Eurordis - Rare Disease Europe

We welcome the European Parliament’s confirmation of Oliver Várhelyi as the new Health Commissioner, as the priorities that he had outlined during his confirmation hearing earlier this month should provide solutions to some of the key unmet needs of people living with rare diseases. His stated commitment to strengthening Europe’s health systems, particularly by fostering resilience and innovation in the pharmaceutical and medical device sectors, reflects a clear understanding of the challenges ahead. His expressed plans to propose the Critical Medicines Act and the EU Biotech Act, advance the European Health Data Space, and explore joint procurement for orphan medicinal products were all highlighted as his key ambitions during his hearing and would be vital steps toward addressing the unique challenges faced by rare disease patients across Europe.

Commissioner Várhelyi’s focus on drug shortages, which he specifically addressed during the hearing, will be particularly important for the rare disease community, who are often disproportionately impacted by such shortages. Furthermore, his stated prioritisation of increasing the accessibility and availability of orphan and paediatric medical devices signals a much-needed response to the needs of patients and their families across Europe – this is important given the reality that one in three people with rare diseases has never received a treatment directly linked to their rare disease, because it does not exist, and 94% of rare diseases don’t have a specific treatment.

We also welcome Commissioner Várhelyi’s strong emphasis on enhancing the European Reference Networks (ERNs), which he has aptly described as a ‘major success,’ along with his commitment to reducing barriers in cross-border healthcare. Given the limited knowledge and medical expertise on rare and complex diseases that often exist at the national level, fostering cross-border collaboration and pooling resources internationally is both logical and . We trust this support will be maintained when the EC will outline the long-term budget commitments of the EU.

Finally, we are hopeful that Commissioner Várhelyi will advance a European Action Plan for Rare Diseases. This initiative has been endorsed by the European Parliament, the European Economic and Social Committee, and last June, the EU’s 27 health ministers in their Council Conclusion on future of the European Health Union. A goals-based Action Plan is urgently needed and long overdue, offering the most comprehensive strategy to address the unmet needs of Europe’s rare disease community.

During her confirmation hearing, Zaharieva was right to state her commitment to securing robust funding for the Horizon Europe programme, amid the threat of cuts to the programme. We also welcome the focus that she promised on simplifying processes for startups and SMEs, which would be critical steps toward fostering innovation in healthcare and medicine that can benefit the rare disease community. Her comments on retaining research talent within the EU and encouraging member state investment in research will have been encouraging to those from our rare disease community who want to see the creation of a stronger foundation for advancing diagnostics and therapies for rare diseases.

We look forward to working closely with Commissioner Zaharieva to ensure that the unique needs of the rare disease community are prioritised in Europe’s research agenda.

The majority of people with rare diseases live with disabilities and face significant barriers to accessing their health, social, and independent living rights on an equal basis with others.

Commissioner Lahbib’s portfolio offers a crucial opportunity to address some of these challenges by building more resilient healthcare systems, enhancing the EU’s capacity to respond to health crises, and advancing disability rights.

As her mandate begins, we call on the Commissioner to ensure that the European Strategy for the Rights of Persons with Disabilities is updated with ambitious actions for its next phase. We also urge her to prioritise supporting Member States in strengthening disability assessment systems, ensuring people with rare diseases are not left behind due to inadequate recognition of their disabilities