Department of Mathematics, Northeastern University, Boston, Massachusetts, USA.
Department of Biostatistics, University of Florida, Gainesville, Florida, USA.
J Biopharm Stat. 2020;30(1):18-30. doi: 10.1080/10543406.2019.1609015. Epub 2019 May 28.
We propose an adaptive enrichment approach to test an active factor, which is a factor whose effect is non-zero in at least one subpopulation. We implement a two-stage play-the-winner design where all subjects in the second stage are enrolled from the subpopulation that has the highest observed effect in the first stage. We recommend a weighted Fisher's combination of the most powerful test for each stage, respectively: the first stage Hotelling's test and the second stage noncentral chi-square test. The test is further extended to cover binary outcomes and time-to-event outcomes.
我们提出了一种自适应富集方法来检验一个活跃因子，该因子在至少一个亚群中具有非零的效应。我们实施了两阶段胜者设计，其中第二阶段的所有受试者都来自第一阶段观察到效果最高的亚群。我们分别推荐了每个阶段最有力检验的加权 Fisher 组合：第一阶段的 Hotelling 检验和第二阶段的非中心卡方检验。该检验进一步扩展到涵盖二分类结局和生存时间结局。
