PURPOSE:The purpose of this project was to gain insight into the role of patient-reported outcome (PRO) data in US Food and Drug Administration (FDA) Oncologic Drugs Advisory Committee (ODAC) reviews and recommendations by documenting PRO-related considerations that appear in transcripts of ODAC meetings over a 6-year period (2016-2021).METHODS:ODAC meeting transcripts were reviewed for any mention of PRO-related concepts. Meetings that reviewed biosimilars and meetings that discussed conceptual matters were excluded. For each identified transcript, the meeting date, brand and generic names of the drug, and indication were collected from the meeting minutes. Comments by ODAC members, FDA reviewers, and study sponsors on PRO data were captured during the review. Qualitative review of transcripts included both reading and searching for key terms, including PROs, quality of life, and health-related quality of life. Discussion of PRO-related topics was captured verbatim, organized thematically, and analyzed by two independent reviewers.RESULTS:Twenty-seven transcripts of reviews were identified for 2016-2021. Topics related to PROs were included in 12 of those 27 reviews. The ODAC was satisfied with PROs included in 2 of those 12 reviews. Reasons for dissatisfaction in 10 of the 12 reviews included key concepts not assessed (5/12), missing data (5/12), and disagreement with sponsors' interpretation (3/12). The ODAC also expressed dissatisfaction with the lack of PRO data in 6 of 15 reviews that did not include PROs.CONCLUSION:Less than half of ODAC reviews in 2016-2021 included PROs, and reviewers expressed frustration at the lack of PRO data. Even when included, evidence on the basis of PROs was rarely deemed adequate for benefit-risk assessments.
展开▼
