在随机试验中估计治疗效果,使用外部控制对治疗交叉进行控制后进行随机试验
Xiner Zhou1,2, Herbert Pang2,3, Christiana Drake1
1Department of Statistics, University of California, Davis, California, USA.
Journal of biopharmaceutical statistics
|April 1, 2024
概括
使用外部控制的新方法使得在初级终点之后,在临床试验中能够对长期治疗效果进行估计. 建议使用差异差异 (DID) 方法,而不是合成控制方法 (SCM),以提高准确性.
科学领域:
- 临床试验设计和方法论
- 生物统计学 生物统计学
- 因果推理因果推理
背景情况:
- 临床试验往往允许与初级终点后的实验性治疗交叉,特别是在开放式扩展阶段.
- 在这些阶段评估长期的安全性和有效性至关重要,但在没有安慰剂对照的情况下具有挑战性.
- 当安慰剂组无法使用时,外部对照提供了一个可靠估计的潜在解决方案.
研究的目的:
- 提出新的因果推断方法,以通过结合随机对照试验 (RCT) 和外部对照来估计长期治疗效果.
- 在现实的场景中评估拟议的差异差异 (DID) 和合成控制方法 (SCM) 的性能.
主要方法:
- 开发了几种差异差异 (DID) 类型估计器.
- 实施一种合成控制方法 (SCM).
- 随机对照试验数据与外部对照数据在因果推理框架内进行组合.
主要成果:
- 模拟研究表明,在各种实际场景中,拟议的估计器具有理想的性能.
- 差异差异 (DID) 方法始终优于合成控制方法 (SCM).
- 这些方法已成功应用于罕见疾病环境中的III期临床试验.
结论:
- 结合RCT与外部对照是可行的,以估计在开放式扩展阶段的长期治疗效果.
- 拟议的DID方法是有效的,并为此目的推.
- 这种方法对于罕见疾病的临床试验特别有价值,因为传统的对照可能是有限的.
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