How to plan early, overcome methodological challenges, and stay ahead in the evolving European access environment.

The European Union’s Joint Clinical Assessment (JCA) is transforming how new therapies are evaluated for patient access across Europe. For orphan and rare disease medicines, these changes bring both unprecedented opportunity and significant methodological risk.

This new RTI Health Solutions report, Mastering EU JCAs for Orphan Drugs, explores how health technology developers can navigate stringent evidence requirements, manage small data sets, and embed JCA requirements into evidence generation strategies to meet the rigorous expectations of JCA assessors.