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偏态结局纵向临床研究中的灵活治疗效应推断框架AI 解读

Longitudinal continuous outcomes in clinical trials are commonly analyzed using mixed models for repeated measures (MMRM) under normality assumptions. However, many clinical outcomes are skewed, making mean-based treatment effects difficult to interpret and potentially reducing statistical efficiency. The Box--Cox MMRM (BCMMRM) approach accommodates skewness by enabling inference on model-based median differences via inverse transformation. However, BCMMRM typically assumes…

利用外部数据的贝叶斯单臂与混合对照临床试验设计：修正与扩展AI 解读

Limited patient availability complicates sample size determination in pediatric clinical trials. Although Bayesian methods incorporating external data offer a solution, rigorously controlling the type I error rate remains difficult. Psioda and Ibrahim (2019) proposed a simulation-based framework as a practical solution. However, although their framework was designed to relax the type I error control, this relaxation fails when the external data exhibit a large treatment…