Help 36 million people in Europe with rare diseases

Baiba Miltoviča, President of the Section for Transport, Energy, Infrastructure and the Information Society at EESC, lifts the lid on rare diseases, charting a European mission to help 36 million people in the EU

Europe needs a strategy to give people with rare diseases the care they need in the EU Member States where the best treatment is available.

At its conference in Bilbao on 10th-11th October 2023, the European Economic and Social Committee (EESC) issued a strong plea urging the European Union to launch a European action plan to fight rare diseases.

The main goal is to increase cooperation between national health systems and provide better diagnoses, treatment and care to patients with rare diseases.

Rare diseases pose a serious problem across all EU Member States, and the nascent European Health Union can play a crucial role in tackling them.

The European Health Union we are building must be strong and look after each of us.

Rare diseases: what are we talking about?

In practice, rare diseases are less rare than we think.

The figures are alarming: currently, there are over 7,000 rare diseases affecting 8% of the EU’s population, approximately 36 million people.

Rare diseases are conditions that are usually chronic and sometimes disabling or life-threatening.

Around 80% are genetic, with 70% beginning in childhood. Up to 95% of these diseases have no specific treatment or cure.

Examples include amyotrophic lateral sclerosis (ALS), sickle cell disease and haemophilia.

The complexity of these diseases and the scarcity of data and knowledge make it difficult for patients, family members, and healthcare professionals to receive a timely diagnosis, appropriate health and social care and any available treatment.

The need for a European strategy

The European Union has supported policy developments and research in the field of rare diseases extensively for more than 25 years. Still, it needs a European strategy to fight rare diseases, as no Member State can succeed alone.

The 24 European Reference Networks (ERNs), established in 2017 with the support of the European Commission, constitute an important milestone in European cooperation between healthcare systems and bring together some of the best professionals and researchers in the EU across almost all areas of medicine and healthcare.

This makes it easier to share experience in diagnosis, provide treatments and generate knowledge and research for European patients with rare diseases.

Since 2009, the EESC has been supporting a comprehensive EU approach that takes account of all the needs of people living with rare diseases.

In 2022, the Committee issued an opinion reiterating this request and called for EU solutions to mitigate the everyday impact of rare diseases on both personal and professional lives.

The time has come to make progress and listen to the calls of patients’ associations, which are active on the ground.

New European funding for rare diseases

The ERNs must work better and be integrated across all EU and Member State healthcare systems. Currently, some EU countries do not participate – or only do so to a limited extent – in the network, which could cause medical inequalities.

The EU should also reflect on the possibility of creating a special EU fund. Member States would contribute to and benefit from it according to their financial capacity, ensuring access to treatment for all European patients with rare diseases, especially those with unmet medical needs, making genuine solidarity in the EU a reality.

Researching the possibility of a solidarity fund for rare diseases, specifically those not included in the ERNs, is also a priority. Such a fund could be a helpful addition when compulsory health insurance does not cover costs for complex or rare disease treatment or cross-border care. It is clear: a European insurance scheme for rare diseases is a necessity.

We can no longer wait. We need to join forces with the European Union.

We need to put together a European action plan to strengthen European cooperation between health systems and give patients with rare diseases access to the most advanced knowledge and treatment wherever they are in the European Union.

Building a common front in Europe to combat rare diseases is needed now more than ever. No single Member State can do it alone.

Patients with rare diseases deserve the best care, no matter where it is available in Europe.