Executive Summary
Rare disease assessments rely on specialist clinical and patient expertise to interpret complex evidence, heterogeneous patient populations and uncertain disease progression. However, under the Commission Implementing Regulation (EU) 2024/2745, the conflict-of-interest (CoI) framework for EU Joint Clinical Assessments (JCAs) may unintentionally restrict access to the very experts best placed to evaluate orphan medicinal products (OMPs). Given the limited pool of rare disease specialists, participation restrictions risk excluding essential expertise from JCA processes, while the application of the Regulation’s “exceptional cases” provision remains insufficiently defined, creating uncertainty for experts and health technology developers.
This paper compares the EU JCA CoI framework with approaches adopted across nine European HTA systems, highlighting how many Member States manage CoI through proportionate, risk-based measures rather than default exclusion. Drawing on these national practices, the paper identifies opportunities to strengthen the EU approach through clearer operational criteria for exceptional cases, managed participation mechanisms for indispensable experts, and a more pragmatic balance between impartiality, transparency and access to specialist expertise. Collectively, these measures could help ensure that rare disease assessments continue to benefit from the high-quality clinical and patient insight needed to support robust, credible and equitable HTA decision-making across the EU.
This publication was sponsored by Alexion, AstraZeneca Rare Disease. The analysis and conclusions were developed independently by Remap Consulting.