News & Events
FDA Strategy for Medicines and Vaccines of Importance to National Security
Remarks as Delivered of Margaret A. Hamburg, M.D.
Commissioner of Food and Drugs
Conference on Preserving National Security: The Growing Role of the Life Sciences
Sponsored by The Center for Biosecurity of UMPC
Thursday, March 3, 2011
Good afternoon—and thank you for inviting me to this important, and very interesting, meeting. The focus is very apt: the life sciences can and must play a critical role in promoting and protecting the security of our nation and we have reached a moment of great opportunity and tremendous promise. If we find a way to fully harness the potential of emerging science and technology, we will be in a position to take major leaps forward in national security that not only benefit the health and well being of the American people—but also the future of our nation.
Of course, the question of how we harness that potential is not an easy one—and it is certainly not one that any single person, agency, or department can answer alone. And that is why meetings like this are so important. You have certainly had a day full of interesting and informed speakers, and hopefully some great discussions have ensued. And I suspect that the hallway conversations have been valuable as well. So I encourage you to keep talking—we have a lot to consider and much progress to make.
I am sorry that I could not be here to participate in some of the earlier sessions, but I am delighted to be here now…and pleased—and proud—to discuss the growing role my agency, the FDA, has played—and will continue to play—in applying cutting-edge science to the mission of protecting the American people.
Last August, we rolled out an initiative designed to strengthen and refocus FDA’s efforts to increase access to and availability of safe, effective medical countermeasures against chemical, biological, radiological and nuclear threats and naturally occurring emerging infectious disease threats. We developed the plan as part of a comprehensive, yearlong review of the HHS Public Health Emergency Medical Countermeasure Enterprise, ordered by Secretary Sebelius to assess the readiness of our nation to reduce the impact of a future public health emergency.
The review laid out a vision for a flexible, nimble system to produce medical countermeasures rapidly in the face of any attack or threat, known or unknown. And it identified our agency as fundamental to the success of the MCM Enterprise. Because FDA evaluation of product safety and efficacy so significantly impacts the course of product development, our agency is a linchpin in the effort to develop countermeasures against deliberate and naturally occurring biological threats.
One of the main components of our plan includes devoting considerable time and resources to advancing regulatory science—the knowledge and tools we need to more efficiently develop medical countermeasures and to facilitate the evaluation of product safety, efficacy, quality and performance.
Biological weapons have the potential to overwhelm public health and medical capabilities in this country—and lead to social, political, and economic disruption, on top of the enormous toll on life and health. And, of course, in our increasingly interconnected global community, we are not the only country affected.
In short, deliberate and naturally occurring biological threats threaten the security and well being of our nation. And we need medical countermeasures to mitigate these threats.
Medical countermeasures contribute to the deterrence of biological weapons development and use, and, should deterrence fail, help minimize the consequences of an attack. They also help minimize the health and social impact of natural and emerging threats by fostering resilience and recovery—and can even improve our ability to pursue US strategic objectives abroad…for example by supporting humanitarian relief and medical diplomacy efforts and facilitating development among partner nations and regions.
While significant progress has been made in the medical countermeasure area, our nation remains vulnerable. And we all recognize that our nation lacks many of the medical countermeasures needed to combat known threats—and we lack the capacity to rapidly develop new countermeasures in the face of new threats. This is unacceptable.
But we are now poised to make some important and exciting changes. The administration has made it clear that developing medical countermeasure capabilities to rapidly and effectively respond to public health threats—which will continue to evolve in unpredictable ways—is not just a priority, but a mandate.
Which brings me back to the role of the FDA. Our plan has been designed to address, in three major ways, some of the key challenges we face as an agency—and as a nation—in the development and availability of medical countermeasures.
First, FDA will support enhanced review and novel manufacturing approaches for the highest priority medical countermeasures. We will work interactively with developers and government partners from very early in the development process to define viable regulatory pathways, speeding progress toward product approval by helping to anticipate and resolve bottlenecks, and to identify and address scientific problems that may emerge.
Second, we will work with HHS and other government partners to conduct an examination of the legal framework, as well as regulatory and policy approaches, toward medical countermeasure development and availability, to assess the adequacy, or improvements needed, to properly support preparedness and response.
And third, as I mentioned earlier, we will work to advance regulatory science and improve countermeasure development and evaluation pathways by strengthening our own scientific capacity and building collaborations with government, industry, and academia. There are many challenges in the development of medical countermeasures, but there is no doubt that scientific opportunities are outstripping our ability to translate new discoveries and opportunities into new products. I believe that the advancement and more effective application of regulatory science will help us bridge that gap…by developing for example: new ways to evaluate product efficacy; more flexible, agile approaches to product development and manufacturing; new methods to improve product stability; and new statistical approaches to assessing efficacy with limited data. This effort will allow FDA to identify and help solve the scientific challenges that hinder medical countermeasure development and, without solutions, will continue to result in long delays in getting the products we need.
Let me give you an example in which regulatory science at FDA was successfully leveraged to encourage product development and speed product release.
Last summer, a meningococcal A conjugate vaccine developed through the Meningitis Vaccine Project was prequalified by the World Health Organization to ensure it met international standards of quality, safety, and efficacy. Since the vaccine—which was manufactured by the Serum Institute of India—was introduced, vaccination campaigns have been held in Burkina Faso, Mali, and Niger…and nearly 20 million people have been vaccinated in just six months. According to the WHO, the vaccine is expected to "eliminate the primary cause of epidemic meningitis in Africa if introduced in all 25 countries of what has become known as the meningitis belt" and save as many as 150,000 lives by 2015.
Here is the back-story. FDA scientists, specifically those in our Center for Biologics Evaluation and Research, were instrumental in facilitating the development of this vaccine. They developed a method called conjugation technology that can increase the immunogenicity and duration of protection afforded by the vaccine. We then established a Cooperative Research and Development Agreement with the Serum Institute of India, and CBER scientists transferred the technology to the Indian scientists and provided them with technical support as they scaled up to produce the vaccine for millions of people.
We have also used regulatory science approaches to avoid unneeded clinical trials and determine drug dosing in infants and children. For example, pediatric dosing recommendations for use of Peramivir IV under Emergency Use Authorization to treat 2009 H1N1 influenza were derived from a model-based analysis of data available from healthy adults and acute uncomplicated influenza infected patients. Also, FDA’s work on dosing the antiviral Tamiflu in children under one was a model adopted by countries around the world.
This is exciting progress for the application of science to the countermeasures enterprise—and a glimpse of immense promise. But, of course, it is only a start.
To continue to advance regulatory science in truly meaningful—and sustainable—ways, we must not only strengthen our internal FDA scientific capacity, but also reach out, far and wide, to build strategic partnerships with industry, academia, the non-profit world, and other government stakeholders.
We must foster an environment that allows all of us to take to advantage of the best available science and pool our products, resources, and, most importantly, our brainpower—in pursuit of the best possible solutions.
FDA has already had some significant success through a Memorandum of Understanding with the Defense Advanced Research Projects Agency, or DARPA, at the Department of Defense. The MOU allows us to work with DOD to facilitate innovation of medical products, including new technologies targeted for medical products and medical countermeasures. We are looking to collaborate on several projects, for example on Modular Immune In vitro Constructs technology, which models an "artificial human immune system" and allows for more human-like "pre-testing" of vaccine formulations—since animal studies are not always a good predictor of a vaccine’s safety and efficacy in a human. Success on this front would have tremendous implications for MCM research and development.
The bottom line is we must come together in new, unprecedented, even unexpected, ways. As our world transforms—and as it becomes increasingly globalized—we must build the interdisciplinary, multi-sectoral community we need to truly realize the potential of science for the future of national security.
After all, we share a common goal: to take all possible action today to ensure the safety and security of our nation. And we must incorporate the best, most modern science available as we do so.
At FDA, we are eager to embrace the unprecedented opportunities before us and deliver the promise of science on behalf of our national security. Ultimately, I believe that investments in regulatory science can truly transform the medical countermeasure enterprise and enable us to achieve a vision that can have major implications for our country.
We are all engaged in efforts that are critical to promoting and protecting the health of the American people. And we are all here today because we are committed to doing more, and doing better. And we must. The future of public health in America depends on it. Together we can, and will, build a safer, healthier, more prepared America.