Brussels, 28 February 2021

Today, 30 million citizens suffer from a rare disease in the EU, with 95% of such diseases remaining without a treatment option. These figures remind us that, ahead of this World Rare Disease Day, we need to join forces to improve the health and quality of life of each and everyone affected. Our goal is clear: to ensure that all rare disease patients in Europe have access to the right diagnosis and treatment they need. This is especially relevant today as, during the first months of the COVID-19 pandemic, an estimated 84% of rare disease patients experienced some sort of disruption of their care.

Progress has been made in the EU. 193 medicines for the benefit of more than 6 million patients living with rare diseases have been authorised by the EU. 24 European Reference Networks (ERNs) are connecting more than 900 specialised healthcare units from more than 300 hospitals across Europe. By striving to speed up diagnosis, improve care standards, and generate knowledge and new evidence for patients living with rare and complex diseases, these Networks are clear examples of what EU cooperation in healthcare can achieve. They follow the principle that knowledge should travel instead of patients. This is particularly important for rare diseases.

Going forward, supported by our new and ambitious EU4Health programme we will make these Networks even stronger, enlarge their geographic coverage and extend them to new or not sufficiently covered diseases.

We are revising EU rules on orphan medicines to foster research and development around treatments for rare diseases, especially in areas of unmet medical needs. Rare cancers have also been given a prominent place in the Europe’s Beating Cancer Plan that we adopted at the beginning of February.

Looking ahead, the strong European Health Union we are building will strengthen our healthcare systems overall so that they can better deliver for all patients, and give hope to those millions of Europeans who have to face the daily reality of a living with a rare disease. Today, we stand and will always stand alongside them in full solidarity.