title: Testing for efficacy in adaptive clinical trials with enrichment.
abstract: 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.
authors.affiliation: Department of Biostatistics, University of Florida, Gainesville, FL 32610, U.S.A.
authors.forename: Samuel S
authors.initials: SS
authors.lastname: Wu
authors.forename: Yi-Hsuan
authors.initials: YH
authors.lastname: Tu
authors.forename: Ying
authors.initials: Y
authors.lastname: He
mesh_terms: D006801:Humans; D015233:Models, Statistical; D016032:Randomized Controlled Trials as Topic / Q000706:statistics & numerical data; D012107:Research Design; D016896:Treatment Outcome
keywords: clinical trial; enrichment; hypothesis test; lower confidence limit; two-stage design