第一类药物在美国和欧洲经历了不同的监管治疗
Jihye Han1, Aaron S Kesselheim2
1Jihye Han (jihan@bwh.harvard.edu), Brigham and Women's Hospital and Harvard University, Boston, Massachusetts.
Health affairs (Project Hope)
|March 3, 2025
概括
第一类药物从食品和药物管理局 (FDA) 获得了大量的监管灵活性,特别是在瘤学领域. 这种方法有助于开发新药,尽管存在固有的不确定性.
科学领域:
- 药理学和药物开发领域
- 监管科学 监管科学
- 临床试验设计 临床试验设计
背景情况:
- 第一类药物提供了新的机制,但缺乏先前的临床经验,呈现了发展的不确定性.
- 了解监管途径对于将创新疗法推向市场至关重要.
研究的目的:
- 分析食品和药物管理局 (FDA) 和欧洲药品管理局 (EMA) 批准的第一类药物的监管途径.
- 为了比较FDA和EMA之间的审查持续时间,加速程序利用率和关键试验特征.
主要方法:
- 对186种第一类药物 (2013-2023) 的FDA批准数据的回顾性分析.
- 对FDA和EMA (2013-2022) 批准的121种药物的数据分析.
- 专注于审查时间,加快指定和关键试验设计 (终点,盲目,比较器).
主要成果:
- 美国食品和药物管理局表现出显著的灵活性,50%的第一类药物缺乏临床终点,30%的关键试验缺乏盲目/比较器.
- 瘤学药物表现出极大的灵活性,高达90%的药物缺乏临床终点和失明.
- 与EMA (30%) 相比,FDA的加速计划指定率很高 (81%);审查时间因治疗领域而异 (FDA的7.7-14.5个月).
结论:
- 美国食品和药物管理局对第一类药物,特别是瘤学药物,采取了相当大的监管灵活性,以促进其进入市场.
- 在FDA和EMA之间,加速程序使用和审查持续时间存在差异.
- 将监管灵活性与严格的证据评估相平衡,对于一流的药物批准至关重要.
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