Use of Real-World Evidence to Support Drug Development

Abstract

Real-world evidence (RWE) generated using real-world data (RWD) is an integral component of drug development. This chapter discusses various uses of RWE across different stages of drug development in support of rare disease therapies including but not limited to the target patient identification, signal contextualization, studies to support long-term safety, and effectiveness as well as the label expansion. The chapter also provides an overview of potential RWD sources that researchers can consider in studying rare diseases and discusses practicalities and challenges in generating the right type of RWE at each key stage of rare disease drug development.