Details

Autor(en) / Beteiligte

• Titievsky, Lina
• Volkova, Nataliya
• Yijie, Zhou
• Yang, Song
• Bo, Yang
• Yang, Bo
• Song, Yang
• Zhou, Yijie

Titel

Use of Real-World Evidence to Support Drug Development

Ist Teil von

• Drug Development for Rare Diseases, 2023, p.101-115

Auflage

1

Ort / Verlag

United Kingdom: CRC Press

Erscheinungsjahr

2023

Quelle

Alma/SFX Local Collection

Beschreibungen/Notizen

• 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. 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. Point prevalence plays a critical role in the current orphan drug legislation where definition of rare diseases includes a prevalence-based threshold. Obtaining prevalence estimates for rare diseases from published literature and/or quantifying prevalence estimates from available data sources is often challenging. Clinical trial data package at the time or regulatory approval may be limited, including small patient population, short follow-up, approval obtained based on a surrogate endpoint, and in some cases absence of control arm.