Beyond exposure-response: A tutorial on statistical considerations in dose-ranging studies

Abstract

Dose-ranging studies are a crucial part of the phase II drug development process. They complement the understanding gained from exposure-response analyses. However, the statistical design issues related to dose-ranging studies are not always keenly understood and a poorly designed study can be costly for later development. In this tutorial, we review five key statistical principles in designing such a study. We also describe some popular statistical approaches, including pairwise comparison, modeling, and Multiple Comparison Procedure modeling in the context of principles.