Practical guidelines for adaptive seamless phase II/III clinical trials that use Bayesian methods
Practical guidelines for adaptive seamless phase II/III clinical trials that use Bayesian methods
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DOI：
10.1002/sim.5326
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发表时间：
2012-08-30
影响因子：
2
通讯作者：
Stallard, Nigel
中科院分区：
文献类型：
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作者：
Kimani, Peter K.;Glimm, Ekkehard;Stallard, Nigel
Hommel（Biometrical Journal; 45：581-589）提出了用于无缝II/III期临床试验的灵活测试程序。Schmidli等人（Statistics in Medicine; 26：4925-4938）、Kimani等人（Statistics in Medicine; 28：91-7936）和Brannath等人（Statistics in Medicine; 28：1445-1463）利用Hommel的灵活检验提出了无缝II/III期临床试验中的适应性，该试验通过使用贝叶斯方法结合了先验知识。在本文中，我们表明，适应结合先验知识可能会导致更高的权力。在这种自适应设计中需要考虑的其他重要问题是，与传统测试相比，功率的增益（或节省患者）以及用于进行自适应的效用值如何用于提前停止试验。与上述作者相比，我们详细讨论了这些问题，并提出了一种统一的方法来解决这些问题，以便实现上述设计和提出类似的设计更加清晰。版权所有（C）2012约翰威利父子有限公司
Hommel (Biometrical Journal; 45:581-589) proposed a flexible testing procedure for seamless phase II/III clinical trials. Schmidli et al. (Statistics in Medicine; 26:4925-4938), Kimani et al. (Statistics in Medicine; 28:91-7936) and Brannath et al. (Statistics in Medicine; 28:1445-1463) exploited the flexible testing of Hommel to propose adaptation in seamless phase II/III clinical trials that incorporate prior knowledge by using Bayesian methods. In this paper, we show that adaptation incorporating prior knowledge may lead to higher power. Other important issues to consider in such adaptive designs are the gain in power (or saving in patients) over traditional testing and how utility values used to make the adaptation may be used to stop a trial early. In contrast to the aforementioned authors, we discuss these issues in detail and propose a unified approach to address them so that implementing the aforementioned designs and proposing similar designs is clearer. Copyright (C) 2012 John Wiley & Sons, Ltd.