Pharmacoepidemiologic Assessment

1. Pharmacoepidemiologic Assessment Judy A. Staffa, PhD, RPh Epidemiology Team Leader Division of Surveillance, Research & Communication Support Office of Drug Safety, CDER

2. When and why are pharmacoepidemiologic studies recommended? • To further characterize potential safety signals from controlled clinical trials • chronic exposures • patients with co-morbid conditions • To further evaluate and quantify a safety signal identified after approval • incidence rate vs. reporting rate

3. Pharmacoepidemiologic Study Design • Cohort • Case-control • Nested case-control • Hybrid designs

4. Minimum Requirements for a Good Pharmacoepidemiologic Study Protocol • Clearly specified study objectives • Critical review of the literature • Detailed research methods • Study population • Data sources • Projected study size • Methods for data collection/management/analysis

5. Studies using Automated Data: Factors affecting Choice of Database • Demographics of patients • Turnover rate/mobility • Plan coverage of study medications • Size of population available for study • Availability of outcomes of interest • Coding of outcomes of interest • Access to medical records

6. Registries • A systematic collection of defined events or product exposures in a defined patient population for a defined period of time

7. Good Registry Protocols • Objectives • Literature review • Detailed research methods • Patient recruitment and follow up • Projected sample size • Methods for data collection, management and analysis • Carefully designed data collection forms • Validation of findings

8. Surveys • Further evaluate safety signals • Assess knowledge of labeled events • Assess product use • Assess compliance with risk management programs1 • Address proprietary name confusion 1Concept Paper: Risk Management Programs

9. Good Survey Protocols • Objectives • Detailed research methods • Patient or provider recruitment and follow up • Projected sample size • Methods for data collection, management and analysis • Carefully designed survey instruments • Validation of findings

10. Reporting Safety Signals to FDA • Submit a synthesis of all available safety information • Provide an assessment of the risk/benefit profile of the product • Propose steps to further investigate through additional studies • Propose risk management programs as appropriate1 1Concept Paper: Risk Management Programs

11. FDA’s Assessment of Safety Signals • Magnitude of the signal • Precision of the signal • Consistency of findings across available data sources • Biological plausibility • Seriousness of the event • Degree of benefit • Availability of other therapies • Mitigate events occurring in population(s) through risk management programs1 1Concept Paper: Risk Management Programs

12. Questions for Public Comment Under what circumstances would a registry be useful as a surveillance tool and when would it cease to be useful?

13. Questions for Public Comment Under what circumstances would active surveillance strategies prove useful to identify as yet unreported adverse events?

14. Questions for Public Comment Under what circumstances would additional pharmacoepidemiologic studies be useful?