On 22 June 2026, a milestone arrived quietly but with significant implications for people living with rare diseases across Europe: the publication of the first Joint Clinical Assessment (JCA) under the EU HTA Regulation.

The webinar marking this moment brought together patient organisations, medical societies, industry and health technology assessment (HTA) bodies, organised by the Alliance for Regenerative Medicine, Cancer Patients Europe, Fondazione Telethon and the European Haematology Association. What emerged from the discussion matters deeply for the CF community.

A new chapter in how medicines are evaluated in Europe

Until now, each EU Member State ran its own clinical assessment of new medicines before making reimbursement decisions. The new HTA Regulation changes this: going forward, a single European clinical evaluation will be produced, on which national authorities can rely when deciding on pricing and reimbursement. The publication of the first JCA is the beginning of that shift in practice.

Rare diseases need a different yardstick

One of the clearest messages from the webinar was also one of the most familiar to our community: rare disease research does not look like mainstream clinical research. Small patient populations, limited comparator data, and single-arm studies are not shortcomings — they are the reality of developing medicines for conditions like CF. Participants stressed firmly that HTA methodologies must be flexible enough to reflect this, rather than applying expectations designed for large-scale trials.

Closing the gap between regulators and HTA bodies

A recurring concern was the disconnect that can arise between regulatory approval and HTA assessment. Companies design clinical development programmes years in advance, often guided by EMA scientific advice. Yet a positive opinion from regulators does not guarantee smooth passage through the JCA, because HTA bodies may have different expectations around comparative evidence. Participants called for much closer alignment between these two systems, so that the evidence generated serves both — and so that patients are not caught in the middle.

Patients at the table, not just in the waiting room

The webinar also examined the role of patients and clinicians in the JCA process. Stakeholders welcomed their involvement in this first assessment, but were clear that consultation alone is not enough. Contributors deserve to understand how their input shaped the final outcome. And with JCAs potentially influencing access across 27 countries, summaries need to be accessible, patient-friendly and available in multiple European languages.

What this means for CF

For the CF community, the success of the EU HTA Regulation will not be measured in technical processes — it will be measured in whether people with CF across Europe, including those with rare mutations and smaller patient groups, can access innovative therapies faster and more equitably. The first JCA is a learning opportunity. The CF community has an important role to play in making sure those lessons lead to a system that works for everyone.