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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...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Public Library of Science
2023
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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. |
format | Online Article Text |
id | pubmed-10270354 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2023 |
publisher | Public Library of Science |
record_format | MEDLINE/PubMed |
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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