Institute of Regulatory Science for Medical Products, China Pharmaceutical University, Nanjing, Jiangsu, China.
NMPA Key Laboratory for Drug Regulatory Innovation and Evaluation, China Pharmaceutical University, Nanjing, Jiangsu, China.
BMJ Open. 2023 Jun 7;13(6):e069132. doi: 10.1136/bmjopen-2022-069132.
We aimed to provide insight into differences in drug review decisions made by the US Food and Drug Administration's (FDA) accelerated approval (AA) pathway and the European Medicines Agency's (EMA) conditional marketing authorisation (CMA) pathway, and to add to the current knowledge base of drug approval processes.
DESIGN, SETTING, PARTICIPANTS: This cross-sectional study thoroughly examines novel oncology drugs with dual approval through FDA AA and EMA CMA between 2006 and 2021. Statistical analysis was performed from June to July 2022.
The study examined the regulatory differences between regions for dually approved novel oncology drugs, including approval decisions, pivotal efficacy clinical trials, speed of review and postmarketing obligations.
During this time period, there was a difference in the use of the FDA AA and the EMA CMA (FDA: EMA: 41.2%: 70.0%, p<0.05). Of the 25 drugs approved by both the FDA AA and the EMA CMA, 22 (88.0%) of the regulatory decisions were based on the same pivotal clinical trials. But there were more differences in the requirements for postmarketing obligations, with the EMA's postmarketing obligations focusing on the efficacy and safety of the drug (EMA: FDA: 63.0%: 27.0%, p＜0.05) and the FDA's postmarketing obligations focusing more on the efficacy (FDA: EMA: 73.0%: 23.9%, p＜0.05). In addition, both the USA and EU had some postmarketing obligations completed beyond the schedule (30.4% and 19.2% in the USA and EU, respectively), with the longest delays lasting 3.7 years (0.2-3.7 years) and 3.3 years (0.04-3.3 years) in the USA and EU, respectively.
The FDA and EMA have different orientations and benefit-risk balance considerations in the use of AA or CMA. It is also the case that the shortcomings in the design and implementation of postmarketing studies have made it a challenge to obtain the evidence needed to confirm a drug's benefits.
本研究旨在深入了解美国食品药品监督管理局（FDA）加速审批（AA）途径和欧洲药品管理局（EMA）有条件上市许可（CMA）途径下的药物审查决策差异，并为当前药物审批流程的知识库增添新内容。
设计、地点和参与者：本研究为一项回顾性研究，对 2006 年至 2021 年间通过 FDA AA 和 EMA CMA 双重批准的新型肿瘤学药物进行了彻底研究。统计分析于 2022 年 6 月至 7 月进行。
本研究考察了区域间对双重批准的新型肿瘤学药物的监管差异，包括批准决定、关键性疗效临床试验、审查速度和上市后义务。
在此期间，FDA AA 和 EMA CMA 的使用情况有所不同（FDA：EMA：41.2%：70.0%，p<0.05）。在 25 种同时通过 FDA AA 和 EMA CMA 批准的药物中，22 种（88.0%）的监管决策基于相同的关键性临床试验。但是，在后市场义务方面存在更多差异，EMA 的后市场义务侧重于药物的疗效和安全性（EMA：FDA：63.0%：27.0%，p<0.05），而 FDA 的后市场义务更侧重于疗效（FDA：EMA：73.0%：23.9%，p<0.05）。此外，美国和欧盟都有一些后市场义务提前完成（美国和欧盟分别为 30.4%和 19.2%），最长的延迟时间为 3.7 年（0.2-3.7 年）和 3.3 年（0.04-3.3 年）。
FDA 和 EMA 在使用 AA 或 CMA 方面有不同的定位和获益-风险平衡考虑。在后市场研究的设计和实施方面存在的不足，也使得获取确认药物获益所需的证据具有挑战性。
