On 29 November 2024 in Budapest, the European Economic and Social Committee (EESC) conference, ‘For an EU Commitment to Tackling Rare Diseases’, brought renewed focus to progress towards a European Action Plan for Rare Diseases by 2030.
ERN eUROGEN was represented at this important event by Peter Mulders, ERN eUROGEN Coordinator and Michelle Battye, Programme Manager.
Hosted under the Hungarian Presidency of the Council of the EU, the event underscored the importance of harmonised European policies, strengthened healthcare systems, and collective action to address rare diseases.
The conference followed the EESC’s adoption of its exploratory opinion, ‘Leaving No One Behind: European Commitment to Tackling Rare Diseases,’ at its October plenary session. The Hungarian Presidency requested this opinion, which calls for an integrated European health framework with specific targets for improving rare disease care by 2030.
During the opening session, Baiba Miltoviča, President of the Section for Transport, Energy, Infrastructure and the Information Society at the EESC, stated: “We strongly believe we need an EU Action Plan on rare diseases. EU and health ministers of Member States must make rare diseases a priority … We already have tools such as the Cross-Border Healthcare Directive and the Pharmaceutical Package; now, we need to implement these regulations and ensure proper enforcement.”
Antonio Parenti, Director at DG SANTE, European Commission, highlighted the importance of EU collaboration, recalling that ERNs are a “flagship activity with clear EU added value and a perfect example of what can be achieved when countries come together, sharing knowledge and resources to advance diagnosis, treatment, and research for rare diseases.”
A key moment of the conference was the presentation of the EESC’s exploratory opinion by Ágnes Cser, its rapporteur, who called for a European coordination body to centralise efforts, stating: “All people suffering from rare diseases should receive a diagnosis before one year. The civil society representatives should, together with the support of EU institutions, create a coordination body to know people’s and families’ needs to have information in their native language. Parents should not have to travel 1,000 kilometres to know the problems of their children.”
Speaking on behalf of Europe’s rare disease community, Virginie Bros-Facer, Chief Executive Officer of EURORDIS, called for urgent and extensive action: “No single country – not even France, Italy, or Germany – can fully address the complex challenges rare disease patients face. By pooling expertise, resources, and efforts, the EU can help bridge gaps, foster collaboration across borders, and address the unmet needs of a very vulnerable population.
“An Action Plan is not about reinventing the wheel. We have a wealth of existing resources, including the ERNs, the Joint Action JARDIN, and national rare disease strategies, that can serve as a strong foundation. However, what is urgently needed is the ‘glue’ to bring these elements and stakeholders together in a cohesive, organised, and coordinated manner.
At the closing session, Mónica García Gómez, Minister for Health of Spain, stressed the need for coordinated action: “Today, we have a chance to change millions of lives. To do this, we need action plans in each country, a European Plan, and a global strategy. It is essential that the European Union consolidates nearly two decades of work by establishing an Action Plan for Rare Diseases.”
Urszula Demkow, Undersecretary of State at the Ministry of Health, welcomed collaboration under Poland’s upcoming Presidency, stating: “We will be next for the Presidency of the Council, and we will be very happy to have you in Warsaw as the conference on rare diseases is supposed to be scheduled in April 2025. Please come to help us organise it!”
The Budapest conference reaffirmed the shared commitment of European institutions, civil society, and patient advocates to achieving a European Action Plan for Rare Diseases. Marking a shift from dialogue to action, it emphasised the urgency of drafting the plan and building the framework needed to turn this vision into reality
