Calling for an EU action plan on rare diseases

Rare diseases affect approximately 8% of the EU population, equivalent to 36 million people. Yet up to 95% of these diseases lack specific treatments, while available therapies come at exorbitant costs. Since 2023, the European Economic and Social Committee (EESC) has pushed for an EU action plan on rare diseases to be included in the Commission’s work programme.

The EESC has always been a frontrunner in calling for a common European approach to the treatment of rare diseases. After bringing together EU institutions, patient organisations and other civil society organisations for the first time at a major summit in Bilbao in 2023, it has kept the need for a comprehensive European action plan on rare diseases firmly in the spotlight. Through its work, the Committee continues to fight the fragmented national approaches to this issue, build bridges and amplify patient voices, ensuring that no patient is left behind. 

To make progress towards an effective European strategy on rare diseases, in 2025 the Committee pushed for an EU policy framework complementing existing legislation, promoting investment and boosting cooperation across borders, at the Warsaw conference Towards an EU action plan on rare diseases.

Technology has a key role to play in this field and that’s why the EESC has put forward a set of recommendations on AI and Big Data. They stress how digital innovation can help shorten diagnostic delays, support personalised medical care and improve crossborder research, while insisting on strong ethics, transparency and patient consent.

The Committee has underscored how the EU can only achieve results if it remains at the forefront of scientific research. With its work on The European Life Science Strategy, the EESC looked to the future and pressed the EU institutions to make Europe the world’s most attractive place for life sciences by 2030. The Committee underlined that research and technology infrastructure is a key enabler for the development of the longevity economy, making advanced personalised treatments possible, for example.

Over time, the undoubted value of the work carried out by the EESC has been recognised within the raredisease community and, for the second time in three years, the EESC received the 2026 Black Pearl Leadership Award from EURORDIS, the European alliance of rare disease patient organisations

The EESC has a unique and vital role to ensure that the voice of organised civil society is heard when shaping the future of technologies such as AI, big data and quantum computing. These fields are not just transforming our economies and societies; they are redefining the very way we live, work and interact. It is therefore essential that ethical, inclusive and human-centred perspectives guide their development.

Baiba Miltoviča, Vice-President of the EESC’s Section for Transport, Energy, Infrastructure and the Information Society

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