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Comparison of Bayesian methods for incorporating adult clinical trial data to improve certainty of treatment effect estimates in children

There are challenges associated with recruiting children to take part in randomised clinical trials and as a result, compared to adults, in many disease areas we are less certain about which treatments are most safe and effective. This can lead to weaker recommendations about which treatments to pre...

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Detalles Bibliográficos
Autores principales: Walker, Ruth, Phillips, Bob, Dias, Sofia
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10270354/
https://www.ncbi.nlm.nih.gov/pubmed/37319173
http://dx.doi.org/10.1371/journal.pone.0281791
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author Walker, Ruth
Phillips, Bob
Dias, Sofia
author_facet Walker, Ruth
Phillips, Bob
Dias, Sofia
author_sort Walker, Ruth
collection PubMed
description There are challenges associated with recruiting children to take part in randomised clinical trials and as a result, compared to adults, in many disease areas we are less certain about which treatments are most safe and effective. This can lead to weaker recommendations about which treatments to prescribe in practice. However, it may be possible to ‘borrow strength’ from adult evidence to improve our understanding of which treatments work best in children, and many different statistical methods are available to conduct these analyses. In this paper we discuss four Bayesian methods for extrapolating adult clinical trial evidence to children. Using an exemplar dataset, we compare the effect of their modelling assumptions on the estimated treatment effect and associated heterogeneity. These modelling assumptions range from adult evidence being completely generalisable to being completely unrelated to the children’s evidence. We finally discuss the appropriateness of these modelling assumptions in the context of estimating treatment effect in children.
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spelling pubmed-102703542023-06-16 Comparison of Bayesian methods for incorporating adult clinical trial data to improve certainty of treatment effect estimates in children Walker, Ruth Phillips, Bob Dias, Sofia PLoS One Research Article There are challenges associated with recruiting children to take part in randomised clinical trials and as a result, compared to adults, in many disease areas we are less certain about which treatments are most safe and effective. This can lead to weaker recommendations about which treatments to prescribe in practice. However, it may be possible to ‘borrow strength’ from adult evidence to improve our understanding of which treatments work best in children, and many different statistical methods are available to conduct these analyses. In this paper we discuss four Bayesian methods for extrapolating adult clinical trial evidence to children. Using an exemplar dataset, we compare the effect of their modelling assumptions on the estimated treatment effect and associated heterogeneity. These modelling assumptions range from adult evidence being completely generalisable to being completely unrelated to the children’s evidence. We finally discuss the appropriateness of these modelling assumptions in the context of estimating treatment effect in children. Public Library of Science 2023-06-15 /pmc/articles/PMC10270354/ /pubmed/37319173 http://dx.doi.org/10.1371/journal.pone.0281791 Text en © 2023 Walker et al https://creativecommons.org/licenses/by/4.0/This is an open access article distributed under the terms of the Creative Commons Attribution License (https://creativecommons.org/licenses/by/4.0/) , which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.
spellingShingle Research Article
Walker, Ruth
Phillips, Bob
Dias, Sofia
Comparison of Bayesian methods for incorporating adult clinical trial data to improve certainty of treatment effect estimates in children
title Comparison of Bayesian methods for incorporating adult clinical trial data to improve certainty of treatment effect estimates in children
title_full Comparison of Bayesian methods for incorporating adult clinical trial data to improve certainty of treatment effect estimates in children
title_fullStr Comparison of Bayesian methods for incorporating adult clinical trial data to improve certainty of treatment effect estimates in children
title_full_unstemmed Comparison of Bayesian methods for incorporating adult clinical trial data to improve certainty of treatment effect estimates in children
title_short Comparison of Bayesian methods for incorporating adult clinical trial data to improve certainty of treatment effect estimates in children
title_sort comparison of bayesian methods for incorporating adult clinical trial data to improve certainty of treatment effect estimates in children
topic Research Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10270354/
https://www.ncbi.nlm.nih.gov/pubmed/37319173
http://dx.doi.org/10.1371/journal.pone.0281791
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