Randomized test-treatment studies with an outlook on adaptive designs

Hot, Amra and Bossuyt, Patrick M. and Gerke, Oke and Wahl, Simone and Vach, Werner and Zapf, Antonia. (2021) Randomized test-treatment studies with an outlook on adaptive designs. BMC Medical Research Methodology, 21 (1). p. 110.

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Official URL: https://edoc.unibas.ch/84160/

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Diagnostic accuracy studies aim to examine the diagnostic accuracy of a new experimental test, but do not address the actual merit of the resulting diagnostic information to a patient in clinical practice. In order to assess the impact of diagnostic information on subsequent treatment strategies regarding patient-relevant outcomes, randomized test-treatment studies were introduced. Various designs for randomized test-treatment studies, including an evaluation of biomarkers as part of randomized biomarker-guided treatment studies, are suggested in the literature, but the nomenclature is not consistent.
The aim was to provide a clear description of the different study designs within a pre-specified framework, considering their underlying assumptions, advantages as well as limitations and derivation of effect sizes required for sample size calculations. Furthermore, an outlook on adaptive designs within randomized test-treatment studies is given.
The need to integrate adaptive design procedures in randomized test-treatment studies is apparent. The derivation of effect sizes induces that sample size calculation will always be based on rather vague assumptions resulting in over- or underpowered study results. Therefore, it might be advantageous to conduct a sample size re-estimation based on a nuisance parameter during the ongoing trial.
Due to their increased complexity, compared to common treatment trials, the implementation of randomized test-treatment studies poses practical challenges including a huge uncertainty regarding study parameters like the expected outcome in specific subgroups or disease prevalence which might affect the sample size calculation. Since research on adaptive designs within randomized test-treatment studies is limited so far, further research is recommended.
Faculties and Departments:05 Faculty of Science > Departement Umweltwissenschaften > Integrative Biologie > Integrative Prähistorische und Naturwissenschaftliche Archäologie (IPNA Schünemann)
UniBasel Contributors:Vach, Werner
Item Type:Article, refereed
Article Subtype:Research Article
Publisher:BioMed Central
Note:Publication type according to Uni Basel Research Database: Journal article
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Last Modified:06 Sep 2021 09:49
Deposited On:06 Sep 2021 09:49

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