Universität zu Köln

Bauer, Peter, Bretz, Frank ORCID: 0000-0002-2008-8340, Dragalin, Vladimir, Koenig, Franz and Wassmer, Gernot (2016). Twenty-five years of confirmatory adaptive designs: opportunities and pitfalls. Stat. Med., 35 (3). S. 325 - 348. HOBOKEN: WILEY. ISSN 1097-0258

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Abstract

Multistage testing with adaptive designs' was the title of an article by Peter Bauer that appeared 1989 in the German journal Biometrie und Informatik in Medizin und Biologie. The journal does not exist anymore but the methodology found widespread interest in the scientific community over the past 25years. The use of such multistage adaptive designs raised many controversial discussions from the beginning on, especially after the publication by Bauer and Kohne 1994 in Biometrics: Broad enthusiasm about potential applications of such designs faced critical positions regarding their statistical efficiency. Despite, or possibly because of, this controversy, the methodology and its areas of applications grew steadily over the years, with significant contributions from statisticians working in academia, industry and agencies around the world. In the meantime, such type of adaptive designs have become the subject of two major regulatory guidance documents in the US and Europe and the field is still evolving. Developments are particularly noteworthy in the most important applications of adaptive designs, including sample size reassessment, treatment selection procedures, and population enrichment designs. In this article, we summarize the developments over the past 25years from different perspectives. We provide a historical overview of the early days, review the key methodological concepts and summarize regulatory and industry perspectives on such designs. Then, we illustrate the application of adaptive designs with three case studies, including unblinded sample size reassessment, adaptive treatment selection, and adaptive endpoint selection. We also discuss the availability of software for evaluating and performing such designs. We conclude with a critical review of how expectations from the beginning were fulfilled, and - if not - discuss potential reasons why this did not happen. (c) 2015 The Authors. Statistics in Medicine Published by John Wiley & Sons Ltd.

Item Type:	Journal Article
Creators:	Creators Email ORCID ORCID Put Code Bauer, Peter UNSPECIFIED UNSPECIFIED UNSPECIFIED Bretz, Frank UNSPECIFIED orcid.org/0000-0002-2008-8340 UNSPECIFIED Dragalin, Vladimir UNSPECIFIED UNSPECIFIED UNSPECIFIED Koenig, Franz UNSPECIFIED UNSPECIFIED UNSPECIFIED Wassmer, Gernot UNSPECIFIED UNSPECIFIED UNSPECIFIED
URN:	urn:nbn:de:hbz:38-284657
DOI:	10.1002/sim.6472
Journal or Publication Title:	Stat. Med.
Volume:	35
Number:	3
Page Range:	S. 325 - 348
Date:	2016
Publisher:	WILEY
Place of Publication:	HOBOKEN
ISSN:	1097-0258
Language:	English
Faculty:	Unspecified
Divisions:	Unspecified
Subjects:	no entry
Uncontrolled Keywords:	Keywords Language MULTIPLE END-POINTS; SAMPLE-SIZE REESTIMATION; GROUP SEQUENTIAL DESIGNS; I ERROR RATE; II/III CLINICAL-TRIALS; PHRMA-WORKING-GROUP; PILOT-STUDY DESIGNS; TREATMENT SELECTION; 2-STAGE DESIGNS; CONFIDENCE-INTERVALS Multiple languages Mathematical & Computational Biology; Public, Environmental & Occupational Health; Medical Informatics; Medicine, Research & Experimental; Statistics & Probability Multiple languages
Refereed:	Yes
URI:	http://kups.ub.uni-koeln.de/id/eprint/28465