OBJECTIVES: To describe industry perspectives related to the collection and use of patient experience data (PED) in Food and Drug Administration (FDA) regulatory submissions.

METHODS: An online survey of experiences (part 1) and best-worst exercise for prioritizing PED advancement efforts (part 2) was completed by industry and contract research organization (CRO) members with ≥2 years of recent experience with patient reported outcome (PRO), natural history study (NHS), and patient preference (PP) data; and direct experience with FDA filings including PED. The survey was pilot tested by 10 industry members, with the final survey disseminated by special interest groups within seven professional societies and consortia. Descriptive summaries were generated.

RESULTS: With 657 views of the survey, 50 eligible participants (84% industry, 16% CRO) completed part 1, among which 46 participants completed part 2. Respondents had PRO (86%), PP (50%), and NHS (18%) experience, most with ≥5 years of experience; and experience meeting with FDA to discuss PED use (72%). All respondents indicated that FDA meetings should have a standing agenda item to discuss PED use in decision-making, and most (78%) reported that FDA meetings to discuss PED use should occur before pivotal trials. Moreover, most responded that the FDA and Sponsor should co-develop the PED Table in the FDA clinical review (74%), and the table should report PED use (90%) or reason(s) for not using PED (96%) in regulatory decision-making. The most important efforts to advance the use of PED in decision-making were a dedicated meeting pathway and expanded FDA guidance (both ranked first in 51% of choices containing this item), followed by a roadmap (26%), collaborative initiatives (19%), and public inventory of acceptable methods (17%).

CONCLUSIONS: While the FDA has policy targets extending PED use, challenges still exist regarding pathways for submission and transparency regarding PED use in regulatory decision-making.