A Dolon Institute presentation
The introduction of Joint Clinical Assessment (JCA) marks a major change in how medicines will be clinically assessed across Europe. Designed to reduce duplication and create greater consistency between Member States, JCA has the potential to streamline health technology assessment (HTA) and support faster patient access. But behind the promise of harmonisation lies a more complex reality, particularly for rare diseases and other areas where evidence is inherently limited.
Pulling back the curtain on Joint Clinical Assessment was presented at World Orphan Drug Congress (WODC) Europe, and takes a practical look at what JCA means for manufacturers, exploring what is now understood about the process, where important uncertainties remain, and what companies need to do differently to prepare. The presentation highlights that while elements of the EU-level process are becoming clearer, significant questions remain around PICO selection and consolidation, evidence requirements, patient and clinical expert involvement, and how Member States will ultimately use JCA outputs.
A particular focus is the challenge of PICOs and evidence requirements. The JCA framework places strong emphasis on randomised controlled trial evidence, while single-arm trials and real-world evidence may carry less weight. This creates potential difficulties for rare diseases, where small populations, limited disease knowledge and the absence of conventional comparators can make traditional evidence generation particularly challenging. The presentation also highlights the risk of PICO proliferation and the fact that manufacturers have limited involvement in the scoping process.
The implications will not stop at the European level. Member States are required to give “due consideration” to JCA outputs, but retain flexibility in how they incorporate them into national HTA and pricing and reimbursement processes. This means the impact on access, timelines, efficiency, predictability and pricing could vary considerably between countries.
For manufacturers, the presentation makes a clear case for getting ahead of JCA rather than treating it as an additional submission requirement. Companies should anticipate potential PICO requests, rethink pre-launch timelines, strengthen statistical and HEOR capabilities, consider JCA evidence requirements early and engage stakeholders wherever possible.
Ultimately, JCA could deliver a more efficient and consistent European HTA system — but this outcome is not guaranteed. Its success will depend on how effectively the new process accommodates the realities of different diseases, particularly rare diseases, and how Member States translate the joint assessment into national decisions.