Optimal sample size for a series of pilot trials of new agents
Optimal sample size for a series of pilot trials of new agents
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DOI：
10.2307/2533060
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发表时间：
1996-09-01
期刊：
影响因子：
1.9
通讯作者：
Livingston, PO
中科院分区：
文献类型：
--
作者：
Yao, TJ;Begg, CB;Livingston, PO
提出了一种新的方法来确定一系列筛选试验的适当样本量，以确定有前途的新的治疗药物。提出这个问题的动机是认识到筛选新制剂是一个持续的过程。因此，像以前的作者所做的那样，固定总样本量似乎并不理想。相反，我们使用经验贝叶斯公式来修复错误率并优化个体样本大小，以最大限度地减少识别有希望代理的时间。当应用于纪念斯隆-凯特琳癌症中心探索性疫苗接种试验的大型历史经验数据时，该方法表明，相对较小的个体筛选试验在这种情况下是最佳的。使用自举技术的结果的可靠性进行评估。
A new approach is presented for determining the appropriate sample sizes for a series of screening trials to identify promising new therapeutic agents. The formulation of the problem is motivated by recognition of the fact that screening of new agents is a continuing process. Consequently, it does not seem ideal to fix the overall total sample size, as previous authors have done. Instead we fix the error rates and optimize the individual sample sizes to minimize the time to identify a promising agent, using an empirical Bayes formulation. When applied to data from the large historical experience of exploratory vaccination trials at Memorial Sloan-Kettering Cancer Center, the method demonstrates that relatively small individual screening trials are optimal in this setting. The reliability of the results is evaluated using bootstrapping techniques.