This paper discusses disadvantages and limitations of the available inferential approaches in sequential clinical trials for treatment comparisons managed via response-adaptive randomization. Then, we propose an inferential methodology for response-adaptive designs which, by exploiting a variance stabilizing transformation into a bootstrap framework, is able to overcome the above-mentioned drawbacks, regardless of the chosen allocation procedure aswell as the desired target.We derive the theoretical properties of the suggested proposal, showing its superiority with respect to likelihood, randomization and design-based inferential approaches. Several illustrative examples and simulation studies are provided in order to confirm the relevance of our results.

A new inferential approach for response-adaptive clinical trials: the variance-stabilized bootstrap

Baldi Antognini, Alessandro
;
Novelli, Marco;Zagoraiou, Maroussa
2022

Abstract

This paper discusses disadvantages and limitations of the available inferential approaches in sequential clinical trials for treatment comparisons managed via response-adaptive randomization. Then, we propose an inferential methodology for response-adaptive designs which, by exploiting a variance stabilizing transformation into a bootstrap framework, is able to overcome the above-mentioned drawbacks, regardless of the chosen allocation procedure aswell as the desired target.We derive the theoretical properties of the suggested proposal, showing its superiority with respect to likelihood, randomization and design-based inferential approaches. Several illustrative examples and simulation studies are provided in order to confirm the relevance of our results.
Baldi Antognini, Alessandro; Novelli, Marco; Zagoraiou, Maroussa
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11585/820917
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