2. FDA ADVISORY COMMITTEES Mary A Foulkes, Ph.D. Office of Biostatistics and Epidemiology Center for Biologics Evaluation and Research, FDA FDA/Industry workshop Bethesda, MD September 19, 2003

3. Range of Advisory Committees CDER Cardio-Renal Drugs AntiInfective Drugs Antiviral Drugs Oncologic Drugs CBER Blood & Blood Products Vaccines CVM Veterinary Meds CDRH Circulatory System Devices Dental Devices Radiologic Devices CFSAN Food Advisory NCTR Ranch Hand

4. FACA Federal Advisory Committee Act Public Law 92-463 Independent advice on scientific issues Enhance, compliment, speed decision- making Provide forum for public input (advanced notice, public meetings) http://www.gsa.gov/Portal/browse/cha nnel.jsp?channelId=-13689http://www.gsa.gov/Portal/browse/cha nnel.jsp?channelId=-13689

5. FACA Scientific – Not Policy Advisory Only One hour for public comment Have Background material fr all sides 18 days prior to mtg (also webposted) Limitations on Last-Minute Material

6. INDEPENDENCE Appointed by Commissioner Fixed Terms Screened for COI/Bias Large Role Played by Chair

7. COMPOSITION Standing mbrs Scientific/Tech experts Statistician Consumer rep Industry rep Other invitees Patient rep Disease/condition experts Occasional Senators

8. Pre-Meeting IAs Generally Limited – Discussion of Issues must be in public Factual questions possible, but Exec Sec must be involved

9. FDA Reviewer General Public Executive Secretary Committee Member Industry

10. TYPICAL AGENDA 8:00 am 8:15 8:30 10:00 10:15 11:45 1:00 pm 2:00 4:30 Administrative remarks FDA Introduction Sponsor’s Presentation Break FDA’s Presentation Lunch Open Public Hearing Committee Disc’n & Vote Adjourn

11. Questions to ACs Were studies adequate to evaluate product effect? Was population studied appropriate? Is it appropriate to pool data? Optimal correction methodology for QT correction? Add’l pt safety monitoring advised? If licensed, should the label describe any limitations in generalizing to certain subgroups?

12. Issues for ACs Discuss trial designs, incl f/up dur’n Endpoints, esp surrogates Measurement/quant’n (e.g. QT interval & mult correction meths) Phase IV commitments 1 st year safety reviews of ped experience mandated by Best Pharmaceuticals for Children Act Generalizability of results

13. Perogative ACs can reword or answer the question they wish had been asked ACs often answer questions that were not asked, offering advice on future considerations ACs often ask for more information, more data (e.g. cross-resistance), more studies (e.g. Phase IV)

14. Statistician’s Responsibility Know their subject Know the process COMMUNICATE