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J. Pers. Med. 2017, 7(1), 1; doi:10.3390/jpm7010001

Biomarker-Guided Non-Adaptive Trial Designs in Phase II and Phase III: A Methodological Review

1
MRC North West Hub for Trials Methodology Research, Liverpool L69 3GL, UK
2
Department of Biostatistics, Institute of Translational Medicine, University of Liverpool, Liverpool L69 3GL, UK
*
Author to whom correspondence should be addressed.
Academic Editor: Stephen B. Liggett
Received: 8 October 2016 / Revised: 6 December 2016 / Accepted: 11 January 2017 / Published: 25 January 2017
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Abstract

Biomarker-guided treatment is a rapidly developing area of medicine, where treatment choice is personalised according to one or more of an individual’s biomarker measurements. A number of biomarker-guided trial designs have been proposed in the past decade, including both adaptive and non-adaptive trial designs which test the effectiveness of a biomarker-guided approach to treatment with the aim of improving patient health. A better understanding of them is needed as challenges occur both in terms of trial design and analysis. We have undertaken a comprehensive literature review based on an in-depth search strategy with a view to providing the research community with clarity in definition, methodology and terminology of the various biomarker-guided trial designs (both adaptive and non-adaptive designs) from a total of 211 included papers. In the present paper, we focus on non-adaptive biomarker-guided trial designs for which we have identified five distinct main types mentioned in 100 papers. We have graphically displayed each non-adaptive trial design and provided an in-depth overview of their key characteristics. Substantial variability has been observed in terms of how trial designs are described and particularly in the terminology used by different authors. Our comprehensive review provides guidance for those designing biomarker-guided trials. View Full-Text
Keywords: biomarker-guided trial design; clinical research design; phase II; phase III; personalized medicine; predictive biomarker; prognostic biomarker; non-adaptive trial designs; clinical trials methodology; sample size biomarker-guided trial design; clinical research design; phase II; phase III; personalized medicine; predictive biomarker; prognostic biomarker; non-adaptive trial designs; clinical trials methodology; sample size
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This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).

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    Doi: doi:10.1371/journal.pone.0149803.s002
    Description: S1 Keywords. Literature review search strategies for both biomarker-guided clinical trial designs and for traditional trial designs.

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MDPI and ACS Style

Antoniou, M.; Kolamunnage-Dona, R.; Jorgensen, A.L. Biomarker-Guided Non-Adaptive Trial Designs in Phase II and Phase III: A Methodological Review. J. Pers. Med. 2017, 7, 1.

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