The aim of the present paper is to provide optimal allocations for comparative clinical trials with survival outcomes. The suggested targets are derived adopting a compound optimization strategy based on a subjective weighting of the relative importance of inferential demands and ethical concerns. The ensuing compound optimal targets are continuous functions of the treatment effects, so we provide the conditions under which they can be approached by standard response-adaptive randomization procedures, also guaranteeing the applicability of the classical asymptotic inference. The operating characteristics of the suggested methodology are verified both theoretically and by simulation, including the robustness to model misspecification.With respect to the other available proposals, our strategy always assigns more patients to the best treatment without compromising inference, taking into account estimation efficiency and power as well.We illustrate our procedure by redesigning two real oncological trials.

Compound optimal allocations for survival clinical trials

Baldi Antognini, Alessandro
;
Novelli, Marco;Zagoraiou, Maroussa;Vagheggini, Alessandro
2020

Abstract

The aim of the present paper is to provide optimal allocations for comparative clinical trials with survival outcomes. The suggested targets are derived adopting a compound optimization strategy based on a subjective weighting of the relative importance of inferential demands and ethical concerns. The ensuing compound optimal targets are continuous functions of the treatment effects, so we provide the conditions under which they can be approached by standard response-adaptive randomization procedures, also guaranteeing the applicability of the classical asymptotic inference. The operating characteristics of the suggested methodology are verified both theoretically and by simulation, including the robustness to model misspecification.With respect to the other available proposals, our strategy always assigns more patients to the best treatment without compromising inference, taking into account estimation efficiency and power as well.We illustrate our procedure by redesigning two real oncological trials.
Baldi Antognini, Alessandro; Novelli, Marco; Zagoraiou, Maroussa; Vagheggini, Alessandro
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11585/765822
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