RTI Health Solutions, Research Triangle Park, NC.
RTI Health Solutions, Ann Arbor, MI.
JCO Oncol Pract. 2023 May;19(5):e745-e762. doi: 10.1200/OP.22.00774. Epub 2023 Feb 28.
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).
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 , , and . Discussion of PRO-related topics was captured verbatim, organized thematically, and analyzed by two independent reviewers.
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.
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.
本项目旨在通过记录在六年期间（2016-2021 年）的 ODAC 会议记录中出现的与患者报告结局（PRO）相关的考虑因素，深入了解 PRO 数据在美国食品和药物管理局（FDA）肿瘤药物咨询委员会（ODAC）审查和建议中的作用。
审查 ODAC 会议记录，以了解任何与 PRO 相关概念的提及。排除了审查生物类似物的会议和讨论概念性问题的会议。对于每个确定的记录，从会议记录中收集会议日期、药物的品牌和通用名以及适应证。ODAC 成员、FDA 审查员和研究赞助商在审查过程中对 PRO 数据的评论被捕获。对记录进行定性审查包括阅读和搜索关键词，包括、和。PRO 相关主题的讨论逐字记录、按主题组织，并由两位独立评审员进行分析。
确定了 2016-2021 年的 27 份审查记录。其中 12 份记录包含与 PRO 相关的主题。在这 12 份记录中，ODAC 对其中 2 份记录中的 PRO 感到满意。在其余 10 份记录中，不满意的原因包括未评估关键概念（5/12）、缺失数据（5/12）以及与赞助商解释不一致（3/12）。ODAC 还对其中 6 份未包含 PRO 的记录表示不满，因为这些记录缺乏 PRO 数据。
在 2016-2021 年期间，ODAC 审查中不到一半包含 PRO，审查员对 PRO 数据的缺乏表示不满。即使包含在内，基于 PRO 的证据也很少被认为足以进行获益-风险评估。
