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临床试验中的自适应多重比较序贯设计 (AMCSD)。

Adaptive Multiple Comparison Sequential Design (AMCSD) for clinical trials.

机构信息

Innovatio Statistics, Inc., Bridgewater, New Jersey, USA.

School of Mathematics and Statistics, Changsha University of Science and Technology, Changsha, Hunan, China.

出版信息

J Biopharm Stat. 2024 May;34(3):424-440. doi: 10.1080/10543406.2023.2233590. Epub 2023 Aug 1.

Abstract

We propose an adaptive sequential testing procedure for clinical trials that test the efficacy of multiple treatment options, such as dose/regimen, different drugs, sub-populations, endpoints, or a mixture of them in one trial. At any interim analyses, sample size re-estimation can be conducted, and any option can be dropped for lack of efficacy or unsatisfactory safety profile. Inference after the trial, including p-value, conservative point estimate and confidence intervals, are provided.

摘要

我们提出了一种适用于临床试验的自适应序贯检验程序,该程序可以测试多种治疗方案的疗效,例如剂量/方案、不同药物、亚组、终点或在一次试验中混合使用它们。在任何中期分析中,都可以进行样本量重新估计,如果某种方案缺乏疗效或安全性不理想,可以将其剔除。在试验后,我们还提供了包括 p 值、保守点估计和置信区间在内的推断结果。

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