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Methods for identification and confirmation of targeted subgroups in clinical trials: A systematic review
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2016 (English)In: Journal of Biopharmaceutical Statistics, ISSN 1054-3406, E-ISSN 1520-5711, Vol. 26, no 1, 99-119 p.Article in journal (Refereed) Published
Abstract [en]

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 a literature review on methodological approaches to the design and analysis of 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, last, fixed-sample, group-sequential, and adaptive designs and illustrate the available trial designs and analysis strategies with published case studies.

Place, publisher, year, edition, pages
2016. Vol. 26, no 1, 99-119 p.
Keyword [en]
Enrichment design, personalized medicine, precision medicine, predictive biomarkers, subgroup identification
National Category
Medical and Health Sciences Probability Theory and Statistics
Identifiers
URN: urn:nbn:se:su:diva-134708DOI: 10.1080/10543406.2015.1092034ISI: 000368725300009OAI: oai:DiVA.org:su-134708DiVA: diva2:1037444
Funder
EU, FP7, Seventh Framework Programme, 602144.
Available from: 2016-10-14 Created: 2016-10-14 Last updated: 2017-11-29Bibliographically approved

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