THIRD PLENARY SESSION: EXAMINING THE ROLE OF PATIENT PREFERENCES TO INFORM REGULATORY DECISIONS
The US Food and Drug Administration (FDA) user fee agreements (MDUFA IV and PDUFA VI) require the FDA to develop systematic approaches to develop fit-for-purpose tools to collect meaningful patient input that can be incorporated into regulatory review. The Center for Drug Evaluation and Research (CDER) and the Center for Devices and Radiological Health (CDRH) have taken different but complementary approaches to incorporate views of patients. CDRH has worked to understand quantitative preference elicitation methods to ascertain patients’ views about benefits and risks of medical products. CDER has focused on the qualitative preference elicitation methods. Preference researchers argue that when carefully conducted, stated-preference studies generate valid scientific evidence on patients’ views about relative desirability of harms, risks, benefits, and other features related to treatment alternatives. However, critics of preference research point out that humans often rely on decision heuristics, have poor numeracy, and fail tests of internal consistency. The panel will debate the strengths and limitations of stated-preference research and the extent to which patients’ views on benefit-risk tradeoffs for medical therapies can be relied upon to inform regulatory decisions.