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时变安慰剂效应确证性临床试验中的半参数加权样条回归AI 解读

In confirmatory Phase 3 clinical trials with recruitment over the years, the underlying placebo effect may follow an unknown temporal trend. Taking a clinical trial on Hidradenitis Suppurativa (HS) as an example, fluctuations or variabilities are common in HS-related endpoints, mainly due to the natural disease characteristics, variations of evaluation from different physicians, and standard of care evolvement. The adjustment of time-varying placebo effects receives some…

减少三期临床试验设计中选择偏差的一类计算方法AI 解读

When designing confirmatory Phase 3 studies, one usually evaluates one or more efficacious and safe treatment option(s) based on data from previous studies. However, several retrospective research articles reported the phenomenon of ``diminished treatment effect in Phase 3'' based on many case studies. Even under basic assumptions, it was shown that the commonly used estimator could substantially overestimate the efficacy of selected group(s). As alternatives, we propose a…

深度神经网络引导的点估计集成学习AI 解读

In modern statistics, interests shift from pursuing the uniformly minimum variance unbiased estimator to reducing mean squared error (MSE) or residual squared error. Shrinkage based estimation and regression methods offer better prediction accuracy and improved interpretation. However, the characterization of such optimal statistics in terms of minimizing MSE remains open and challenging in many problems, for example estimating treatment effect in adaptive clinical trials…