DC10: Statistical methodologies to evaluate efficacy of tailored single-patient treatments

Daria Zhuravlevla, Uppsala Universitet (supervisors: Andrew Hooker, Mats Karlsson)

Objectives: to design methodology and models that allow treatment efficacy to be determined based on a single or multiple endpoint(s) followed over time and relevant prior information; to design a framework of linked endpoint models and prior information that allows optimization of trial designs for single subject quasi-experiments of natural history followed by treatment; to illustrate the relative merits of different designs using optimal experimental design strategies and clinical trial simulations.