Inference on Selected Subgroups in Clinical Trials

• 成果类型: Article
• 署名作者: Guo, Xinzhou; He, Xuming
• 署名单位: University of Michigan System; University of Michigan
• 刊物名称: JOURNAL OF THE AMERICAN STATISTICAL ASSOCIATION
• ISSN/ISSBN: 0162-1459
• DOI: 10.1080/01621459.2020.1740096
• 发表日期: 2021
• 页码: 1498-1506
• 关键词: confidence-intervals; phase-iii

摘要

When existing clinical trial data suggest a promising subgroup, we must address the question of how good the selected subgroup really is. The usual statistical inference applied to the selected subgroup, assuming that the subgroup is chosen independent of the data, may lead to an overly optimistic evaluation of the selected subgroup. In this article, we address the issue of selection bias and develop a de-biasing bootstrap inference procedure for the best selected subgroup effect. The proposed inference procedure is model-free, easy to compute, and asymptotically sharp. We demonstrate the merit of our proposed method by reanalyzing the MONET1 trial and show that how the subgroup is selected post hoc should play an important role in any statistical analysis.for this article are available online.