Addressing challenges to European multi-country collaboration models for rare diseases

Study 23-05-2024

Rare diseases – defined as diseases that affect no more than one person in 2 000 – currently impact between 30 and 35 million EU citizens. Limited patient volume hinders research on these diseases, to the detriment of understanding of their biology, recruitment to clinical trials and survival rates. Real-time sharing of primary health data within and between rare disease healthcare centres and across Member States is feasible but there are obstacles. This report focuses on childhood cancer as a rare disease prototype, addressing volume challenges by reviewing cooperative structures, exploring attitudes towards data sharing, and presenting policy options for multi-country collaboration. Interviews show support for collaboration among rare disease healthcare centres, and for sharing medical files and expertise. The policy options proposed seek to promote research and expertise-building for the benefit of rare disease patients.