Introduction to FDA’s Office of Vaccines Research and Review Norman W. Baylor, Ph.D. Director, Office of Vaccines Research and Review

OVRR Mission Statement Protect and enhance the public health by assuring the availability of safe and effective vaccines, allergenic extracts, and other related products.

We accomplish our mission by performing the following activities Review, evaluate and take appropriate action on INDs, BLAs, and amendments and supplements to these applications for vaccines and related products Plan and conduct research related to the development, manufacture and testing of vaccines, and related products Develop policy and procedures governing the pre-market review and evaluation of vaccines and related products Evaluate and test licensed vaccines and related products as appropriate prior to release and distribution of these products into the market by manufacturers

We accomplish our mission by performing the following activities (cont’d) Evaluate and monitor clinical experience and reports of adverse events as necessary in coordination with CBER’s Office of Biostatistics and Epidemiology Participate in inspections of manufacturing facilities Participate in national and international outreach activities, including collaborations with global National Regulatory Authorities

OFFICE OF VACCINES RESEARCH AND REVIEW DIRECTOR Norman Baylor Ph.D. DEPUTY DIRECTOR Florence Houn M.D. Associate Director for Regulatory Policy: Marion Gruber, Ph.D. Special Assistant For Communications Maureen Hess MPH, RD Associate Director For Management and Scientific Affairs Erik Henchal, Ph.D. Associate Director of Research: Michael Brennan, Ph.D. Division of Bacterial, Parasitic and Allergenic Products. Director: Richard Walker, Ph.D. Deputy: Milan Blake, Ph.D. Division of Vaccines and Related Products Applications Act. Dir: Paul Richman, Ph.D.. Act. Deputy: Loris McVittie, Ph.D. Division of Viral Products Director: Jerry Weir, Ph.D. Deputy: Philip Krause M.D. updated 4/13/06 Division of Product Quality Dir: William McCormick, PhD

The Role of Research in OVRR Supports science-based regulatory review and decision making. –Provides expert review of regulatory submissions, e.g., INDs and BLAs, in various scientific disciplines –Addresses product-related issues in the laboratory, as the need arises –Influences policy and guidance on new technologies and the application of existing technologies to new areas Essential component of the regulatory review process to assure the safety, purity, potency, and effectiveness of vaccines and related products. –Needs to be sufficiently open-ended to provide the ability to respond to new areas Serves to recruit and maintain highly qualified scientists.

Research Priorities Within the broad range of scientific disciplines, certain programs must be maintained for their importance to the general needs of OVRR. –These areas are established by the OVRR leadership, in concert with the scientific staff –The needs of the regulatory review process drives research priorities. Broad research expertise in vaccine-related disciplines, e.g., bacteriology, virology, immunology and biochemistry. –Allows OVRR to shift priorities when public health emergencies arise. Research projects and their relative priority change over time, –Necessary to continually evaluate research needs.

The Process of Setting Priorities The ultimate decision on prioritization results from a subjective, but reasoned, evaluation of the following: –Priority setting by relevance Nature of research programs depends on the importance, and outcomes have implications for an extensive set of existing issues, e.g., product safety and characterization. –Priority setting by uniqueness and feasibility Are there specific considerations that compel the project to be done by OVRR scientists, i.e., are OVRR scientists in a special position, due to specialized knowledge, availability of reagents or technical expertise, to conduct the research. –E.g., potency and serological assay development –Special Considerations Research programs must be able to rapidly respond to emergencies as they arise.

High Priority Research Areas Safety issues related to vaccines and related products Product characterization Identification of immunological mechanisms Mechanisms of pathogenicity Emerging Issues

Evaluation of OVRR Research Programs Performed on at least an annual basis in OVRR Process begins in Divisions -Evaluation of PI Res Prog by Lab Chief-Division Director -Evaluates research progress (pubs; presentations; outreach) and Regulatory Workload (INDs, BLAs, Meetings w/ Pharma) Evaluation of Division Research Programs by OVRR to address: - FDA regulatory needs - emerging issues - future issues ( years ) -recommendations made by external Advisory groups Individual PIs evaluated for promotions by CBER PCE committee

Sources of Funding for OVRR Research National Vaccine Program Office Awards - 6 in FY 05, 7 in FY 06 Biodefense related awards – ORDC, DARPA, DOD - 9 ORDC in FY 06 Intraagency Agreements – NIH, CDC - Cell Substrates IAA w NIAID - NIAID-CBER Vaccine Development Partnership CRADAs – Universities, Foundations - TB assay- AERAS Foundation

Workshops Co-sponsored by OVRR “Assaying Potency of Novel Vaccines” with DAIDS, DMID, VRC October 2005, Bethesda, MD “The 14th International Pathogenic Neisseria Conference”, September 2004, Milwaukee, WI “NIAID/NIH – CBER/FDA TB Vaccine Regulatory Workshop”, December 2003, Bethesda, MD “Fourth International Conference on Tularemia”, September Bath, UK Global Activities WHO Collaborating Center – e.g., annual Flu strain selection, TB standards WHO/PAHO Expert Advisors BioTechnology Engagement Program [Former Soviet Union collaborations] CBER Global Vaccine Initiative OVRR OUTREACH ACTIVITIES

Summary OVRR’s research program serves to recruit, train and retain highly qualified scientists who possess the necessary knowledge and technical skills to conduct research and review that will facilitate the development of new and innovative vaccines and related products that are safe and effective, and will contribute to the health and well-being of the public.