Wu Samuel S, Tu Yi-Hsuan, He Ying
Department of Biostatistics, University of Florida, Gainesville, FL 32610, U.S.A.
Stat Med. 2014 Jul 20;33(16):2736-45. doi: 10.1002/sim.6127. Epub 2014 Feb 27.
Adaptive design of clinical trials has attracted considerable interest because of its potential of reducing costs and saving time in the clinical development process. In this paper, we consider the problem of assessing the effectiveness of a test treatment over a control by a two-arm randomized clinical trial in a potentially heterogenous patient population. In particular, we study enrichment designs that use accumulating data from a clinical trial to adaptively determine patient subpopulation in which the treatment effect is eventually assessed. A hypothesis testing procedure and a lower confidence limit are presented for the treatment effect in the selected patient subgroups. The performances of the new methods are compared with existing approaches through a simulation study.
由于自适应设计在临床试验开发过程中具有降低成本和节省时间的潜力，因此备受关注。在本文中，我们考虑在一个可能存在异质性的患者群体中，通过双臂随机临床试验评估试验治疗相对于对照治疗有效性的问题。具体而言，我们研究富集设计，该设计利用来自临床试验的累积数据来自适应地确定最终评估治疗效果的患者亚组。针对所选患者亚组中的治疗效果，提出了一种假设检验程序和一个下限置信区间。通过模拟研究将新方法的性能与现有方法进行了比较。
