p-value calculation for multistage phase II cancer clinical trials
p-value calculation for multistage phase II cancer clinical trials
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DOI：
10.1080/10543400600825645
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发表时间：
2006-11-01
影响因子：
1.1
通讯作者：
Lee, Taiyeong
中科院分区：
文献类型：
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作者：
Jung, Sin-Ho;Owzar, Kouros;Lee, Taiyeong
由于伦理和实际问题，临床试验分多个阶段进行，但报告的p值往往不能反映试验的设计方面。我们研究了在分析以应答等二元变量作为主要终点的多阶段II期临床试验中p值计算的一些方法。样本空间由停止阶段的配对结果和响应数量组成，共同定义了真实二项比例的完整和充分的统计量。计算p值需要对配对的结果进行排序，以便能够识别比观察到的结果更极端的结果。我们考虑了基于极大似然估计和一致最小方差无偏估计的排序。我们将使用一些例子来比较基于这些替代排序的p值和忽略第二阶段试验的多阶段设计方面的p值。
Due to ethical and practical issues, clinical trials are conducted in multiple stages, but the reported p-values often fail to reflect the design aspect of the trials. We investigate some approaches to p-value calculation in analyzing multi-stage Phase II clinical trials that have a binary variable, such as response, as the primary endpoint. The sample space consists of the paired outcomes of the stopping stage and the number of responses, which jointly define a complete and sufficient statistic for the true binomial proportion. Calculating a p-value requires an ordering of the paired outcomes so that outcomes more extreme than the observed can be identified. We consider the orderings based on the maximum likelihood estimator and the uniformly minimum variance unbiased estimator. We will compare, using some examples, the p-values based on these alternative orderings and the one ignoring the multistage design aspect of phase II trials.