Eurordis - Rare Disease Europe

We are grateful for the focus the Draghi report places on healthcare and innovation, which are of critical importance to the rare disease community. With over 30 million people in Europe impacted, treatments exist for less than 6% of known conditions, and patients face an average five-year wait for a diagnosis. It is vital for the EU to not only strengthen but also revitalise Europe’s healthcare, medical technology, and pharmaceutical sectors. We hope that the recommendations in this report could lay the foundations for a stronger, more resilient health policy framework – one that fosters innovation, ensures equity and guarantees long-term sustainability in meeting the needs of rare disease patients.

We are pleased to see the report highlight joint procurement and the need to step up cross-country initiatives for joint pricing and reimbursement negotiations for specific medicines, both of which represent important steps toward ensuring rare disease patients have coordinated, cost-effective access to the treatments they urgently need. We also fully support the report’s vision of positioning Europe as a global leader in innovation, fostering a robust environment for research and development. This must include bolstering the rare disease ecosystem and critical infrastructures like the European Reference Networks, which connect scarce expertise across borders.

Additionally, the report’s emphasis on strengthening the European Health Data Space (EHDS), promoting multi-country clinical trials, and enhancing the use of artificial intelligence in healthcare presents a real opportunity to accelerate the development of orphan medicines. These measures, alongside increased investment in research and innovation, particularly in collaboration with the European Reference Networks (ERNs), could transform the landscape for rare disease patients across Europe, providing them with faster access to diagnosis and new therapies.

As the new European Commission takes office, we strongly urge policymakers to keep health and research, particularly for rare diseases, at the forefront of their agenda. While we are concerned about proposed cuts to the EU4Health programme and the lack of updates on future health strategies, this report presents a timely opportunity to address the challenges faced by our community and foster collaboration among Member States, ensuring that no patient is left behind.