Patient Experience Data (PED) in 2019-2023 US FDA NME Drug Approvals: Analysis and Recommendations.

Background: Since 2012, the United States Congress enacted several laws requiring the U.S. Food and Drug Administration (FDA) to consider the voice and perspective of patients in the regulatory review process. The goal of the paper is to evaluate the implementation and impact of this provision by assessing the inclusion of Patient Experience Data (PED) (i.e., in PED Tables and benefit-risk frameworks) in approval documents for FDA approved drugs from 2019 through 2023. It builds on previous analyses by providing a comprehensive assessment of the use of PED by sponsors and FDA.

Methods: Authors assessed whether PED was submitted and reported in approval documents of 277 drugs or biologics approved by FDA's Center for Drug Evaluation and Research (CDER) and Center for Biologic Evaluation and Research (CBER). PED reported in drug approval documents and in product labels were analyzed for each indication.

Results: Of the 277 approval documents analyzed, 252 included the PED Table in some form, where 179 tables were considered complete with PED. PED was included in the benefit risk framework for 85 (30%) applications and included in the label in 75 (27%) applications.

Conclusions: Efforts to standardize and improve the systematic approach to collecting and utilizing PED has been ongoing for over 10 years. Our analysis has shown that both FDA and sponsors are increasingly considering patient voice to inform drug development and decision-making; however, more transparency is needed to ensure external stakeholders understand how FDA is reviewing and considering PED to inform regulatory decision-making.