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

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 t...

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Autores principales: Antoniou, Miranta, Kolamunnage-Dona, Ruwanthi, Jorgensen, Andrea L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2017
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5374391/
https://www.ncbi.nlm.nih.gov/pubmed/28125057
http://dx.doi.org/10.3390/jpm7010001
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author Antoniou, Miranta
Kolamunnage-Dona, Ruwanthi
Jorgensen, Andrea L.
author_facet Antoniou, Miranta
Kolamunnage-Dona, Ruwanthi
Jorgensen, Andrea L.
author_sort Antoniou, Miranta
collection PubMed
description 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.
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spelling pubmed-53743912017-04-10 Biomarker-Guided Non-Adaptive Trial Designs in Phase II and Phase III: A Methodological Review Antoniou, Miranta Kolamunnage-Dona, Ruwanthi Jorgensen, Andrea L. J Pers Med Review 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. MDPI 2017-01-25 /pmc/articles/PMC5374391/ /pubmed/28125057 http://dx.doi.org/10.3390/jpm7010001 Text en © 2017 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Antoniou, Miranta
Kolamunnage-Dona, Ruwanthi
Jorgensen, Andrea L.
Biomarker-Guided Non-Adaptive Trial Designs in Phase II and Phase III: A Methodological Review
title Biomarker-Guided Non-Adaptive Trial Designs in Phase II and Phase III: A Methodological Review
title_full Biomarker-Guided Non-Adaptive Trial Designs in Phase II and Phase III: A Methodological Review
title_fullStr Biomarker-Guided Non-Adaptive Trial Designs in Phase II and Phase III: A Methodological Review
title_full_unstemmed Biomarker-Guided Non-Adaptive Trial Designs in Phase II and Phase III: A Methodological Review
title_short Biomarker-Guided Non-Adaptive Trial Designs in Phase II and Phase III: A Methodological Review
title_sort biomarker-guided non-adaptive trial designs in phase ii and phase iii: a methodological review
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5374391/
https://www.ncbi.nlm.nih.gov/pubmed/28125057
http://dx.doi.org/10.3390/jpm7010001
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