Adaptive designs for confirmatory clinical trials with subgroup selection

J Biopharm Stat. 2014;24(1):168-87. doi: 10.1080/10543406.2013.857238.

Abstract

Growing interest in stratified medicine is leading to increasing importance of subgroup analyses in confirmatory clinical trials. Conventionally, confirmatory clinical trials either focus on a subgroup identified in advance or assess subgroup effects once the trial is completed. The focus of this article is methodology for adaptive clinical trials that both identify whether a treatment is particularly effective in a predefined subgroup, potentially enabling alteration of recruitment, and assess the effectiveness in the subgroup and/or whole population. Methods for such adaptive trials are described and compared, and the logistical and regulatory issues associated with such approaches are discussed.

Publication types

  • Review

MeSH terms

  • Algorithms
  • Clinical Trials as Topic / legislation & jurisprudence
  • Clinical Trials as Topic / statistics & numerical data*
  • Computer Simulation
  • Endpoint Determination
  • Humans
  • Neoplasms / drug therapy
  • Neoplasms / therapy
  • Organization and Administration
  • Patient Selection*
  • Research Design / statistics & numerical data*
  • Sample Size
  • Software
  • Treatment Outcome