A bit lengthy, but worth reading. Here's the link to the PDG file I obtained this from...
avantimmune.com
Testimony of Una S. Ryan, Ph.D.
President and CEO, AVANT Immunotherapeutics, Inc.
Before the House Government Reform Committee
Project BioShield Act of 2003
Friday, April 4, 2003
Mr. Chairman and Members of the Committee, thank you for inviting me to testify before you on Project BioShield. I am the President and CEO of AVANT Immunotherapeutics, Inc., a
60-person biotechnology firm headquartered in Needham, Massachusetts, with a division located in Overland, Missouri. I serve on the Board of Directors of the Biotechnology Industry Organization (BIO) and I am Vice-Chair of the Massachusetts Biotech Council, although I appear before you this morning exclusively in my capacity as AVANT’s CEO. My comments are based, of course, on my experience as the CEO of a company that develops and produces vaccines that support the national biodefense effort, as a former research scientist at a large biotech company, Monsanto, and as an academic scientist for my entire professional career.
We sit this morning near ground zero of the war against bioterrorism. The last time I testified before members of this Committee, during a National Security Subcommittee hearing in October 2001, we had to convene at the Department of Health and Human Services, as House office buildings were closed due to possible anthrax contamination. Just across the Hill from where we sit is where the anthrax-laden letter addressed to Senator Daschle was opened; just over a mile away is the Brentwood facility where postal workers were lethally infected by the contents of that same letter. As awful as these events were, we all know that in some sense we
were lucky in that a larger, coordinated, camouflaged anthrax attack could have been far deadlier. As the federal government embarks on BioShield, a new and innovative program to fight bioterrorism and biological warfare, let me assure you that the biotechnology industry stands ready to contribute and work towards its success. Our eagerness to participate, however, cannot be unqualified. As the leader of a small company, I cannot embark on the development and supply of biodefense vaccines if doing so does not make business sense. I appear before you today to provide the perspective of a small biotechnology company on the concerns that my colleagues and I have about participating in the
biodefense effort and how I hope Project BioShield will address them.
BioShield—A Small Biotech’s View
Let me speak briefly of how the biodefense effort looks from my vantage point as the leader of a small biotech company. At AVANT, we develop a variety of therapies that harness the body’s immune system, including a vaccine to manage cholesterol levels (raising HDL “good” cholesterol). The area of AVANT’s work most relevant to the national biodefense effort is our development of vaccines that fight both bacterial and viral diseases. When it comes to vaccines, small companies like ours have much to offer. Vaccines have economics that scare away large pharmaceutical companies, who spend their research and development dollars seeking therapeutics that offer greater return. The vaccine world offers great opportunities for small companies like mine, for whom the promise of a smaller, yet distinct market offers
reasonable return for the application of our cutting-edge scientific expertise. Our vaccine business prior to September 11th focused on the market for travelers’ vaccines—protecting against cholera, typhoid, and dysentery—and on anti-viral vaccines to combat diarrhea in babies. However, we have worked with the Department of Defense, in particular the Army, in the biodefense effort well before the fall of 2001. One result of that work was that in October 2001, AVANT licensed its recombinant protective antigen for anthrax to
DynPort Vaccine Company (DVC) a Defense Department contractor developing a secondgeneration anthrax vaccine. This protective antigen is the crucial ingredient of an anthrax
vaccine, the protein that prompts the body to develop immunity to the disease so that if the person is infected, it already has protective antibodies in its arsenal. Although we are proud of this initial contribution to the biodefense effort, we are now playing a much more significant role. Our most advanced technology offers the prospect of biodefense vaccines that are far more effective, safer, less expensive, and faster acting than current generations of vaccines. For example, the current inventory anthrax vaccine provided to
U.S. troops is administered through multiple injections, which are often painful because of the side effects of the vaccine. Once the series of injections is begun, immunity develops gradually over several months. Compare this to the vaccine that we at AVANT, using our live attenuated vaccine vector technology, have successfully developed to fight cholera. This vaccine, called CholeraGarde, is administered in a single oral dose. It is safe and easily tolerated by the recipient. Immunity develops very quickly, in as little as 7 days. Manufacture of this vaccine is easy and inexpensive compared to current generation vaccines. While this particular vaccine fights cholera, our vector technology enables us to develop quickly a biodefense vaccine that is similarly effective, safe,and convenient. Our technology lets us adapt our vaccines to fight a wide range of bioterror agents. To my great pride, we signed a contract in January that allows us to apply our most advanced vaccine know-how to the biodefense effort. Under our contract with DVC, the prime contractor to the Defense Department’s Joint Vaccine Acquisition Program, we have begun development of a single-dose oral vaccine that will protect our troops against both anthrax and plague. As designed, the vaccine will have the same features of rapid protection and minimal side effects that characterize our commercial travelers’ vaccines. Furthermore, using our proprietary VitriLife drying process, our vaccines will be storable in powdered form at room temperature, eliminating the cost and burden of refrigerated storage. Under the current plan, we expect to complete preclinical development of this vaccine by the end of calendar year 2004. To my knowledge, ours is the most advanced technology vaccine currently under development
anywhere in the government’s biodefense program, civilian or military.
Making BioShield Work—Size, Consistency, Transparency
How then, does this experience shape my view of BioShield? Let me discuss two majorissues related to how BioShield will take shape that will affect its ability to attract biotechnology companies to participate. My greatest concern revolves around whether the BioShield initiative and corresponding efforts at the Defense Department represent a long-term commitment on the part of the government of sufficient size to make the venture worthwhile for our companies. An assumption underpinning BioShield is that the federal government, not the private sector, must create a
sufficient market to pull biotech companies into developing and producing biodefense countermeasures. For the government to create an adequate market, I believe it must ensure that
BioShield will possess three key attributes pertaining to the size, scope, and durability of the program.
First, the market created by BioShield must be of sufficient size to convince the industry that we have a partner who understands the cost, complexity, and risk of developing therapeutics and bringing them to market. The development of a successful biomedical product costs in a range that begins in the tens of millions of dollars and extends into the hundreds of millions. Research and development activities are expensive, protracted clinical trials even more so. Initial indications are that the application of the animal testing rule by FDA, the process by which efficacy of biodefense vaccines will be tested, will entail significant Phase IV postlicensing clinical trials to ensure efficacy and safety. This all adds up to a sizable bill for each and every countermeasure that is developed, licensed, and employed. Biotech companies will want to see a federal program of sufficient size to convince them that our effort can be funded throughout the life cycle, and that the risk we endure and success we achieve will be fairly
compensated.
The second necessary feature for a sufficient federal biodefense market is the long-term commitment of funding. As I mentioned, the development of biomedical countermeasures takes time. Five to ten years or more is not uncommon. Although we are making quick progress on anthrax, plague, and some other well-known agents, the broader list of agents includes many which are less well known and more technically challenging, such as viral agents and hemorrhagic fevers, like Ebola. I believe the government understands that meeting the biological weapons threat is of similar importance to the nuclear threat in many respects. The Strategic Defense Initiative, which celebrated its twentieth anniversary last month, launched a missile defense program whose funding has not dropped below $3 billion dollars annually over two decades. That kind of long-range commitment will convince companies that the government is a serious partner in this undertaking.
The third element of a reliable and consistent biodefense market is that it be run by a coordinated and transparent policy process, and that the policy itself be consistent over time. There will be two agencies playing the lead on BioShield, NIH for discovery and the Department of Homeland Security for procurement. For its part, the Defense Department will play both of these roles with respect to countermeasures for our troops. Careful coordination among the agencies will be crucial for developing clear and consistent policy. The industry understands that acquisition priorities must vary in response to evolving threats and advances in technology. To the extent attainable, however, acquisition priorities that remain consistent from year to year will boost our confidence that we have a reliable goverment partner. We look forward to working closely with the coordinating agencies to ensure that both the government and industry understand the goals, requirements, and preferences of its partners. In general, the greater the
transparency and consistency in BioShield policy, the more readily it will attract biotech industry partners.
Department of Homeland Security Plays a Crucial Role
I would like to make one last comment regarding the market-creation challenge faced by BioShield. In some ways, the crucial role of the BioShield acquisition function will be played by the Department of Homeland Security. The NIH, with its responsibility for funding and promoting research into biodefense technologies, will be doing more of what it has long done so well. The biomedical procurement authority in this scheme will be the DHS, a role that none of its component bureaus has ever played. This is a daunting task for a well-established organization, not to mention a brand new department. We will be watching carefully to see that
DHS is given the authority and funding it will need to fill this crucial role. If DHS is suitably empowered, the industry will sit up and take notice. By enumerating these challenges, I am not trying to sound pessimistic. I believe that the
goal of creating a market for biodefense of sufficient size and reliability to attract and maintain the participation of small biotech firms is eminently achievable. If this is achieved, the government will find us eager and committed partners. But we will be watching continuously to discern whether our partner is truly in it for the long haul. To meet these challenges, we are eager to work with the BioShield authorities as they shape the program over the months and
years ahead to ensure that it meets the requirements of the federal government and industry, the surest route to a successful partnership. Although I remain optimistic about the opportunity for success, I would like to mention a
personal experience that indicates how the partnership can founder. At AVANT we have taken a considerable risk by devoting a sizable chunk of my young company’s resources to our oral anthrax and plague vaccine project. We have found the Defense Department’s Joint Vaccine Acquisition Program to be an excellent partner that has extensive understanding of the countermeasures development process, from start to finish. But already, our willing JVAP partner has found its FY 2004 budget slashed from the level it received in fiscal year 2003. Already I am up at night worrying whether even if we succeed in the first phase of our vaccine development, whether funding will be available to finish the preclinical segment, let alone enter into clinical trials. Seeing JVAP have its funding slashed makes no sense to me given the high
priority placed on biodefense by both Congress and the Administration, as evidenced by this hearing today. I hope that this instance does not become an example of how, despite the best of intentions, failure of the many agencies involved to keep their coordinated eye on the biodefense ball could undermine effective programs and partnerships.
The Need for Liability Protections
After an adequate and reliable market, my second great concern revolves around indemnification. As in all business decisions, I can lead my company to accept risk only in
return for a commensurate reward. I cannot envision a situation in which I could risk my stockholders’ investments and my employees’ livelihoods by venturing unprotected into the biodefense realm where large portions of the population will be inoculated in a program of administration over which we have little control. Our advanced technology vaccines have
proved both safer and more effective than previous generation vaccines for the same diseases. Moreover, the FDA regulatory system sets the world standard for ensuring that only the safest and most effective therapeutics ever reach the
America n public. To then be vulnerable to lawsuits that could put us out of business just by their existence rather than their merit is a position in which I cannot place my company. There are many examples of product liability
protection extended to companies that supply much-needed vaccines, most recently the protection offered by the Homeland Security Act to manufacturers of the smallpox vaccine. I believe I speak for most if not all of my colleagues in the industry when I say that unless we are
protected from this risk to a fair and reasonable degree, we will not be able to participate in this national effort.
Mr. Chairman, My colleagues and I in the biotechnology industry have devoted our careers to creating products that serve the health and well being of the American public. I can
think of no better way to continue that work than by playing a role in the BioShield program we discuss here today. BioShield is a comprehensive effort on the part of the Congress and the Administration to tackle the urgent and complex task of protecting the American people from
biological threats. As a participant in this effort, I thank you and the entire Committee for your leadership. I hope that by laying out my concerns and requirements for a program in which my company and others like it will be able to participate that I have supported your efforts to devise
an effective program. Thank you very much. |
