Ondra, Thomas, Dmitrienko, Alex, Friede, Tim, Graf, Alexandra, Miller, F. (Frank), Stallard, Nigel and Posch, Martin (2015) Methods for identification and confirmation of targeted subgroups in clinical trials : a systematic review. Journal of Biopharmaceutical Statistics . (In Press)

Preview

PDF WRAP_2015-08-13 Review submitted.pdf - Accepted Version - Requires a PDF viewer. Download (586Kb) | Preview

Request Changes to record.

Abstract

Important objectives in the development of stratified medicines include the identification and confirmation of subgroups of patients with a beneficial treatment effect and a positive benefit-risk balance. We report the results of literature review on methodological approaches to the design and analysis clinical trials investigating a potential heterogeneity of treatment effects across subgroups. The identified approaches are classified based on certain characteristics of the proposed trial designs and analysis methods. We distinguish between exploratory and confirmatory subgroup analysis, frequentist, Bayesian and decision-theoretic approaches and, lastly, fixed-sample, group-sequential and adaptive designs and illustrate the available trial designs and analysis strategies with published case studies.

Item Type:	Journal Article
Subjects:	R Medicine > RA Public aspects of medicine
Divisions:	Faculty of Medicine > Warwick Medical School > Health Sciences > Statistics and Epidemiology Faculty of Medicine > Warwick Medical School
Library of Congress Subject Headings (LCSH):	Clinical trials -- Methodology
Journal or Publication Title:	Journal of Biopharmaceutical Statistics
Publisher:	Taylor & Francis Inc.
ISSN:	1054-3406
Official Date:	14 August 2015
Dates:	Date Event 14 August 2015 Accepted 14 July 2015 Submitted
Date of first compliant deposit:	28 July 2016
Number of Pages:	54
Status:	Peer Reviewed
Publication Status:	In Press

Request changes or add full text files to a record

Repository staff actions (login required)

View Item

Downloads