PREDICTIVE PROBABILITY EARLY TERMINATION PLANS FOR PHASE-II CLINICAL-TRIALS
PREDICTIVE PROBABILITY EARLY TERMINATION PLANS FOR PHASE-II CLINICAL-TRIALS
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DOI：
10.2307/2530109
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发表时间：
1979-01-01
期刊：
影响因子：
1.9
通讯作者：
HERSON, J
中科院分区：
文献类型：
--
作者：
HERSON, J
第二阶段临床试验旨在收集数据，以帮助决定实验性治疗是否具有足够的有效性，以证明进一步研究的合理性。在一项具有二分结果的单臂试验中，在N个患者的样本中，对Bernoulli参数的简单零假设与单侧替代方案进行了测试。当治疗无效时，应该存在提前终止试验的规则。基于二项分布以及二项参数的贝塔分布和均匀先验分布的预测概率可用作成组序贯设计的基础。讨论了这些设计的尺寸、功耗和平均样本量。其中包括说明提前终止计划的过程、关于量化先前信念的建议和说明性实例。
A phase II clinical trial is designed to gather data to help decide whether an experimental treatment has sufficient effectiveness to justify further study. In a one-arm trial with dichotomous outcome, a simple null hypothesis on the Bernoulli parameter was tested against a 1-sided alternative in a sample of N patients. A rule to terminate the trial early should exist when the treatment is ineffective. Predictive probabilities based on the binomial distribution and beta and uniform prior distributions for the binomial parameter are useful as the basis of group sequential designs. Size, power and average sample size for these designs are discussed. A process for the specification of an early termination plan, advice on the quantification of prior beliefs and illustrative examples are included.