The use of response-adaptive designs in clinical trials

Abstract

Response-adaptive designs in clinical trials are schemes for patient assignment to treatment, the goal of which is to place more patients on the better treatment based on patient responses already accrued in the trial. While ethically attractive at first glance, these designs have had very little use in practice; yet the statistical literature is rich on this subject. We discuss procedures and properties of these designs. Particular focus is given to the randomized play-the-winner rule of Wei and Durham, which was used in the ECMO trial. We also discuss reasons for the lack of use of these models, and areas of current and future research to address the weaknesses of these methods. We conclude that these designs may be applicable in some situations and describe conditions under which such a trial may be feasible.