美国食品和药物管理局 (FDA) 指定对经济和发展的影响:系统审查和元分析
Joab Williamson1,2, Syed Shahzad Hasan3, Vijay S Gc1,4
1School of Human and Health Sciences, University of Huddersfield, Huddersfield, UK.
概括
美国FDA的指定,如突破疗法和快速跟踪,提供经济效益,加速药物批准. 虽然Fast Track显示出最强的市场影响,但突破疗法导致批准时间最短.
科学领域:
- 药物经济学 药物经济学
- 药物发展政策 药物发展政策
- 监管科学 监管科学
背景情况:
- 美国FDA的指定 (突破疗法,快速跟踪,孤儿药物,再生医学高级疗法) 旨在加速药物开发.
- 这些名称对经济和发展的影响的综合性需要进一步研究.
研究的目的:
- 系统地审查FDA指定对第1天累积平均异常回报 (CAAR) 的影响.
- 评估FDA指标对药物批准时间表的影响,从调查性新药提交.
主要方法:
- 在多个数据库 (PubMed,Scopus等) 进行系统的文献搜索. 从1997年1月开始到2024年9月.
- 随机效应模型用于聚合估计和异质性评估 (Cochrane-Q, I2).
- 使用德拉蒙德和调整的巴克检查清单来评估研究质量.
主要成果:
- 审查中包括了25项研究.
- 所有名称的1日合并CAAR为6.12% (95%CI:3.64-8.61).所有名称中的1日合并CAAR为6.12% (95%CI:3.64-8.61).
- 快速通道指定显示了最强的直接市场影响 (8.20%),而突破性治疗的平均批准时间最短 (69.96个月).
结论:
- 美国食品和药物管理局 (FDA) 的指定提供了显著的经济优势,特别是对于较小的公司.
- 这些名称可以加快高优先级疗法的批准过程.
- 异质性,特别是RMAT,表明需要进一步研究疾病区域和公司规模的影响.
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