1. Center for Biologics Evaluation and Research, FDA Site Visit Introduction Kathryn M. Carbone, M.D. Associate Director for Research

6. Why CBER? Unique Role vis-à-vis the Science of Biologics Product Evaluation Innovators create scientific tools that are typically applicable to their specific products and not shared with others in industry CBER scientists are expert in biological products AND scientific disciplines as part of product development--they see the successes, failures, and missed opportunities CBER Guidance documents and other public communications of evaluation science provide a clearer and more predictive path for products CBER can play a convening and coordinating role for scientific needs across sponsors

7. Multitasking at the FDA: Research Supports Regulatory Mission CBER researchers fully integrated into the regulatory process (~50% average time) “Researcher-Regulator” model (unique to CBER) Review INDs, BLAs, 510ks, PMAs, IDEs, HDEs Development of Policy and Guidance Documents Meeting with Sponsors and Advisory Committees Participation in Pre-license and Biennial Inspections Evaluation of Adverse Drug Reactions and Risk Assessment Performing research relevant to product evaluation of safety, efficacy, manufacturing: Developing/evaluating scientific tools & knowledge Outreach, communication with stakeholders

8. Applicability of Research Programs to Biologics Development It’s not “basic vs. applied science”, it’s how the research output is applicable to supporting complex biologics product approvals and product safety

9. CBER RESEARCH MANAGEMENT INITIATIVE REGULATORY AND PUBLIC HEALTH PORTFOLIO ANALYSIS & KEY SCIENTIFIC CHALLENGES RESEARCH PRIORITIES END OF FY SCIENTIFIC PROGRAM REVIEW AND ANNUAL REPORT OFFICE & CENTER RESEARCH PLANS & BUDGET ADVISORY COMMITTEE PRESENTATIONS AND OTHER INPUT OUTCOMES FUTURE PLANS

10. Types of Research at CBER http://www.fda.gov/cber/research/rschovr.htm Creating regulatory pathways where there are few or none (e.g., Cell & Gene therapy) Applying 21 st Century science to improve standard regulatory pathways Product Safety ~40 % Product Quality ~25 % Product Efficacy ~ 25% Horizon Scanning ~ 10%

11. Guiding Principles The CBER research program will be highly collaborative and include laboratory, epidemiological, statistical, and clinical sciences. Its scope will encompass the scientific basis of pre-clinical and clinical studies, manufacturing, regulatory submissions, inspections, postmarketing surveillance and Guidances. The research will be of high quality, efficient, and directed and managed to provide outcomes that address scientific and regulatory challenges in product development, safety, efficacy and quality.

12. Site Visit Today: Staff Definitions Senior Investigators or Staff Scientists: Already “converted” staff = tenured Evaluated for Promotion or Progress Service Fellows, Non permanent FTEs= Service Fellows, Non permanent FTEs = Staff Fellow/Visiting Associate: Staff Fellow/Visiting Associate: Evaluated for Conversion to Staff Scientist (support scientist) Senior Staff Fellow/Visiting Scientist Senior Staff Fellow/Visiting Scientist Evaluated for Conversion to Senior Investigator

13. 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

14. Site-Visit Report Oral summary is presented at end of review today to Research Management Draft report is distributed to full Advisory Committee by Dr. Freas’ staff Final report is approved by full Advisory Committee Final report used for research evaluation and as part of material provided to Promotions and Conversion Evaluation Committee (PCE) review for personnel actions

15. Peer Evaluation for Conversion (“Tenure”) or Promotion of a Research-regulator Nominated by Division Director or Self- nominated Nominating Memorandum CV and Bibliography Recent Publications Letters of Recommendation AC approved report of Site-Visit Team AC approved report of Site-Visit Team Review by CBER PCE Committee Makes recommendation to Center Director

16. Thank you To the Advisory Committee members for their time, expertise and suggestions for continuing improvement of CBER research programs