在盲目混合控制设计中重新估计样本大小,使用反向概率权重
Masahiro Kojima1, Shunichiro Orihara2, Keisuke Hanada3
1Department of Data Science for Business Innovation, Chuo University, Tokyo, Japan.
Statistics in medicine
|February 10, 2026
概括
混合控制设计使用历史数据,但可能会因共变量差异而损失功率. 本研究提出了两种盲目样本大小重估策略,使用反向概率加权 (IPW) 来保持在这种差异出现时的统计能力.
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
- 现实世界的数据分析.
背景情况:
- 综合历史和现实数据的混合控制设计越来越多地用于临床研究.
- 从历史控制中借鉴预先规定的信息至关重要,但可因共变量分布差异而受到挑战.
- 这种差异可能会限制有效的数据借用,可能会使统计能力低于目标水平.
研究的目的:
- 为混合控制设计提出新的样本大小重新估计策略.
- 为了应对当前和历史研究之间出现显著基线共变量差异时保持统计能力的挑战.
- 确保临床试验评估的稳定性,尽管潜在的数据异质.
主要方法:
- 开发两种在盲目的临床研究中适用的样本大小重新估计策略.
- 基于对当前或历史研究的分配概率的逆概率权重 (IPW) 的利用.
- 模拟研究,以评估在不同共变量分布下拟议策略的性能.
主要成果:
- 提出的基于IPW的策略在检测到基线共变量的巨大差异时,有效地将样本大小调整向上.
- 这些调整有助于防止统计能力的损失,否则会发生这种情况.
- 模拟演示了这些策略能够维持目标功率水平的能力.
结论:
- 本文所介绍的样本大小重新估计方法为面临共变异不平衡的混合控制设计提供了实际解决方案.
- 这些策略提高了利用外部数据进行的临床试验的可靠性和实力.
- 该案例研究说明了这些方法在现实世界临床研究中的可行应用.
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