美国食品和药物管理局的药物开发工具资格计划是否改善了药物开发?
Felix Yang1,2, Imein Bousnina1, Anne Madej1
1Genentech, A Member of the Roche Group, South San Francisco, USA.
Therapeutic innovation & regulatory science
|May 4, 2025
概括
美国食品和药物管理局 FDA
科学领域:
- 药物开发 药物开发
- 监管科学 监管科学
- 临床结果评估 临床结果评估
背景情况:
- 药物开发工具 (DDT) 资格计划评估用于药物开发的临床结果评估 (COA).
- 合格的COA通过测量特定感兴趣的概念来帮助监管决策.
- 这项分析评估了FDA的审查表现和合格COA的采用情况.
研究的目的:
- 评估美国食品和药物管理局 (FDA) 在临床结果评估 (COA) 资格计划中审查申请的表现.
- 评估合格的COA在药物开发中被采用和使用的程度.
主要方法:
- 对FDA批准的总结基础 (SBA) 和COA汇编的分析.
- 从FDA数据库中检索了资格计划 (QP) 和完整资格包 (FQP) 的提交和审查时间表.
- 检查过的FDA COA 资格计划网站数据.
主要成果:
- 列出了86个COA,主要是患者报告的结果 (PRO).
- 46.7%的提交超出了目标审查时间;平均资格需要6年.
- 只有7种COA (8.1%) 符合条件,其中3种在11种药物批准中使用,主要是作为二级终点.
结论:
- 漫长和不可预测的审查时间表风险工具开发者和赞助商.
- 迪迪特资格计划对将合格的COA纳入临床开发的影响有限.
- 食品和药物管理局应该分享时间表并澄清COA的使用情况,以提高程序的实用性.
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