title: Improving the flexibility and efficiency of phase II designs for oncology trials.
abstract: Phase II trials in oncology are usually conducted as single-arm two-stage designs with binary endpoints. Currently available adaptive design methods are tailored to comparative studies with continuous test statistics. Direct transfer of these methods to discrete test statistics results in conservative procedures and, therefore, in a loss in power. We propose a method based on the conditional error function principle that directly accounts for the discreteness of the outcome. It is shown how application of the method can be used to construct new phase II designs that are more efficient as compared to currently applied designs and that allow flexible mid-course design modifications. The proposed method is illustrated with a variety of frequently used phase II designs.
authors.affiliation: Institute of Medical Biometry and Informatics, University of Heidelberg, D-69120 Heidelberg, Germany. englert@imbi.uni-heidelberg.de
authors.forename: Stefan
authors.initials: S
authors.lastname: Englert
authors.forename: Meinhard
authors.initials: M
authors.lastname: Kieser
mesh_terms: D001699:Biometry; D017322:Clinical Trials, Phase II as Topic / Q000379:methods / Q000706:statistics & numerical data*; D006801:Humans; D008495:Medical Oncology / Q000379:methods / Q000706:statistics & numerical data; D015233:Models, Statistical; D009369:Neoplasms / Q000628:therapy*; D016896:Treatment Outcome