Current Practices and Challenges When Submitting Patient Experience Data for Regulatory Decisions by the US Food and Drug Administration: An Industry Survey

Cathy Anne Pinto (), Tommi Tervonen, Cecilia Jimenez-Moreno, Bennett Levitan, Montse Soriano Gabarró, Cynthia Girman, Josephine M. Norquist and Brett Hauber
Additional contact information
Cathy Anne Pinto: Merck & Co., Inc.
Tommi Tervonen: Kielo Research
Cecilia Jimenez-Moreno: Evidera
Bennett Levitan: Janssen Research and Development, LLC
Montse Soriano Gabarró: Bayer AG
Cynthia Girman: CERObs Consulting, LLC
Josephine M. Norquist: Merck & Co., Inc.
Brett Hauber: Pfizer

The Patient: Patient-Centered Outcomes Research, 2024, vol. 17, issue 2, No 4, 147-159

Abstract: Abstract Objectives To understand industry practices and challenges when submitting patient experience data (PED) for regulatory decisions by the US Food and Drug Administration (FDA). Methods A two-part online survey related to collection, submission, and use of PED by FDA in regulatory decision-making (part 1) and a best-worst exercise for prioritizing potential PED initiatives (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), or patient preference (PP) data; and direct experience with FDA filings including PED. Results A total of 50 eligible respondents (84% industry) completed part 1 of the survey, among which 46 completed part 2. Respondents mostly had PRO (86%) and PP (50%) experience. All indicated that FDA meetings should have a standing agenda item to discuss PED. Most (78%) reported meetings should occur before pivotal trials. A common challenge was justifying inclusion without knowing if and how data will be used. Most agreed that FDA and industry should co-develop the PED table in the FDA clinical review (74%), and the table should report reason(s) for not using PED (96%) in regulatory decision-making. Most important efforts to advance PED use in decision-making were a dedicated meeting pathway and expanded FDA guidance (51% each). Conclusions FDA has policy targets expanding PED use, but challenges remain regarding pathways for PED submission and transparency in regulatory decision-making. Alignment on the use of existing meeting opportunities to discuss PED, co-development of the PED table, and expanded guidance are encouraged.

Date: 2024
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DOI: 10.1007/s40271-023-00653-8

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