Joint Clinical Assessment: The finger on the scale for orphan sustainability in Europe?
Emilie Neez, Elisa Garau, Adam Hutchings
January 2025
EU Joint Clinical Assessment (JCA) has the potential to transform the evaluation of medicines in Europe, including orphan drugs. While there’s hope that JCAs will streamline fragmented processes and improve patient access, the road ahead is far from simple.
Concerns include the complexity of implementation at regional and country level, and the lack of adaptation of the assessment of evidence for rare diseases as the European market for orphan medicines becomes increasingly constrained, the implications for orphan sustainability are vast. In this report, we tackle two critical questions:
1. What will JCA look like for orphan medicines?
2. How might JCA impact the sustainability of orphan innovation and access in Europe?
