关于药物开发决策的概念,方法和"成功概率"的使用:一个范围审查
Aysun Cetinyurek Yavuz1, Muhammad Bergas Nur Fayyad1, Ce Jiang2
1IQ Health Department, Radboud University Medical Center, Nijmegen, The Netherlands.
Clinical pharmacology and therapeutics
|January 25, 2025
概括
成功概率计算有助于药物开发决策. 利用外部数据源可以提高预测III期试验成功的准确性,从而更好地评估候选药物.
科学领域:
- 药物开发 药物开发
- 临床试验方法论 临床试验方法论
- 制药领域的决策过程
背景情况:
- 药物开发涉及漫长的过程,每个里程碑都存在固有的不确定性.
- 进入III期试验的决定在很大程度上取决于II期试验的结果.
- 成功概率 (PoS) 在制药决策中越来越多地使用,但不一致的术语阻碍了比较评估.
研究的目的:
- 对计算第三阶段试验成功概率的方法进行范围审查.
- 探索不同的数据源和终端可用性如何影响PoS计算.
- 通过基于PoS的程序,确定改善药物开发决策的方法.
主要方法:
- 对现有方法进行范围审查,以计算III期试验的成功概率.
- 基于数据源 (如真实世界数据,历史试验数据) 和终点可用性的方法分析.
- 评估外部数据如何优化PoS计算.
主要成果:
- 当第二阶段试验包括主要第三阶段终点数据时,PoS计算是直接的.
- 由于实际约束,II期试验经常使用代理或生物标记者终点.
- PoS程序可以有效地结合外部数据来完善试验成功概率和药物候选价值预测.
结论:
- 基于成功的程序的概率为优化药物开发决策提供了一个框架.
- 整合外部数据源显著提高了PoS计算的预测能力.
- 在工业和学术界进一步探索替代方法和应用是有必要的.
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