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Utility-based dose selection for phase II dose-finding studies

Abstract : Dose selection is a key feature of clinical development. Poor dose selection has been recognized as a major driver of development failure in late phase. It usually involves both efficacy and safety criteria. The objective of this paper is to develop and implement a novel fully Bayesian statistical framework to optimize the dose selection process by maximizing the expected utility in phase III. The success probability is characterized by means of a utility function with two components, one for efficacy and one for safety. Each component refers to a dose-response model. Moreover, a sequential design (with futility and efficacy rules at the interim analysis) is compared to a fixed design in order to allow one to hasten the decision to perform the late phase study. Operating characteristics of this approach are extensively assessed by simulations under a wide range of dose-response scenarios.
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https://hal.inria.fr/hal-02491551
Contributor : Gwladys Toulemonde <>
Submitted on : Wednesday, February 26, 2020 - 11:21:13 AM
Last modification on : Thursday, February 27, 2020 - 1:55:23 AM
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  • HAL Id : hal-02491551, version 1

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Jihane Aouni, Jean Bacro, Gwladys Toulemonde, Pierre Colin, Loic Darchy. Utility-based dose selection for phase II dose-finding studies. 2020. ⟨hal-02491551⟩

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