Phase II dose–response trials: A simulation study to compare analysis method performance under design considerations. Issue 5 (3rd September 2017)

• Record Type: Journal Article
• Authors: Rekowski, Jan; Köllmann, Claudia; Bornkamp, Björn; Ickstadt, Katja; Scherag, André
• Is Part Of: Journal of biopharmaceutical statistics. Volume 27:Issue 5(2017)
• Page Start: 885
• Page End: 901
• Publication Date: 2017-09-03
• Subjects: Dose finding -- dose-response curve -- minimum effective dose -- phase II trials -- proof of concept -- study design; Pharmacy -- Statistical methods -- Periodicals; Drugs -- Testing -- Statistical methods -- Periodicals; Biometry -- Periodicals; Biopharmaceutics -- Periodicals; Pharmacokinetics -- Periodicals; 615.19
• DOI: 10.1080/10543406.2017.1293078
• Languages: English
• ISSNs: 1054-3406

Abstract

ABSTRACT: Phase II trials are intended to provide information about the dose–response relationship and to support the choice of doses for a pivotal phase III trial. Recently, new analysis methods have been proposed to address these objectives, and guidance is needed to select the most appropriate analysis method in specific situations. We set up a simulation study to evaluate multiple performance measures of one traditional and three more recent dose-finding approaches under four design options and illustrate the investigated analysis methods with an example from clinical practice. Our results reveal no general recommendation for a particular analysis method across all design options and performance measures. However, we also demonstrate that the new analysis methods are worth the effort compared to the traditional ANOVA-based approach.