A Bayesian approach to clinical trial designs in dermatology with multiple simultaneous treatments per subject and multiple raters

We consider the statistical analysis of clinical trial designs with multiple simultaneous treatments per subject and multiple raters. The work is motivated by a clinical research project in dermatology where different hair removal techniques were assessed based on a within-subject comparison. We ass...

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Bibliographic Details
Main Authors: Uhlmann, Lorenz (Author) , Stock, Christian (Author) , Vandemeulebroecke, Marc (Author) , Müller-Christmann, Christine (Author) , Kieser, Meinhard (Author)
Format: Article (Journal)
Language:English
Published: August 2023
In: Contemporary clinical trials
Year: 2023, Volume: 131, Pages: 1-8
ISSN:1559-2030
DOI:10.1016/j.cct.2023.107233
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Author Notes:Lorenz Uhlmann, Christian Stock, Marc Vandemeulebroecke, Christine Mueller-Christmann, Meinhard Kieser
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Summary:We consider the statistical analysis of clinical trial designs with multiple simultaneous treatments per subject and multiple raters. The work is motivated by a clinical research project in dermatology where different hair removal techniques were assessed based on a within-subject comparison. We assume that clinical outcomes are assessed by multiple raters as continuous or categorical scores, e.g. based on images, comparing two treatments on the subject-level in a pairwise manner. In this setting, a network of evidence on relative treatment effects is generated, which bears strong similarities to the data underlying a network meta-analysis of clinical trials. We therefore build on established methodology for complex evidence synthesis and propose a Bayesian approach to estimate relative treatment effects and to rank the treatments. The approach is, in principle, applicable to situations with any number of treatment arms and/or raters. As a major advantage, all available data is brought into a network and analyzed in one single model, which ensures consistent results among the treatment comparisons. We obtain operating characteristics via simulation and illustrate the method with a real clinical trial example.
Item Description:Gesehen am 23.01.2024
Physical Description:Online Resource
ISSN:1559-2030
DOI:10.1016/j.cct.2023.107233