1. Center for Biologics Evaluation and Research FDA Overview Site Visit Carolyn A. Wilson, Ph.D. Associate Director for Research

2. CBER’s Mission To ensure the safety, purity, potency, and effectiveness of biological products, including vaccines, blood and blood products, and cells, tissues and gene therapies for the prevention, diagnosis, and treatment of human diseases, conditions or injury.

3. Vision for CBER Protect and improve public and individual health in the US and, where feasible, globally Facilitate development, approval and access to safe and effective products and promising new technologies Strengthen CBER as a preeminent regulatory organization for biologics INNOVATIVE TECHNOLOGY ADVANCING PUBLIC HEALTH

4. CBER Regulates Complex Products XenotransplantationProducts Tissues Cell & Gene Therapies Blood, Blood Components and Derivatives Vaccines: Preventive & Therapeutic Related Devices Allergenic Products

5. CBER Mission: Role of Research Regulatory Challenge Challenge Public Health Novel Product CBERResearch Discovery New Tools RegulatoryPolicy/Decision LicensedProduct Improved Data – Benefit/Risk +

6. CBER Organization Director Deputy Associates Management Compliance, Biologics Quality Communication, Outreach and Development Biostatistics and Epidemiology Cellular, Tissue, and Gene Therapies Vaccines Research And Review Blood Research And Review

7. CBER Research Facilities Biotechnology Core Facility –Oligonucleotide, siRNA, PNA, and peptide synthesis –Peptide and DNA sequencing –Taqman Probe synthesis –HPLC; Capillary electrophoresis –Mass Spectrometry/Proteomics –Amino acid analysis Core support for –Flow Cytometry: Sorting/Analytic –Confocal microscopy Vivarium with procedure rooms –Rodents, NHP, BSL-2 capacity for infectious agents BSL-3 and ABSL-3 laboratories

8. Scientific Expertise Novel technologies: NMR, mass spec, flow cytometry, high throughput sequencing Microbiology: parasitology, bacteriology, virology Immunology Biochemistry and molecular biology Cell and developmental biology

9. CBER researcher = “Researcher-Regulator” ~20% CBER Staff Integration of research and review ensures Relevance, Expertise, Timeliness, and Usability

10. CBER RESEARCH MANAGEMENT OVERVIEW IDENTIFICATION OF REGULATORY AND PUBLIC HEALTH NEEDS CBER RESEARCH PRIORITIES OFFICE RESEARCH PLANS AND PRIORITIES RESEARCH PROGRAMS EXTERNAL REVIEW AND INPUT

11. CBER Research Priorities* Ensure safety, efficacy and availability of biologic products and use and development of appropriate regulatory pathways through Development and evaluation of methods, reagents, standards Evaluation, development, integration of novel scientific technologies and preclinical models for use in product regulation, including development and analysis of novel approaches that reduce, refine, or replace use of animals (3R’s) Facilitation of the development of new biological products for control of high priority public health threats, including pandemic influenza, emerging infectious diseases, and agents of bioterrorism. *FY10

12. Improving clinical trial design and evaluation, including adaptive design approaches Enhance risk management, risk assessment, and risk communication sciences Developing improved analytical tools and new means to access large medical databases to perform active population-based safety surveillance, including support of FDA's Sentinel Initiative. CBER Research Priorities - continued

13. Cyclic Peer Review of Every PI Every 4 Years Internal – Promotion, Conversion, Evaluation Committee External – peer review by scientific experts, Site Visits

14. Annual Review of Research PI provides For each project Progress report Future plans Budget Request Presentations, Pubs Other output Information reviewed Lab chief, DD, ADR, OD Relevance Productivity Quality Research Reporting Database Funding Allocated Relevance to priority Scientific/Reg Output Feasiblity

15. Site-Visit Report Draft report is distributed to full Advisory Committee Final report is approved by full Advisory Committee Final report used in many ways: –Internal peer review of research/PI by Promotion, Conversion, Evaluation Committee (PCE) for personnel actions –By PIs for improving research program –By management, resource allocation decisions may be impacted by report (pending resource availability)

16. Thank you! To the Site Visit reviewers and Advisory Committee Your input improves CBER’s research programs External review is critical to fulfilling our regulatory mission!