Power Determination During Drug Development: Is Optimizing the Sample Size Based on Exposure‐Response Analyses Underutilized?

Abstract

The use of model‐based drug development (MBDD) has been demonstrated to improve the efficiency of clinical trial design. However, MBDD complexity can limit its use, particularly early in clinical development. In this tutorial, a simple and generalizable exposure‐response analysis approach to determine the power for dose‐ranging studies is presented and described. We identified situations in which higher power and sample size reduction is achieved by utilizing the exposure‐response powering methodology compared with conventional power calculations.