PURPOSE: To discuss opportunities, challenges, and approaches to collecting patient preference information in clinical trials.

DESCRIPTION: New treatments are increasingly evaluated in a patient-centred manner in which benefit-risk assessments combine clinical data with patient preference information (PPI). For example, the FDA has issued specific guidance on PPI, and the EMA issued a positive opinion on PPI qualification. To ensure regulators can consider PPI, studies need to rise to the level of valid scientific evidence. For example, in most cases, the study population in the PPI study should reflect as closely as possible the proposed indicated population. While most patient-preference studies are constrained in their ability to comply with clinical recruitment standards, including PPI in clinical trials seems an intuitive response to such concerns. Preference elicitation within the trial population can also aid in exploring preference heterogeneity based on robust clinical data. However, the decision to collect PPI in a trial requires careful consideration of multiple factors, including but not limited to the alignment of instrument development and protocol timelines, managing the cognitive burden imposed on patients, legal/ethical requirements and opportunity costs. Dr. Ng will discuss how PPI collected in clinical trials can support regulatory benefit-risk assessments. Dr. Katz will draw on her experience using PPI in clinical trials to inform regulatory benefit-risk assessments from an industry perspective. Dr. Danyliv will discuss the role that PPI collected in trials can take in HTAs. Dr. Heidenreich will cover the operational perspective, with a focus on best practice. To enhance the discussion, session leaders will use interactive polling to elicit facilitators and barriers experienced by session participants.