OBJECTIVES: This research aims to identify trends regarding types of patient experience data (PED) submitted to FDA and insights about how FDA considers PED.

METHODS: We identified all new molecular entities and new therapeutic biological products approved by the FDA’s Center for Drug Evaluation and Research in 2022 and reviewed the corresponding new drug application/biologics license application review documents to determine whether or not a PED Table was included. Next, we extracted information about 1) which types of PED were submitted by the sponsor; 2) additional information not submitted by the sponsor but identified by FDA staff; and 3) qualitative information about how PED guided decisions. All data was reviewed by two researchers.

RESULTS: Among the 37 drugs approved by CDER in 2022, clinical/multidisciplinary reviews were available for 32 at the time of this analysis. Of these, the vast majority included a PED Table (n=30). The tables documented that 21 drugs included clinical outcome assessments (COAs) as the type of PED submitted, with patient-reported outcomes (PRO) the most common type of COA. Several qualitative studies, patient-focused drug development meeting summary reports, natural history studies and patient preference studies were also considered. We observed significant differences across reviews in the level of detail included in the “Patient Experience Data” section. This ranged from checkmarks with little context to a detailed description of how qualitative interviews and other interactions with a patient organization informed FDA staff’s overall benefit-risk determination.

CONCLUSIONS: Among our sample of approved products, COAs, especially PROs were the most common type of PED submitted. We also identified other types of PED including data from qualitative interviews and patient-focused drug development meetings contributing to regulatory decisions. There is an opportunity to improve consistency in how PED Tables are filled in by FDA staff and to provide additional information about how PED guides decisions.