Adaptive design of confirmatory trials: Advances and challenges

被引：13

作者：

Lai, Tze Leung ^{[1
]}

Lavori, Philip W. ^{[2
]}

Tsang, Ka Wai ^{[3
]}

机构：

[1] Stanford Univ, Dept Stat, Stanford, CA 94305 USA

[2] Stanford Univ, Dept Hlth Res & Policy, Stanford, CA 94305 USA

[3] Stanford Univ, Inst Computat & Math Engn, Stanford, CA 94305 USA

来源：

CONTEMPORARY CLINICAL TRIALS | 2015年 / 45卷

基金：

美国国家科学基金会; 美国国家卫生研究院;

关键词：

Adaptive design; Adaptive randomization; Bayesian inference; Early stopping; Hybrid resampling; Multi-arm bandits; FDA DRAFT GUIDANCE; GROUP SEQUENTIAL DESIGNS; CLINICAL-TRIALS; SAMPLE-SIZE; CONFIDENCE-INTERVALS; ISCHEMIC-STROKE; TREATMENT SELECTION; STOCHASTIC-SYSTEMS; TARGETED THERAPY; TESTS;

D O I：

10.1016/j.cct.2015.06.007

中图分类号：

R-3 [医学研究方法]; R3 [基础医学];

学科分类号：

1001 ;

摘要：

The past decade witnessed major developments in innovative designs of confirmatory clinical trials, and adaptive designs represent the most active area of these developments. We give an overview of the developments and associated statistical methods in several classes of adaptive designs of confirmatory trials. We also discuss their statistical difficulties and implementation challenges, and show how these problems are connected to other branches of mainstream Statistics, which we then apply to resolve the difficulties and bypass the bottlenecks in the development of adaptive designs for the next decade. (C) 2015 Elsevier Inc. All rights reserved.

引用

页码：93 / 102

页数：10

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