U.S. Food and Drug Administration

1. U.S. Food and Drug Administration Notice: Archived Document The content in this document is provided on the FDA’s website for reference purposes only. It was current when produced, but is no longer maintained and may be outdated.

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

3. 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.

4. FD&C and PHS Acts Combined with Regulations Review of Data Submitted to IND Active Research at CBER External Discussion Internal CBER Discussion Rational Policy and Decisions CBER’s Approach to Regulation

5. CBER researcher =“Researcher-Regulator”~10% CBER Staff Integration of research and review ensures Relevance, Expertise, Timeliness, and Usability

6. 2. CBER RESEARCH PRIORITIES EXTERNAL REVIEW AND INPUT • IDENTIFICATION • OF • REGULATORY • AND • PUBLIC • HEALTH NEEDS 3. OFFICE RESEARCH PLANS AND PRIORITIES 4. RESEARCH PROGRAMS CBER RESEARCH MANAGEMENT OVERVIEW

7. 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 technologiesand preclinical models for use in product regulation • Facilitation of the development of new biological products for control of emerging high priority public health threats, including pandemic influenza, infectious diseases, and agents of bioterrorism. *FY09, Developed by CBER’s Research Leadership Council

8. CBER Research Priorities - continued • Development and analysis of novel approaches to evaluate biologics that reduce, refine, or replace use of animals (3R’s) • Improving clinical trial design and evaluation, includingadaptive design approaches • Enhance risk management, risk assessment, and risk communication sciences • Building on CBER pilots with CMS, CDC, VSD, others to enhance and extend active population-basedsafety surveillance by developing improved analytical tools and accessing large databases, including support of Sentinel Initiative.

9. Each Office Performs Annual Review of Research Programs Evaluation used to allocate research resources • Relevance • Office/CBER research priorities • Utilizes CBER’s unique expertise/perspective • Productivity: • Scientific publications in peer-reviewed journals • Patents (or patents filed) • Regulatory guidance, other output • Quality: • Impact factor of journal • Invited presentations • Recognition by peers – science citation index, work on editorial boards, grant awards, etc.

10. Site Visit Process • Each laboratory unit reviewed approximately every 4 years by outside peer review group of scientists (subcommittee to existing FDA Advisory Committee) • Site Visit Team receives written materials in advance: • Lab Structure • Regulatory responsibilities • Research accomplishments and future plans • One-two day site visit – team hears presentations in open session; followed by individual interviews with lab members • Team writes draft report – needs to be submitted to parent Advisory Committee, reviewed, approved or modified • Once finalized, submitted to CBER

11. Site-Visit Team: Evaluate individual PI’s and Service Fellows in a Laboratory Unit • RESEARCH accomplishments since last review cycle • RESEARCH proposals for next four years • Relative to time for research and research support available • Administrative/Management comments welcome • Regulatory activities and regulatory work quality NOT assessed by Site Visit Team

12. Site-Visit Team: Suggestions to get or continue “on the right track” • Evaluation of the quality of science • New research directions and approaches to be considered • Needed laboratory expertise • Changes in laboratory organization • New collaborations

13. CBER Uses Site Visit Reports • Justify resource allocations to laboratory programs (increase or decrease, FTEs, budget, space, etc) • Programmatic decisions • Adjustment of research path by PI • Included in promotion and conversion packages to the Promotion, Conversion and Evaluation Committee (internal peer review)

14. Report of the Subcommittee on Science and TechnologyPrepared for the FDA Science Board, November, 2007 “…CBER has a rigorous process for establishing priorities and the impact of Center research on regulation. In addition, the leadership of CBER insists upon integration of laboratory scientists both in the review and manufacturing site inspection process. External peer review of research programs is the norm rather than the exception.”