Covariate-Adaptive Sample Size Re-estimation for Population-Standardized Historical Control Designs in Single-Arm Trials
单臂试验总体标准化历史对照设计的协变量自适应样本量重估
Externally controlled single-arm trials are increasingly considered when randomized controls are infeasible, but baseline imbalance between the active-arm trial and historical controls complicates both estimation and sample size planning. We propose a population-standardized design framework in which the target estimand is defined for the actually enrolled active-arm population and historical-control outcomes are standardized to that population through a pre-specified…