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.