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A novel equivalence probability weighted power prior for using historical control data in an adaptive clinical trial design: A comparison to standard methods

A standard two‐arm randomised controlled trial usually compares an intervention to a control treatment with equal numbers of patients randomised to each treatment arm and only data from within the current trial are used to assess the treatment effect. Historical data are used when designing new tria...

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Autores principales: Bennett, Maxine, White, Simon, Best, Nicky, Mander, Adrian
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8611797/
https://www.ncbi.nlm.nih.gov/pubmed/33474798
http://dx.doi.org/10.1002/pst.2088
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author Bennett, Maxine
White, Simon
Best, Nicky
Mander, Adrian
author_facet Bennett, Maxine
White, Simon
Best, Nicky
Mander, Adrian
author_sort Bennett, Maxine
collection PubMed
description A standard two‐arm randomised controlled trial usually compares an intervention to a control treatment with equal numbers of patients randomised to each treatment arm and only data from within the current trial are used to assess the treatment effect. Historical data are used when designing new trials and have recently been considered for use in the analysis when the required number of patients under a standard trial design cannot be achieved. Incorporating historical control data could lead to more efficient trials, reducing the number of controls required in the current study when the historical and current control data agree. However, when the data are inconsistent, there is potential for biased treatment effect estimates, inflated type I error and reduced power. We introduce two novel approaches for binary data which discount historical data based on the agreement with the current trial controls, an equivalence approach and an approach based on tail area probabilities. An adaptive design is used where the allocation ratio is adapted at the interim analysis, randomising fewer patients to control when there is agreement. The historical data are down‐weighted in the analysis using the power prior approach with a fixed power. We compare operating characteristics of the proposed design to historical data methods in the literature: the modified power prior; commensurate prior; and robust mixture prior. The equivalence probability weight approach is intuitive and the operating characteristics can be calculated exactly. Furthermore, the equivalence bounds can be chosen to control the maximum possible inflation in type I error.
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spelling pubmed-86117972021-11-29 A novel equivalence probability weighted power prior for using historical control data in an adaptive clinical trial design: A comparison to standard methods Bennett, Maxine White, Simon Best, Nicky Mander, Adrian Pharm Stat Main Papers A standard two‐arm randomised controlled trial usually compares an intervention to a control treatment with equal numbers of patients randomised to each treatment arm and only data from within the current trial are used to assess the treatment effect. Historical data are used when designing new trials and have recently been considered for use in the analysis when the required number of patients under a standard trial design cannot be achieved. Incorporating historical control data could lead to more efficient trials, reducing the number of controls required in the current study when the historical and current control data agree. However, when the data are inconsistent, there is potential for biased treatment effect estimates, inflated type I error and reduced power. We introduce two novel approaches for binary data which discount historical data based on the agreement with the current trial controls, an equivalence approach and an approach based on tail area probabilities. An adaptive design is used where the allocation ratio is adapted at the interim analysis, randomising fewer patients to control when there is agreement. The historical data are down‐weighted in the analysis using the power prior approach with a fixed power. We compare operating characteristics of the proposed design to historical data methods in the literature: the modified power prior; commensurate prior; and robust mixture prior. The equivalence probability weight approach is intuitive and the operating characteristics can be calculated exactly. Furthermore, the equivalence bounds can be chosen to control the maximum possible inflation in type I error. John Wiley & Sons, Inc. 2021-01-20 2021 /pmc/articles/PMC8611797/ /pubmed/33474798 http://dx.doi.org/10.1002/pst.2088 Text en © 2021 The Authors. Pharmaceutical Statistics published by John Wiley & Sons Ltd https://creativecommons.org/licenses/by/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.
spellingShingle Main Papers
Bennett, Maxine
White, Simon
Best, Nicky
Mander, Adrian
A novel equivalence probability weighted power prior for using historical control data in an adaptive clinical trial design: A comparison to standard methods
title A novel equivalence probability weighted power prior for using historical control data in an adaptive clinical trial design: A comparison to standard methods
title_full A novel equivalence probability weighted power prior for using historical control data in an adaptive clinical trial design: A comparison to standard methods
title_fullStr A novel equivalence probability weighted power prior for using historical control data in an adaptive clinical trial design: A comparison to standard methods
title_full_unstemmed A novel equivalence probability weighted power prior for using historical control data in an adaptive clinical trial design: A comparison to standard methods
title_short A novel equivalence probability weighted power prior for using historical control data in an adaptive clinical trial design: A comparison to standard methods
title_sort novel equivalence probability weighted power prior for using historical control data in an adaptive clinical trial design: a comparison to standard methods
topic Main Papers
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8611797/
https://www.ncbi.nlm.nih.gov/pubmed/33474798
http://dx.doi.org/10.1002/pst.2088
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