Executive Summary 

There are 30 million people in Europe living with rare diseases, many of whom have unmet medical needs. Creating new therapies for rare diseases is challenging due to large knowledge gaps in our understanding of the disease and small population sizes, which can make learning about the disease and designing a clinical trial difficult.

Healthcare data collected in the “real-world” (Real-World Data, RWD) could be used to generate reliable evidence (Real-World Evidence, RWE) to support better decision-making and develop new therapies for rare diseases.

This whitepaper:

- Defines the opportunity: It examines how RWD and RWE can supplement clinical trial data for drug approval applications, enable a better understanding of the disease itself, expand the on-label indications for existing products, and more.

- Explores how to execute on the opportunity: It looks at the multiple challenges – developing infrastructure, GDPR, etc. – that must be addressed, and identifies key success factors.

- Examines the way forward: It discusses how, in the future, RWE could be used to demonstrate the value or effectiveness of new medicines as part of the approval dossier.