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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Main Authors: Ruth Walker, Bob Phillips, Sofia Dias
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2023-01-01
Series:PLoS ONE
Online Access:https://doi.org/10.1371/journal.pone.0281791
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author Ruth Walker
Bob Phillips
Sofia Dias
author_facet Ruth Walker
Bob Phillips
Sofia Dias
author_sort Ruth Walker
collection DOAJ
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 doaj-art-172dd337bb0845ad9b5b6ea49cc55ea92025-08-20T03:25:20ZengPublic Library of Science (PLoS)PLoS ONE1932-62032023-01-01186e028179110.1371/journal.pone.0281791Comparison of Bayesian methods for incorporating adult clinical trial data to improve certainty of treatment effect estimates in children.Ruth WalkerBob PhillipsSofia DiasThere 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.https://doi.org/10.1371/journal.pone.0281791
spellingShingle Ruth Walker
Bob Phillips
Sofia Dias
Comparison of Bayesian methods for incorporating adult clinical trial data to improve certainty of treatment effect estimates in children.
PLoS ONE
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
url https://doi.org/10.1371/journal.pone.0281791
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AT bobphillips comparisonofbayesianmethodsforincorporatingadultclinicaltrialdatatoimprovecertaintyoftreatmenteffectestimatesinchildren
AT sofiadias comparisonofbayesianmethodsforincorporatingadultclinicaltrialdatatoimprovecertaintyoftreatmenteffectestimatesinchildren