EESC calls for more support and faster treatment for rare disease patients

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In an own-initiative opinion, the European Economic and Social Committee (EESC) has called for the EU to adopt a comprehensive approach to providing more support and faster treatment for rare disease patients.

These diseases may be rare, but they affect up to 30 million people in the EU, as well as their caregivers and families. Patients and relatives often face difficulties in access to diagnoses, treatment and care, and difficulties accessing the labour market and being included in society.

Several measures proposed by the EESC in the opinion Ensuring strong European solidarity for rare disease patients, adopted at its October plenary, aim to facilitate access to diagnoses and treatment, while speeding up the process.

Opinion rapporteur Alain Coheur noted: When it comes to healthcare accessibility today, it takes five to seven years for a rare disease to be diagnosed correctly and for the appropriate treatment to be provided.

All rare disease patients should receive a diagnosis within a year. It is also essential that pregnant women receive diagnoses for their children during perinatal or neonatal screening, as Mr Coheur explained during the plenary. 70% of these rare diseases occur during childhood, and it is vital that they be detected early. Possibly even at the prenatal stage, because that is when the most effective intervention can take place. We are calling on Member States and the Commission to provide more support and faster treatments for rare disease patients.

The proposed measures on faster diagnoses and treatment include support for fundamental research on rare diseases. The European Health Data Space (EHDS), proposed by the European Commission, must be based on the FAIR principle (findability, accessibility, interoperability and reusability). In parallel, Orphanet, a database for rare diseases and drugs, should be made available in all EU languages.

The Health Emergency Preparedness and Response Authority (HERA) mandate offers several opportunities to foster coordination and solidarity. The EESC suggests extending HERA or using it as a model to create a new European authority for non-communicable diseases.

Equal access

Establishing a comprehensive European action plan, with SMART goals attainable by 2030, would help to ensure that all rare disease patients in the EU enjoy equal opportunities for diagnosis, treatment and care.

Accessing healthcare for rare diseases can incur a high price tag due to travel costs and other factors. The EESC advises that the EU research a solidarity fund to cover costs for treatments or for cross-border care where it is not covered by compulsory health insurance, or not covered enough.

Rare disease policy must be kept on the agenda, in line with the 2022 evaluation of the European Reference Networks (ERNs) and the Commission's pledge to revise its rare disease strategy by early 2023.