Approaches for Optimal Dose Selection for Adaptive Design Trials

Abstract

Adaptive designs use accumulating data to modify in a prospectively planned manner certain design aspects of a clinical study without undermining its validity and integrity. The aim of this chapter is to review adaptive design approaches for dose finding and optimal dose selection and to demonstrate that adaptivity is a fundamentally important concept, which can be applied to dose selection in different stages of clinical development. We review the major statistical methods available for planning and analyzing adaptive designs in Phase I, II, and III. To illustrate the ideas, we refer to examples and case studies from the literature, where available.