A new inferential approach for response-adaptive clinical trials: the variance-stabilized bootstrap
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Alessandro Baldi Antognini [1] ; Marco Novelli [1] ; Maroussa Zagoraiou [1]
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[1] University of Bologna
University of Bologna
Bolonia, Italia
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- Localización: Test: An Official Journal of the Spanish Society of Statistics and Operations Research, ISSN-e 1863-8260, ISSN 1133-0686, Vol. 31, Nº. 1, 2022, págs. 235-254
- Idioma: inglés
- Texto completo no disponible (Saber más ...)
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Resumen
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 as well 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.
