Adaptive two-stage test procedures to find the best treatment in clinical trials

Wolfgang Bischoff and Frank Miller

Biometrika, 2005, vol. 92, issue 1, 197-212

Abstract: A main objective in clinical trials is to find the best treatment in a given finite class of competing treatments and then to show superiority of this treatment against a control treatment. The traditional procedure estimates the best treatment in a first trial. Then in an independent second trial superiority of this treatment, estimated as best in the first trial, is to be shown against the control treatment by a size α test. In this paper we investigate these two trials of this traditional procedure as a two-stage test procedure. Additionally we introduce competing two-stage group-sequential test procedures. Then we derive formulae for the expected number of patients. These formulae depend on unknown parameters. When we have a prior for the unknown parameters we can determine the two-stage test procedure of size α and power β that is optimal, in that it needs a minimal number of observations. The results are illustrated by a numerical example, which indicates the superiority of the group-sequential procedures. Copyright 2005, Oxford University Press.

Date: 2005
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