基于模拟的贝叶斯预测成功概率对临床试验与竞争事件数据的临时监测:两个案例研究
Chiara Micoli1, Alessio Crippa1, Jason T Connor2,3
1Department of Medical Epidemiology and Biostatistics, Karolinska Institutet, Stockholm, Sweden.
Pharmaceutical statistics
|November 25, 2025
概括
本研究引入了一种基于模拟的新方法,用于计算具有竞争风险的临床试验的贝叶斯预测成功概率 (PPoS). 这种方法使得更好的试验优化和徒劳停止决策.
科学领域:
- 生物统计学 生物统计学
- 临床试验设计 临床试验设计
- 流行病学 流行病学
背景情况:
- 贝叶斯预测成功概率 (PPoS) 对于优化临床试验设计和决策至关重要.
- 现有的PPoS计算方法不足以处理具有竞争事件数据的临床试验.
研究的目的:
- 开发和描述一种基于模拟的方法,用于计算PPoS在涉及竞争事件的临床试验.
- 为利用临时数据提供一种方法来预测试验成功概率.
主要方法:
- 用贝叶斯模型对因果特异性危险进行事件时间和事件类型的联合分布建模.
- 采用基于模拟的程序来预测试验结果和计算PPoS.
- 在模型参数的后面分布上数值平均成功的概率.
主要成果:
- 拟议的方法允许在存在竞争风险的情况下计算PPoS.
- 使用COVID-19和前列腺癌试验数据演示了该方法的应用.
- 展示了先前的分配选择如何在各种场景下影响PPoS评估.
结论:
- 开发的基于模拟的方法为竞争风险临床试验中的PPoS计算提供了可行的方法.
- 这种方法可以帮助优化试验规模,并做出关于徒劳性的知情决策.
- 该研究强调了在贝叶斯试验监测中考虑竞争事件的重要性.
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