Innovatio Statistics, Inc., Bridgewater, New Jersey, USA.
School of Mathematics and Statistics, Changsha University of Science and Technology, Changsha, Hunan, China.
J Biopharm Stat. 2024 May;34(3):424-440. doi: 10.1080/10543406.2023.2233590. Epub 2023 Aug 1.
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.
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。