title: Adaptive designs for confirmatory clinical trials with subgroup selection.
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.
authors.affiliation: a Warwick Medical School , University of Warwick , Coventry , United Kingdom.
authors.forename: Nigel
authors.initials: N
authors.lastname: Stallard
authors.forename: Thomas
authors.initials: T
authors.lastname: Hamborg
authors.forename: Nicholas
authors.initials: N
authors.lastname: Parsons
authors.forename: Tim
authors.initials: T
authors.lastname: Friede
mesh_terms: D000465:Algorithms; D002986:Clinical Trials as Topic / Q000331:legislation & jurisprudence / Q000706:statistics & numerical data*; D003198:Computer Simulation; D023381:Endpoint Determination; D006801:Humans; D009369:Neoplasms / Q000188:drug therapy / Q000628:therapy; D009934:Organization and Administration; D018579:Patient Selection; D012107:Research Design / Q000706:statistics & numerical data*; D018401:Sample Size; D012984:Software; D016896:Treatment Outcome