EUCOPE on First Joint Clinical Assessment (JCA) Report: Critical Milestone – Readiness for Orphan Drug Assessments Needed

Brussels 9 June 2026

The European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) takes note of today’s publication of the first Joint Clinical Assessment (JCA) report.

This marks a critical milestone in the implementation of the Regulation (EU) 2021/2282 on health technology assessment. Over the coming 18 months, full attention must be directed towards ensuring the readiness of EU HTA for Orphan Medicinal Products (OMPs), that will become subject to JCA from 13 January 2028.

EUCOPE has welcomed the creation of the framework for joint assessments of innovative pharmaceuticals by Member States at the EU level, which was established with the overall aim to reduce the burdens for both HTA bodies and companies, caused by parallel requests for submissions of evidence and assessment across the 27 EU Member States.

As a key stakeholder representing the small and mid-sized innovative companies, we support our member companies with navigating the new procedures and the implementation process, as a member of the HTA Stakeholder Network.

Alexander Natz, Secretary General (EUCOPE): “We congratulate the Member States HTA Coordination Group and HTA Secretariat of the European Commission on the publication of the first JCA report —an important milestone has been achieved, and we hope full attention is now directed towards the readiness of EU HTA for orphan medicinal products”

The publication of the first JCA report demonstrates the important progress that has been made in collaboration on HTA at EU level: it is developed on the basis of common procedures, with the submission of one single dossier from the company. However, significant steps still remain to achieve harmonisation between Member States’ differing requirements and reducing the market access burdens for companies launching innovative medicinal products in Europe.

We in particular note that the overall number of PICOs that companies are requested to submit evidence and analyses for remains too high. This has critical implications for the workability of the procedure, especially for small and mid-sized companies with limited resources and smaller market access teams and with less prior exposure to the European market.

To work towards a lower set of relevant PICOs for the assessment, and increase the predictability for companies, the underutilised opportunities that exist for interaction between companies and the assessors in JCA must be leveraged.

Additionally, the specificities of OMPs must be recognised in the EU HTA guidance documents, as foreseen in Regulation (EU) 2021/2282 on health technology assessment.

A standard randomised controlled trial (RCT) is not always possible for treatments for rare (and ultra rare) diseases, due to the small patient populations and serious nature of the disease. Such exceptional circumstances are already recognised by the European Medicines Agency. To provide a complete picture for national access decision, the JCA report must also reflect the specificities of the disease context.

The success of the EU HTA procedure remains to be seen, based on the use of the JCA reports for national decision-making to inform national HTA procedures and access decisions. EUCOPE remains committed to supporting the implementation of the new procedure, to improve workability for small and mid-sized companies, launching innovative medicinal products in Europe.