A high-level conference convened in Warsaw on April 10, 2025, under the Polish Presidency of the Council of the European Union, focused on the need for a comprehensive policy framework on rare diseases. Experts and policymakers discussed the future direction of rare disease policy during the “Towards an EU Action Plan on Rare Diseases” event.
‘Rare diseases affect millions of people in the EU, and only 6% of them have treatment options, and not in every EU country. We must end the ‘geographical health lottery’ of unequal access to experienced specialists. The pandemic has created grassroots public support for greater European involvement in health, and we cannot be oblivious to these public expectations. Regarding rare diseases, Europe may be the most appropriate response,’ Adam Jarubas, Chair of the European Parliament Committee on Public Health, highlighted.
Organised by the European Economic and Social Committee (EESC), the Polish Ministry of Health, and the Medical University of Warsaw, the conference highlighted the urgency of creating a European policy framework with clear objectives to improve patients’ lives. The European Commission and EU Member States are urged to prioritise an EU-wide Action Plan that addresses the needs of patients and healthcare providers.
‘In recent years, the EESC has been a steadfast advocate for a European Action Plan on Rare Diseases that addresses the diverse needs of patients and healthcare providers. We need to ensure equal opportunities for rare disease patients across all Member States and set additional measures to support health authorities at the national, regional, and local levels, as well as civil society organisations, in their efforts to provide high-quality and affordable care for rare disease patients,’ Baiba Miltoviča, President of the Section for Transport, Energy, Infrastructure and the Information Society, EESC, said.
With over 36 million people affected in Europe, access to timely diagnosis, treatment, and care remains inconsistent. National rare disease plans are vital, but greater European cooperation is essential for sustainable progress. Effective healthcare systems and collaboration between European Reference Networks (ERNs) and national systems are crucial for enhancing patient pathways.
‘Patients with rare diseases need rapid diagnosis and effective therapy. Reducing the time from the onset of symptoms to diagnosis, by improving access to genetic diagnostics and supporting research into new treatments, is a priority. Deepening cooperation in this area makes it possible to ensure effective help for people with rare diseases. A joint strategy by the Member States will enable better access to diagnostics and therapies throughout Europe,’ Izabela Leszczyna, Health Minister of Poland, highlighted.
‘An important element of an efficient patient care system is the identification and establishment of Rare Disease Expertise Centres to ensure the best possible care for patients with rare diseases and close cooperation with European Reference Networks. For better monitoring of processes, we are developing IT tools – the Polish Rare Disease Registry and the electronic Patient Card with a rare disease – new information and data resources to improve care and increase patient safety,’ explained Urszula Demkow, Undersecretary of State of Polish Ministry of Health.
Challenges in accessing effective treatments persist due to regulatory and economic barriers. The ongoing war in Ukraine has severely affected care for citizens with rare diseases, pushing Poland and the EU to unite in addressing these challenges. Timely and accurate diagnosis is critical for better management and outcomes, with advances in genomics and data-sharing initiatives offering new opportunities. The conference followed previous EESC discussions on rare diseases in 2023 and 2024.
‘Following the impactful EESC conferences organised with the EU Council Presidencies in Bilbao, Brussels, and Budapest, I am grateful to the Polish and upcoming Danish Presidencies for advancing towards a European Action Plan on Rare Diseases and hope the Cyprus Presidency will follow. We must provide optimal support for patients with rare diseases, ensuring access to early diagnosis, prevention, and affordable treatment, regardless of their location within the EU,’ noted Ágnes Cser, EESC member, rapporteur for the EESC opinion Leaving No One Behind: European Commitment to Tackling Rare Diseases.
