From Adaptive Design to Modern Protocol Design for Drug Development: Part I. Editorial and Summary of Adaptive Designs Session at the Third FDA/DIA Statistics Forum

Abstract

The Adaptive Designs session at the Third FDA/DIA Statistics Forum presented an important step in the scientific merits and evaluation of two recently emerging drug development adaptive paradigms: exploratory versus confirmatory. After highlighting the different paradigms, several questions addressing critical issues in confirmatory clinical trials were posed and discussed by an expert panel. So far, adaptive design and analysis methods have focused mostly on individual trials. The merits of a single adaptive trial may be better suited to the context of a drug development program. We articulate the roles of modeling and simulation for modem protocol design via clinical scenario planning in a phase 3 development program. The clinical scenarios consist of a combination of effect sizes, variability, placebo response, and type I and type II errors. The models are mainly used to improve the precision of effect estimate. These topics are further discussed in Part II.