Presentation on theme: "Discussion: Biostatistics in Public Policy"— Presentation transcript:
1Discussion: Biostatistics in Public Policy Greg Campbell, Ph.D.Director, Division of Biostatistics Center for Devices and Radiological HealthFood and Drug AdministrationStatistics in Public Policy, ASA, February 3, 2012
2Outline Biostatisticians FDA Advisory Committees Guidance Documents Statistical ResearchBiostatistician Roles
3Biostatisticians Federal Government: Academic Departments of Biostatistics in Schools of Public Health and Schools of MedicineStatistics Departments and Mathematics DepartmentsFederal Government:NIH, FDA, CMS, CDC (including NCHS), EPA, VAState and local governmentIndustry (pharma, devices, biologics)Contract Research Organizations (CROs) and Academic Research Organizations (AROs)
4Food and Drug Administration Science-based agency that values transparency and innovationRegulates about 25% of the GDP of U.S.Makes enormous amount of pre-market and post market (adverse event reports) information available; preliminary information for FDA Adv. Com. and transcriptsCompanies generate data on which FDA bases almost all decisionsDoes not take cost into account in approval decisionsThe tradition of pre-specification in trial planning
5Federal Advisory Committee Act (FACA) About 1000 committees with a total of 45,000 members serving 54 federal agencies of the US government provide expert advice, shaping the programs and policies of the U.S. Government.Meetings are generally open unless there is a good reason for a closed meetingMeetings and committees announced in the Federal RegisterActivities are tracked by GSA
6FDA Advisory Committees FDA has at the agency level Advisory Committees for Science Board, Risk Communication, and PediatricsCenter for Drug Evaluation and Research has 16Center for Devices and Radiological Health has 4, one of which is Medical Device Advisory Committee, which has 174 members in 18 PanelsCenter for Biologic Evaluation and Research has 5Center for Food Safety and Nutrition has 1Center for Vet. Med. has 1National Center for Tox. Res. has 1
7FDA and Advisory Committees Usually one or more statisticians on each panel.Two types of meetings (all with public comment and patient and industry non-voting reps)Advice about a particular submission (PMA, HDE, sometimes a 510(k)) with presentations by the company and FDAAdvice about trial design or post-market issuesAll conflicts of participants are reviewedAll issues are discussed openly
8Statistical Issues at Panels Bayesian StatisticsUse of propensity score methodology for non-randomized studiesTreatment of missing dataBias in diagnostic studiesChallenge is to make the issues understandable to non-statisticians on the panel and in the audience.
9FDA Guidance Documents Guidance for Clinical Trial Sponsors: Establishment and Operation of Clinical Trial Data Monitoring Committees (March, 2006).Statistical Guidance on Reporting Results from Studies Evaluating Diagnostic Tests (March, 2007).The Use of Bayesian Statistics in Medical Device Clinical Trials (February, 2010).
11Pivotal Clinical Study Design Draft Guidance Discusses several concepts that are fundamental to Good Device Development Practices with respect to clinical trials.Some of these concepts have always been true, but have not been promulgated widely by the Agency
13Other Draft Guidance Documents Draft Guidance for Industry and Food and Drug Administration Staff - Evaluation of Sex Differences in Medical Device Clinical Studies (December, 2011).Adaptive Design Clinical Trials for Drugs and Biologics (issued by CDER and CBER, February, 2010).Non-Inferiority Clinical Trials (issued by CDER and CBER, March, 2010).
14Quantitative Decision Analysis Initiative in CDRH Led by Telba IronyHow FDA makes decisions on the approval or clearance of pre-market submissions and on post-market and compliance actionsQuantifying not only risks but also potential benefits.HDEs versus PMAs, first-of-a-kind device
15Some Products with Public Health Implications Prostate Screening Antigen (PSA) testsTest for Human Papilloma Virus (HPV)Evaluation of Digital MammographyClinical Trial Designs for Left Ventricular Assist DevicesPediatrics medical productsPersonalized medicine (Companion diagnostics, Drug–Diagnostic Co-developmentMicroarrays (MAQC-II)
16Biostatistical Research Funded by NSF, NIH and a little by FDAResearch review of scientists by NIH and FDAIntramural lab reviewsFDA Science Board SubcommitteeFDA Regulatory Statistical ResearchInternal to CDRH there is a Center Science Council
17National AcademiesIncludes National Academy of Science, Institute of MedicineStatistical committees CATS and CNSTATReportsThe Prevention and Handling of Missing Data in Clinical TrialsSmall n Clinical Trials
18The Role of the Biostatistician Communicate wellUnderstand the subject matter areaEducate non-statisticiansCommunicate not just point estimates but uncertainty (within the model and of the model)
19Closing RemarksWe need to be sure to communicate well with non-statisticians.We need to be able to partner well with decision-makers in many other disciplines.We statisticians can make a difference in people’s lives very day!