Rare Focus is a brief update designed to help people working in rare diseases keep track of important developments in Europe.
EU Joint Clinical Assessment is still early in its implementation - and the signals that matter for rare disease programmes are scattered across multiple sources: HTA Coordination Group outputs, EMA guidance, national body positions, and emerging precedent from the first oncology and ATMP assessments.
For rare disease teams, mandatory JCA scope does not begin until 2028. But the window for useful preparation is now and staying informed about how the process is developing is one of the most practical things teams can do in the meantime.
RAREfocus tracks what matters and distils it for the people who need it most.
Each edition covers a small number of developments with practical implications for rare disease asset teams not a summary of everything that is happening, but a considered view of what is worth paying attention to and why.
- what EU JCA and national HTA bodies are signalling
- how expectations for patient input are evolving
- areas where teams may need to prepare earlier
- changes that could influence evidence planning or coordination
The aim is to give teams a clear, straightforward summary they can read in a few minutes, without needing to follow multiple sources.
Rare Focus is relevant for clinical teams, patient-engagement leads, HEOR, market access, medical affairs and anyone supporting rare-disease programmes across Europe or from the US.
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