Improving the Flexibility and Efficiency of Phase II Designs for Oncology Trials
Improving the Flexibility and Efficiency of Phase II Designs for Oncology Trials
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DOI：
10.1111/j.1541-0420.2011.01720.x
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发表时间：
2012-09
期刊：
影响因子：
1.9
通讯作者：
S. Englert;M. Kieser
中科院分区：
文献类型：
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作者：
S. Englert;M. Kieser
摘要肿瘤学的第二阶段试验通常采用双终点的单臂两阶段设计。目前可用的适应性设计方法是针对具有连续试验统计的比较研究而定制的。将这些方法直接转移到离散检验统计中会导致保守的程序，因此会损失力量。我们提出了一种基于条件误差函数原理的方法，它直接考虑了结果的离散性。说明了如何应用该方法来构造新的第二阶段设计，这些设计比当前应用的设计更有效，并且允许灵活的中途设计修改。提出的方法是以各种常用的第二阶段设计为例进行说明的。
Summary Phase II trials in oncology are usually conducted as single‐arm two‐stage designs with binary endpoints. Currently available adaptive design methods are tailored to comparative studies with continuous test statistics. Direct transfer of these methods to discrete test statistics results in conservative procedures and, therefore, in a loss in power. We propose a method based on the conditional error function principle that directly accounts for the discreteness of the outcome. It is shown how application of the method can be used to construct new phase II designs that are more efficient as compared to currently applied designs and that allow flexible mid‐course design modifications. The proposed method is illustrated with a variety of frequently used phase II designs.