ABSTRACT
An important step in drug development is to identify the dose or dose range of a compound that offers adequate efficacy with minimal safety risk. Phase II often fulfills a critical step in learning about the dose-response relationship in the patient population to be treated. The main objective of phase II dose-finding trials is to identify dosing procedures and target patient populations for further clinical development. The hope is to identify one or two doses with the appropriate marketable characteristics that can be confirmed in phase III of the development program, or to stop development. Therefore, the design of the phase II dosefinding trials should be optimized for the trial’s ability to quantify the key features of the dose-response relationship and the trial’s ability to determine the “right” dose(s), if any, for further development.
