Jesse Goodman M.D., M.P.H.
Director Center for Biologics, Evaluation and Research
Food and Drug Administration
U.S. Department of Health and Human Services
Regulation of Biological Products
Committee on Appropriations
Subcommittee on Agriculture, Rural Development, Food and Drug Administration
U.S. House of Representatives
Wednesday April 2, 2008
Good morning, Chairwoman DeLauro, Representative Kingston, and members of the Subcommittee. I am Jesse L. Goodman, M.D., M.P.H., Director of the Center for Biologics Evaluation and Research (CBER) at FDA. I am also a practicing infectious diseases physician. Joining me for today’s hearing is Michael A. Chappell, Deputy Associate Commissioner for Field Operations in FDA’s Office of Regulatory Affairs (ORA).
CBER is responsible for the regulation of most biological products. These products include vaccines, blood and blood products, and cellular, tissue and gene therapies. These products touch people’s lives on a daily basis with over 200 million vaccinations, 29 million transfusions of blood and blood components, and 1.6 million musculoskeletal tissue transplants annually. Safety expectations are extremely high, and we approach those expectations intensely and innovatively.
Biological products play a crucial role in the nation’s public health and preparedness, where they are critical in addressing emerging infectious disease threats such as pandemic influenza and terrorism. In these efforts, we seek to stay ahead of the curve and work closely and in an integrated manner with colleagues throughout the government, especially with the Centers for Disease Control and Prevention (CDC), National Institutes of Health (NIH), and the Department of Health and Human Services (HHS). As we advance our critical public health portfolio and work with cutting edge biotech innovations, we strive to stay scientifically up-to-date and work closely with product developers and the public. I appreciate this opportunity to be here today to thank you for your continued support, to discuss our ongoing efforts to help make needed products available, and to assure they meet the highest expectations of safety and effectiveness.
Funding for the FDA Biologics Program
The FY 2008 Omnibus Appropriations Act contains increases for the Biologics Program of $2.2 million for safety activities, $4.0 million for pandemic influenza, $1.1 million for the Critical Path Initiative, and funding to meet Biologic Program payroll obligations. These amounts are all subject to the FY 2008 across-the-board rescission.
The President’s budget for FY 2009 contains a total program level of $245 million for the Biologics Program. This includes a $3 million increase in budget authority and an increase of $6.6 million in user fees to support activities under the Prescription Drug User Fees Act (PDUFA) and the Medical Devices User Fee and Modernization Act (MDUFMA). The PDUFA fee increase supports expanded post-market safety activities, increases to reflect PDUFA workload, an adjustment for rent costs, and increases to support salary and benefit costs. The MDUFMA increase also includes increases for salary and benefit costs. These increases will help FDA meet PDUFA and MDUFMA performance goals. The FY 2009 increase in budget authority also provides resources to help support pay increases and enhancements to the human tissue and blood safety programs that will help support collaborative activities for early detection and response to emerging safety threats.
We greatly appreciate your interest in and support for our preparedness efforts. The accomplishments flowing from these investments are already tangible. We accelerated the preparation for a future influenza pandemic, including global and U.S. development and evaluation of new vaccines and the enhanced infrastructure to support rapid vaccine testing, release, and deployment.
I am pleased to note that in April 2007, FDA licensed the first vaccine to immunize individuals against H5N1 avian influenza. We are proactively involved in facilitating the review and development of additional novel products including recombinant and cell based technologies and vaccines that employ adjuvants to stimulate an immune response. We also put forth an accelerated approval pathway for annual flu vaccines based on a biomarker, and we worked expeditiously to evaluate new vaccines and enhance manufacturing quality. As a result, since 2004, we doubled the number of U.S. licensed manufacturers of flu vaccine from three to six and expanded vaccine production to a record of 132 million doses this year. We also recently extended the age indication of live attenuated influenza vaccine to include children two and older.
These successes provide the diversity of current and future vaccine supply and the manufacturing capacity to meet national public health and immunization goals and to save thousands of lives. The improved infrastructure for annual immunization helps make us far better prepared for future pandemics and other emerging infectious disease threats. Additional scientific and support staff, hired in multiple disciplines with pandemic funds, are now on board. The additional staff is available to review proposed vaccine studies, licensing, and emergency use applications, to analyze product manufacturing and quality, and to establish new approaches to monitoring product safety. These professionals are working with our public health partners and manufacturers to develop globally coordinated and expedited approaches to vaccine production, to develop new molecular tools to evaluate these vaccines, and to initiate collaborative research projects. These projects include developing better, faster assays for vaccine potency and safety and new approaches and systems to use healthcare data to monitor vaccine safety. We also enhanced our critical infrastructure and ability to help sustain safe supplies of blood and tissue products during a pandemic or other emergency.
Emerging science in areas such as bioinformatics, genetics, systems biology, and nanotechnology, as well as the globalization of infectious disease risks, knowledge, and industry make CBER’s mission increasingly critical and complex. These factors also present tremendous opportunities to help lead and shape the future of medicine, public health, and product development.
Other Preparedness Activities
Enhancing the nation’s preparedness for new or emerging threats is one of our highest priorities. This priority prevails whether the threat is pandemic preparedness, protecting the safety of our blood supply from emerging threats like West Nile Virus, or facilitating the development of vaccines needed to face other natural or deliberate threats such as TB, malaria, resistant bacterial infections, and smallpox or anthrax.
To address these threats, CBER is working proactively within HHS and with the World Health Organization (WHO), other federal agencies, industry, nongovernmental organizations, and regulatory authorities from other nations. We fostered information sharing and rapid development and evaluation of pandemic and other vaccines that will be effective, safe, and high quality for U.S. and global use. We initiated a collaborative effort with regulators from around the world, including regulators from Asia, to establish standards for pandemic vaccines and develop guidances for industry and regulatory agencies, including provisions for accelerated approval, to speed and assist vaccine development. We are also strengthening our emergency response infrastructure, including information technology and communication systems, and our laboratory testing, standards and methods, and quality systems. These steps will provide the tools and capacity that FDA and industry need to help assure vaccine and other critical product availability and quality, should an influenza pandemic or other outbreak occur.
It is essential to do all we can, even when markets for these products are uncertain, to help assure that products needed to face such threats are available, effective, and safe. Therefore, while working closely with many partners to achieve our preparedness goals, our most critical and unique responsibility is to do all that is possible to provide an objective, independent, expert scientific assessment of the safety and efficacy of these and other biologic products.
CBER emphasizes interdisciplinary, proactive approaches to safety throughout our products’ lifetimes. We are using modern methods to prevent, detect, promptly investigate, communicate and, where needed, take early action on potential safety signals and other problems. These activities begin prior to approval and continue afterwards. CBER’s multidisciplinary review teams include clinicians, statisticians, laboratory scientists, and product safety, quality, and manufacturing experts who help identify, evaluate, and, where possible, resolve potential concerns before product approval. They also work together to design and analyze results of post-approval monitoring. Recent innovations include implementing a new regulatory framework for enhanced oversight of tissue safety and creating formal interdisciplinary product safety teams for tissue, blood, and vaccines. These safety teams meet both regularly and during emergencies, with greater frequency if needed. Like our review teams, they bring together diverse disciplines, including product scientists, clinicians, epidemiologists and safety experts, and manufacturing and communications experts to share, analyze, communicate, and act on all available safety information. They also help develop and guide needed strategic and scientific approaches to enhance safety. Our safety teams collaborate closely with other FDA components and other agencies, particularly CDC. In addition, we work with CDC and its Vaccine Safety Datalink, the Centers for Medicare & Medicaid Services, the Department of Veterans Affairs, and academic colleagues, to develop and extend the use of large healthcare databases to detect and investigate potential product safety issues. For example, we responded to recent reports of Guillain-Barré Syndrome cases following meningococcal vaccination and monitored adverse events following rotavirus vaccination. In these and other instances, healthcare data and other new methods helped us to more rapidly and accurately detect, understand, and communicate potential risks and uncertainties. The public has appreciated early recognition and transparency about potential safety issues and corresponding prompt actions such as changes to product labeling.
We have also used quantitative risk assessment and computer modeling to evaluate complex risks, inform FDA, manufacturers, patients, and health care providers about such risks, and better evaluate potential risk mitigation options. An example is our computer modeled risk assessment, and subsequent risk communications, concerning the potential, but unproven, risk to plasma-derived products from the agent of variant Creutzfeld-Jacob disease, the human counterpart of “Mad Cow” disease. Our team and life cycle approaches to product safety and our use of better bioinformatics and scientific approaches to detecting and mitigating risks are consistent with the Institute of Medicine recommendations concerning drug safety and are being continually strengthened.
Innovation, Critical Path, and Scientific Management
I noted how we work collaboratively to facilitate rapid product development, review, and availability, and to enhance the quality of products needed for our nation’s public health preparedness. We also use these approaches to promote efficient science-based development and evaluation of other needed medical products. With your support and support from MDUFMA and PDUFA, we met or exceeded our goals for timely review of product applications and implemented innovations in review quality and efficiency, including increased use of electronic systems to support product evaluation and safety. Many innovative products, for example stem cells and gene therapies, present great promise to help cure or treat serious diseases. Yet, there is great uncertainty about their development, evaluation, and safety. In these areas, we worked collaboratively to help establish new and better development pathways and tools to evaluate product effectiveness and safety. CBER recently issued draft guidance to industry providing new approaches to licensing cord blood stem cells. We held workshops with NIH and product innovators to help define how best to safely advance products such as gene therapies, cancer vaccines, and stem cells for treating heart and nervous system disorders. We set up FDA’s first cross-Center review team and held workshops to provide expert scientific advice, review, and interactions with innovators developing engineered tissue products. These efforts, which are part of our Critical Path initiative, are helping define better, faster approaches to bringing needed innovations to patients, while assuring safety and quality.
To meet public health challenges and to modernize product evaluation for safety, quality, and effectiveness, CBER also conducts highly collaborative mission-related research. We implemented a new Managed Research Program to set scientific priorities and to seek and incorporate external input through our Advisory Committees.
Examples of recent or ongoing scientific activities and accomplishments include:
- innovations in the use of Centers for Medicare & Medicaid Services and the Department of Veterans Affairs databases to assess vaccine safety
- collaborative studies with the National Toxicology Program to reduce risks from retroviral gene therapies
- new methods (developed with NIH support) to test the safety of cell culture derived vaccines and other products, which resulted in new draft FDA guidance, with advice for industry and paths forward for cell culture-based flu vaccines
- numerous standards, such as the rapid and successful implementation of West Nile Virus testing to protect the safety of blood and tissues.
These unique scientific activities not only promote availability of safe, high quality products, but also enhance FDA’s expertise and help assure that we practice smart regulation. FDA’s web site provides additional information for the public on these and other regulatory scientific programs. FDA leverages its resources through our Collaborative Scientific Training Program, the National Cancer Institute / FDA Oncology Joint Fellowship Program, numerous other interactions with NIH, and through Critical Path collaboration.
Protecting and Promoting Global Health
FDA’s international activities and the importance of actively playing both leadership and partnership roles in global health have never been more significant. We live in an increasingly global and interdependent environment. Infectious disease threats such as malaria, chikungunya virus, and dengue fever (for which mosquito vectors are present in the U.S.), avian flu, new hepatitis viruses, HIV, TB, and diarrheal diseases not only affect millions around the world, but also directly or indirectly threaten our population. In addition, medical innovation, manufacturing, and regulation are increasingly global – posing challenges and opportunities. We therefore place a high priority on our global efforts, including fostering the development of quality products such as vaccines to address these threats. We are highly engaged in global information sharing, capacity building, and harmonization. We are a recognized WHO Collaborating Center both for influenza and for biological products and standards. We work through WHO and many other organizations to provide leadership and support to global efforts in blood, vaccine, and tissue safety, and to provide standards and guidance to enhance the development and quality of needed products. Our Global Vaccine Initiative helps to provide technical assistance and enhance regulatory capacity, and supports the work with WHO, with other regulators, and with manufacturers and nongovernmental groups to enhance efforts to control infectious diseases of major importance.
This is just a snapshot of our commitment to protect and promote global and U.S. health and address major global diseases such as tuberculosis, HIV, and malaria. Through these efforts, CBER seeks to help fulfill unprecedented opportunities to improve lives throughout the world while also meeting pressing needs to protect our nation and its people.
With dedication and teamwork from our diverse and knowledgeable staff, and through collaboration with many others inside and outside government, CBER has achieved and is committed to outstanding accomplishments in promoting innovation and the quality, safety and availability of the products we regulate. Despite new threats, our blood supply is safer than ever, and our vaccine infrastructure has dramatically improved, with record influenza vaccine capacity. Efforts to be better prepared for future pandemics are being supported and there is substantial progress.
We are proud of these and other accomplishments. Yet we face continuing challenges, risks, and tremendous opportunities. We must remain vigilant and continue to strive for improvement. Our Vision is of Innovative Technology Promoting Public Health – in the United States and globally. We are committed to this vision and our mission, and we welcome and appreciate your input, support and participation to achieve our common goals.
Thank you again for the opportunity to meet with you. I very much look forward to continuing to work together to succeed in our challenging but critical mission.
Last revised: April 19, 2011