russell. I think that the structure of cost of doing
nothing is going to be the products that come out of the
research community, potential products, will not be developed
and we will not have the vaccines and other pharmaceuticals
that are necessary to counter a bioterrorism attack or another
emerging infectious disease situation. I think the current
system has proven to be inadequate. It is slow, cumbersome. It
is inefficient.
And this concept that is being talked about today is a
means of really streamlining the advanced development and the
manufacturing and a means of providing the countermeasures that
could be available based on our current scientific knowledge if
we move ahead with this. If we do not, we are going to have a
much longer period of vulnerability.
Mr. Altmire. Thank you.
No further questions, Senator.
Senator Specter. Thank you very much, Congressman Altmire.
Dr. Gellin, why do you not step forward and have a chair at
the end? Just a question or two.
What do you think about the proposal? You said earlier you
did not have too much idea. I will not ask you a leading
question. I will just ask you what do you think of it?
Dr. Gellin. Well, I think I told you in a meeting--it was
not in front of the microphone when we were speaking before--
that I was impressed with this proposal. I am also impressed
that in a 45-minute period you can assemble this panel to tell
you about the details of their proposal.
You also heard in this period that there is a need to move
forward. Dr. Russell has often told me that when things are
more than 10 years away, they are going to stay 10 years away
unless we begin to move them. So I think that there is a
commitment clearly by the U.S. Government, as I mentioned
before, by both HHS and DOD to begin to explore a path forward
to fill some of the gaps that we recognize.
Senator Specter. And what do you think of the synergy that
you find here with the research capabilities of the University
of Pittsburgh and the implementation capabilities of UPMC?
Dr. Gellin. Well, in his opening remarks, the Congressman
spelled out the attributes that this project brings forward and
you were able to assemble the people to tell us all about that
and just to really scratch the surface of what I know at least
of some of the things that are going on here. So I think that
what they presented to you is clearly an interesting proposal
and is one that we are obviously aware of and need to move
forward to make a decision on how to fill these gaps.
Senator Specter. Especially with the joinder of GE and
their capability with plastic disposables to expedite the
production of vaccines?
Dr. Gellin. It is clear, as Dr. Darby commented, that this
requires a multitude of talents to be able to solve this
problem, and I think that this is clearly an opportunity to
show how different elements that come to a table in partnership
are needed to do something like that.
Senator Specter. I thank Senator Harkin, chairman of the
subcommittee, and Senator Inouye, chairman of the full
committee, and Ms. Ellen Murray, who has contributed mightily
and done so much work in arranging for this hearing. She has
the benefit of coming to her hometown. So thank you, Ms.
Murray, and thank you, John Myers, for the work you have done.
I think this has been a very important hearing to put a lot
of pieces together, and my colleagues in Washington on the
subcommittee and the full committee and the full Congress will
be reviewing it. Congressman Altmire will carry the words in
the House of Representatives because this is a matter of
urgency for the country. I, obviously, have been explicit
before in promoting this as a Senator from Pennsylvania, and I
am authorized to say that Senator Casey joins me in this
effort. But there is a very important national interest in
public health and very important interests in economic
development, which we have specified.
And as I said, we are going to be introducing authorizing
legislation, and I will be working with it on the
Appropriations Committee where I serve. We will be coordinating
with the DOD and HHS, which we have talked about. Secretary
Sebelius is well aware of the program. I had a chance to talk
to her about it a little more. We did have a quiet moment,
believe it or not, 2 weeks ago in Philadelphia with the first
of the public demonstrations. And I look forward to a chance to
talk to Secretary Gates. One small personal, not irrelevant
note. Secretary Gates and I went to the same grade school in
Wichita, Kansas.
CONCLUSION OF HEARING
Thank you all for coming, and that concludes our hearing.
[Whereupon, at 11:57 a.m., Wednesday, August 21, the
hearing was concluded, and the subcommittee was recessed, to
reconvene subject to the call of the Chair.]
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