Biostatistics and Research Decision Sciences (BARDS), Department of Epidemiology, Merck & Co., Rahway, NJ, USA.
Evidera, Bethesda, MD, USA.
Drug Discov Today. 2023 Oct;28(10):103719. doi: 10.1016/j.drudis.2023.103719. Epub 2023 Jul 17.
The European Medicines Agency (EMA) and FDA have policy goals of strengthening benefit-risk (B-R) capabilities; but how this has been translating into regulatory practice is unclear. A systematic review of oncology drug approvals between 2015 and 2020 was conducted with approvals identified through review of FDA and EMA annual reports, with extraction of information on submission, clinical program and B-R assessment from publicly available review documents. Data were extracted from 236 reviews (EMA: 66 new submissions, 100 label extensions; FDA: 70 new submissions). The standard of evidence for B-R assessments seems to have diversified over time; yet, despite policy targets to extend their use, these assessments rarely include patient experience or real-world data.
欧洲药品管理局（EMA）和美国食品药品监督管理局（FDA）都有加强获益-风险（B-R）评估能力的政策目标；但这在监管实践中是如何体现的尚不清楚。对 2015 年至 2020 年期间肿瘤药物批准的情况进行了系统评价，通过对 FDA 和 EMA 年度报告的审查确定了批准情况，并从公开的审查文件中提取了提交、临床计划和 B-R 评估的信息。数据取自 236 次审查（EMA：66 项新提交，100 项标签扩展；FDA：70 项新提交）。B-R 评估的证据标准似乎随着时间的推移而多样化；然而，尽管有政策目标来扩大其使用范围，但这些评估很少包括患者体验或真实世界的数据。
