Leaving No One Behind: European Commitment to Tackling Rare Diseases

Social rights
Health

Background

In Europe, a Rare Disease (RD) is one that affects fewer than five in 10 000 people, with a very rare disease affecting one in 50 000. In many cases, this concerns highly complex illnesses that are potentially fatal, chronic or degenerative. Most of them entail a disability, reduction or loss of independence which affects patients’ quality of life. The physically and mentally demanding long-term care that the patient needs causes significant upheaval to family life. The psychological burden is borne both by the patient and their family.

RD pose a significant burden on healthcare, care systems and society. Responses and initiatives surrounding RD have been developed randomly, meaning there is no integrated assessment system attached to them (indicators, standards, follow-up, etc.).

The EESC has previously adopted two opinions focusing on RD: an opinion on the Proposal for a Council Recommendation on a European action in the field of rare diseases (2009) and an opinion on Ensuring strong European solidarity for rare disease patients (2022). Both these opinions drew the attention of the EU institutions to their responsibility towards rare disease patients.

This opinion aims to address the remaining shortcomings in the EU's response to RD, including the creation of a European Action Plan on Rare Diseases (APRD) with clear, achievable targets.

Key points

The EESC:

  • calls on the Commission to:
    • launch the proposal for  a comprehensive European Action Plan on Rare Diseases (APRD) with SMART targets that can be achieved by 2030 to enable the diagnosis of rare disease patients within one year;
    • set up a Steering Group for the European APRD composed of experts from Member States, EURORDIS members and the EESC to ensure coordination and cooperation, monitoring and supervision of the APRD;
    • encourage agreements with Member States on the content, updating, application and monitoring of national plans for RD;
    • guarantee financing and feasibility of the Action Plan and ensure the necessary budgetary resources for health in the Multiannual Financial Framework for 2028-2035.
  • recommends that the APRD set common and measurable goals that will help define and implement national plans and strategies for RD, encourage Member States and continue cooperation at EU level;
  • calls for specific training to strengthen diagnostic capacities both for healthcare workers, and for social workers and social welfare staff to be able to care more effectively for people with RD.

The text of the draft opinion can be found here.

Additional information

Section: Employment, Social Affairs and Citizenship (SOC)

Opinion number: SOC/806

Opinion type: Exploratory

Rapporteur: Ágnes Cser

Date of adoption by section: 3/10/2024

Result of the vote: 80 in favour/1 against/3 abstentions

Date of adoption in plenary: xxxx – xxxx

Result of the vote:   in favour/ against/ abstentions

Contacts

Press officer: Leonard Mallett

Tel.: 00 32 2 546 93 37

Email: Leonard.Mallett@eesc.europa.eu  

 

Administrator: Valeria Atzori

Tel.: +32 2 546 87 74

Email: Valeria.Atzori@eesc.europa.eu 

Work organisation