Testimony

Statement by
Anthony S. Fauci, M.D.
Director National Institute of Allergy and Infectious Diseases, Department of Health and Human Services
on
BioShield: Countering the Bioterrorist Threat
before the
The Select Committee on Homeland Security United States House of Representatives

May 15, 2003

Mr. Chairman and Members of the Committee, I appreciate the opportunity to discuss the Administration's proposal, Project BioShield, with you today. The events of September 11, 2001, and the subsequent anthrax attacks, have changed forever how the biomedical research community responds to the emerging threat of terrorism. While the National Institutes of Health (NIH) and other Department of Health and Human Services (DHHS) agencies, including the Centers for Disease Control and Prevention (CDC) and the Food and Drug Administration (FDA), have been preparing to address the threat of bioterrorism for several years, we have been called to accelerate our efforts vastly since the attacks of 2001.

Overview

Today, we know that there is a real threat to our nation, and one of the most important ways that we can respond to this threat is through the development of medical countermeasures to address potential agents of terrorism. We are now in a "wartime" mode and must modify the way we do business, while protecting the elements of our system that have made us so successful.

For decades, the NIH has led the biomedical research effort to improve the Nation's public health. The NIH research enterprise, fortified by a rigorous system for ensuring that only the best science is supported by Federal dollars, has served our country extraordinarily well. Through the traditional funding mechanisms of grants, contracts, cooperative agreements, and other partnerships, as well as time-tested personnel practices, this system has resulted in numerous major scientific advances that have improved the health of people around the globe, such as the development of interventions for emerging and re-emerging infectious diseases, including HIV/AIDS and Ebola.

With the unprecedented budget increases provided by Congress for biodefense research, NIH has hit the ground running with a comprehensive research agenda to address bioterrorism. However, there is an important issue that must be addressed: we must expedite and greatly accelerate the research, development, purchase, and availability of effective medical countermeasures against biological, chemical, radiologic, and nuclear terrorism. There is no time to wait.

When all Americans must confront the realities of terrorism directed at the United States, it is imperative that the Federal government be prepared to protect its citizens from the scourge of terrorism. We are particularly challenged by the biological threats that are known to us or could be modified, as well as those that are unknown. To address these threats, we must build not only a strong biomedical research base, but we must create incentives for the companies upon whom we are reliant to produce the needed medical countermeasures to defend us.

NIH stands ready to push forward its biodefense research agenda to support the development of "proof of concept" for diagnostics, therapeutics, and vaccines to address agents of potential bioterror. However, without the expertise, resources, and proven capabilities of the pharmaceutical companies who develop these products and bring them to the market so efficiently and safely, we will not be able to meet the challenges set forth to us. Project BioShield would provide this needed incentive to industry, by giving it the necessary assurances that we will be reliable partners with them in meeting the challenge to develop the critical medical countermeasures to protect our citizens from acts of terror.

Project BioShield

Project BioShield would use the resources of NIH, FDA, and the DHHS Secretary to work together to accelerate the research, development, purchase and availability of effective medical countermeasures against chemical, biological, radiologic and nuclear terrorism. It takes a three-pronged approach. First, Project BioShield would increase authorities and flexibility for NIH, particularly the National Institute of Allergy and Infectious Diseases, to expedite research towards the development of critical medical countermeasures for biodefense, such as vaccines and therapeutics. Second, it would establish a secure funding source, via a mandatory authority, for the purchase of such countermeasures. And third, it would establish an FDA Emergency Use Authorization for critical countermeasures.

With regard to the first component of Project BioShield, the legislation would provide NIH with additional authorities to expedite the conduct of research and development in promising areas of medical countermeasures against potential agents of bioterrorism. This authority would provide NIH additional flexibility in awarding contracts, cooperative agreements, and grants for research and development of medical countermeasures including vaccines, drugs, biologics, and diagnostics. It also would streamline procurement authority, bolster authorities for acquisition and renovation of facilities, expedite personal services contracts and provide flexibility for certain personnel decisions to hire the necessary technical experts for biodefense research. Funding awards would remain subject to rigorous scientific peer review, but expedited peer review procedures could be used, when appropriate, without compromising scientific, technical, and programmatic standards. These new authorities would give NIH the tools it needs to expedite and push forward the pathway from basic research to effective biodefense countermeasures.

With regard to the second component of Project BioShield, the secure funding authority for procurement of countermeasures, it is worth noting that, historically, pharmaceutical research and development has focused on the development of products likely to attract significant commercial interest and a long-run market. We have found with experience, particularly in our numerous efforts to develop vaccines against some of the world's most devastating diseases, that uncertainties in the marketplace can create barriers to industry's willingness to invest resources and make long-term commitments to manufacture the needed products to prevent and treat disease. The recent shortages of vaccines for common and naturally occurring diseases are evidence of this problem. This lack of industry incentive is compounded with regard to the development of medical countermeasures to address bioterrorism, where the probability of a bioterrorist attack and the actual threats themselves remain unknown.

Our colleagues in the pharmaceutical industry - from small biotech firms to "Big Pharma," - particularly those in the vaccine industry, have stressed that, they are willing and eager to help in the development of biodefense countermeasures. However, these companies are businesses, not non-profit organizations, and they need a tangible incentive to get involved in the critical effort to ensure adequate defense against bioterrorism.

When it is evident that a given pharmaceutical product has a potential to make a profit, no incentives are needed to engage industry. However, with the development of a product for which there is no guarantee of a return, or for which the market is uncertain, industry prefers some assurance that there would ultimately be a return on its investment. Without such assurances, companies likely will pursue the development of other products.

When NIH meets with industry, we hear that, first, companies already may be involved in the early stages of development of biodefense countermeasures at their own initiative and are willing to assume a degree of risk of failure. However, they would like assurances that a market would exist for their product if indeed they are successful in its development. Also, many state quite frankly that they do not want to be vulnerable to the vicissitudes of the cyclical appropriations process.

In the other case, when NIH tries to engage reluctant companies to get involved in biodefense research, we try to "push" them into action using discretionary research dollars. However, in many cases, this does not seem to be enough to convince them to become engaged. With Project BioShield, we would be able to tell these companies that if they partner with us, meet certain milestones, and devise a licensable countermeasure, they will have our assurances that there will be money available to them for the purchase of that product. These are examples of the "pull" in the process: to the extent that the Federal government can define its requirements and assure up-front that funds will be available to purchase critical countermeasures, regardless of the level of appropriations for the year in question, then industry will have a real incentive to meet the biodefense research challenge. We feel that such assurances can only be given by a mandatory funding authority.

With regard to the third component of Project BioShield, the FDA Emergency Use Authorization, it is worth noting that the FDA approval process for drugs, devices, and biological products is the gold standard for the world. The FDA's policies and regulations help ensure that products that get to market are safe and effective. In addition to animal studies, sponsors of new drugs and vaccines typically conduct three phases of clinical trials in humans to demonstrate the safety and efficacy of a product. This process, however, can take years.

In preparing for the challenges we face today, we may not always have a desirable amount of time to address the threat presented by agents of bioterrorism. While the FDA has several mechanisms in place to get products to market faster, these alone are not sufficient in an emergency.

Project BioShield would permit the Federal government to make new and promising treatments that are still under development available quickly, if needed, for use in emergency situations where no effective approved or licensed products are available, potentially saving many lives. Specifically, Project BioShield would authorize the DHHS Secretary to grant an emergency authorization for the use of unapproved products in the event that the Secretary determines that there is no adequate and approved alternative available. This authorization would require the Secretary to determine that the benefits associated with using the countermeasure would outweigh the potential risks. Project BioShield would provide authority to the Secretary to apply conditions on the authorization, including limitations on distribution of the product, requirements to convey specific information to health care providers and patients, and requirements for recordkeeping, records access, and adverse event reporting. This authorization could be revoked by the Secretary and would be be limited in duration to the period of the emergency or not later than 1 year, unless renewed. It is important to note that the critical countermeasures would be tested for safety to the extent that the situation permits.

Conclusion

In summary, the need for medical countermeasures for biodefense is exigent and real, and we have a responsibility to the American people to make these products available now. The accelerated development of effective countermeasures against terrorism requires a new biomedical research paradigm, new ways to engage our industrial partners, and an ability to make promising products available for use during an emergency more quickly. Project BioShield would help us meet the challenges of terrorism effectively and expeditiously, improving our Nation's preparedness for and capability to respond to the threat of bioterrorism.

Thank you again for the opportunity to testify today about this important initiative to improve our homeland security. I would be pleased to answer your questions.

Last Revised: May 19, 2003