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Do Bayesian adaptive trials offer advantages for comparative effectiveness research? Protocol for the RE-ADAPT study

BACKGROUND: Randomized clinical trials, particularly for comparative effectiveness research (CER), are frequently criticized for being overly restrictive or untimely for health-care decision making. PURPOSE: Our prospectively designed REsearch in ADAptive methods for Pragmatic Trials (RE-ADAPT) stud...

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Autores principales: Connor, Jason T, Luce, Bryan R, Broglio, Kristine R, Ishak, K Jack, Mullins, C Daniel, Vanness, David J, Fleurence, Rachael, Saunders, Elijah, Davis, Barry R
Formato: Online Artículo Texto
Lenguaje:English
Publicado: SAGE Publications 2013
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3834735/
https://www.ncbi.nlm.nih.gov/pubmed/23983160
http://dx.doi.org/10.1177/1740774513497293
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author Connor, Jason T
Luce, Bryan R
Broglio, Kristine R
Ishak, K Jack
Mullins, C Daniel
Vanness, David J
Fleurence, Rachael
Saunders, Elijah
Davis, Barry R
author_facet Connor, Jason T
Luce, Bryan R
Broglio, Kristine R
Ishak, K Jack
Mullins, C Daniel
Vanness, David J
Fleurence, Rachael
Saunders, Elijah
Davis, Barry R
author_sort Connor, Jason T
collection PubMed
description BACKGROUND: Randomized clinical trials, particularly for comparative effectiveness research (CER), are frequently criticized for being overly restrictive or untimely for health-care decision making. PURPOSE: Our prospectively designed REsearch in ADAptive methods for Pragmatic Trials (RE-ADAPT) study is a ‘proof of concept’ to stimulate investment in Bayesian adaptive designs for future CER trials. METHODS: We will assess whether Bayesian adaptive designs offer potential efficiencies in CER by simulating a re-execution of the Antihypertensive and Lipid Lowering Treatment to Prevent Heart Attack Trial (ALLHAT) study using actual data from ALLHAT. RESULTS: We prospectively define seven alternate designs consisting of various combinations of arm dropping, adaptive randomization, and early stopping and describe how these designs will be compared to the original ALLHAT design. We identify the one particular design that would have been executed, which incorporates early stopping and information-based adaptive randomization. LIMITATIONS: While the simulation realistically emulates patient enrollment, interim analyses, and adaptive changes to design, it cannot incorporate key features like the involvement of data monitoring committee in making decisions about adaptive changes. CONCLUSION: This article describes our analytic approach for RE-ADAPT. The next stage of the project is to conduct the re-execution analyses using the seven prespecified designs and the original ALLHAT data.
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spelling pubmed-38347352013-11-25 Do Bayesian adaptive trials offer advantages for comparative effectiveness research? Protocol for the RE-ADAPT study Connor, Jason T Luce, Bryan R Broglio, Kristine R Ishak, K Jack Mullins, C Daniel Vanness, David J Fleurence, Rachael Saunders, Elijah Davis, Barry R Clin Trials Design BACKGROUND: Randomized clinical trials, particularly for comparative effectiveness research (CER), are frequently criticized for being overly restrictive or untimely for health-care decision making. PURPOSE: Our prospectively designed REsearch in ADAptive methods for Pragmatic Trials (RE-ADAPT) study is a ‘proof of concept’ to stimulate investment in Bayesian adaptive designs for future CER trials. METHODS: We will assess whether Bayesian adaptive designs offer potential efficiencies in CER by simulating a re-execution of the Antihypertensive and Lipid Lowering Treatment to Prevent Heart Attack Trial (ALLHAT) study using actual data from ALLHAT. RESULTS: We prospectively define seven alternate designs consisting of various combinations of arm dropping, adaptive randomization, and early stopping and describe how these designs will be compared to the original ALLHAT design. We identify the one particular design that would have been executed, which incorporates early stopping and information-based adaptive randomization. LIMITATIONS: While the simulation realistically emulates patient enrollment, interim analyses, and adaptive changes to design, it cannot incorporate key features like the involvement of data monitoring committee in making decisions about adaptive changes. CONCLUSION: This article describes our analytic approach for RE-ADAPT. The next stage of the project is to conduct the re-execution analyses using the seven prespecified designs and the original ALLHAT data. SAGE Publications 2013-10 /pmc/articles/PMC3834735/ /pubmed/23983160 http://dx.doi.org/10.1177/1740774513497293 Text en © The Author(s), 2013 http://creativecommons.org/licenses/by-nc/3.0/ This article is distributed under the terms of the Creative Commons Attribution-NonCommercial 3.0 License (http://www.creativecommons.org/licenses/by-nc/3.0/) which permits non-commercial use, reproduction and distribution of the work without further permission provided the original work is attributed as specified on the SAGE and Open Access page (http://www.uk.sagepub.com/aboutus/openaccess.htm).
spellingShingle Design
Connor, Jason T
Luce, Bryan R
Broglio, Kristine R
Ishak, K Jack
Mullins, C Daniel
Vanness, David J
Fleurence, Rachael
Saunders, Elijah
Davis, Barry R
Do Bayesian adaptive trials offer advantages for comparative effectiveness research? Protocol for the RE-ADAPT study
title Do Bayesian adaptive trials offer advantages for comparative effectiveness research? Protocol for the RE-ADAPT study
title_full Do Bayesian adaptive trials offer advantages for comparative effectiveness research? Protocol for the RE-ADAPT study
title_fullStr Do Bayesian adaptive trials offer advantages for comparative effectiveness research? Protocol for the RE-ADAPT study
title_full_unstemmed Do Bayesian adaptive trials offer advantages for comparative effectiveness research? Protocol for the RE-ADAPT study
title_short Do Bayesian adaptive trials offer advantages for comparative effectiveness research? Protocol for the RE-ADAPT study
title_sort do bayesian adaptive trials offer advantages for comparative effectiveness research? protocol for the re-adapt study
topic Design
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3834735/
https://www.ncbi.nlm.nih.gov/pubmed/23983160
http://dx.doi.org/10.1177/1740774513497293
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