Testing for efficacy in adaptive clinical trials with enrichment

Samuel S. Wu, Yi Hsuan Tu, Ying He

Research output: Contribution to journalArticlepeer-review

2 Citations (Scopus)


Adaptive design of clinical trials has attracted considerable interest because of its potential of reducing costs and saving time in the clinical development process. In this paper, we consider the problem of assessing the effectiveness of a test treatment over a control by a two-arm randomized clinical trial in a potentially heterogenous patient population. In particular, we study enrichment designs that use accumulating data from a clinical trial to adaptively determine patient subpopulation in which the treatment effect is eventually assessed. A hypothesis testing procedure and a lower confidence limit are presented for the treatment effect in the selected patient subgroups. The performances of the new methods are compared with existing approaches through a simulation study.

Original languageEnglish
Pages (from-to)2736-2745
Number of pages10
JournalStatistics in Medicine
Issue number16
Publication statusPublished - 2014 Jul 20

All Science Journal Classification (ASJC) codes

  • Epidemiology
  • Statistics and Probability


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