Ensuring strong European solidarity for rare disease patients

Key points

The EESC:

• reaffirms its support and solidarity with rare diseases patients and their families and calls for the EU to adopt a comprehensive approach to mitigate the impact of rare diseases on daily, family and professional life;

• to enable patients to get a faster diagnosis and treatment, suggests supporting fundamental research and a European Health Data Space (EHDS) based on the FAIR principle (findability, accessibility, interoperability and reusability), recognising and fully promoting Orphanet’s expertise and making its website available in all EU languages recommends to establish a comprehensive European action plan on rare diseases with SMART goals attainable by 2030, to ensure that all rare disease patients in the EU enjoy equal opportunities to diagnosis, treatment and a holistic perspective on integrated care. The aim must be for patients to receive a diagnosis of their rare disease within a year and to stress the importance of perinatal or neonatal screening;

• suggests extending the Health Emergency Preparedness and Response Authority (HERA) mandate or using it as a model to create a new European authority for non-communicable diseases that would foster coordination on and solidarity for rare diseases;

• recommends that the next trio-presidency 2023–2024 Spain, Belgium, Hungary keep the rare disease policy on the agenda, bearing in mind the 2022 evaluation of the European Reference Networks (ERNs) and the Commission's pledge to revise its rare disease strategy by early 2023;

• requests that the functioning of the ERNs be optimised and calls for their integration into the entire EU and healthcare systems of the Member States;

• recommends a reflection on the possibility of creating a Special EU Financial Fund which Member States contribute to and benefit from according to their financial capacity in order to ensure access to treatment for all European rare disease patients, especially those with unmet medical needs, ensure genuine solidarity in the EU;

• advises researching a solidarity fund for rare diseases, specifically those not included in the ERNs. Such a fund can be a useful addition when the compulsory health insurance does not cover costs for complex or rare disease treatment or cross-border care and the EESC believes that a mutualisation at the European level is a necessity.