Diagnosing rare diseases, treating them, and facilitating access to care when they occur: the EU is lagging far behind and will have to rely on cooperation to make progress on all three fronts, insisted the seven groups in the European Parliament on Wednesday 24 November in plenary.
6,000 rare diseases affecting 30 million citizens have been registered in Europe, many of which are life-threatening, according to the NGO Eurordis, which brings together 984 rare disease patient organisations.
“Given the limited number of patients and the scarcity of relevant knowledge and expertise, EU action is even more important” in this area, Health Commissioner Stella Kyriakides acknowledged to MEPs.
The Commission, she said, is aware that more needs to be done and that the measures implemented over the last 20 years are not enough.
95% of rare diseases still lack a cure and existing orphan medicines are not available in all Member States, noted various stakeholders.
The EPP and S&D have therefore called on the Commission to focus on joint procurement of treatments and even to set up a dedicated platform for this purpose.
The Commissioner did not respond to this proposal. However, she noted that a proposal for a revision of the European rules on orphan medicines would be presented in early 2022 (see EUROPE 12719/8).
Ms Kyriakides was also called upon by several MEPs to give more support to the 24 European Reference Networks on Rare Diseases (see EUROPE 11735/33).
The Greens/EFA coordinator, Tilly Metz (Luxembourg), even recommended the creation of a data space connected to these networks. “Data on rare disease patients are still scattered amongst different healthcare systems. Collecting this data could help us better understand the specific diseases”, she said.
This issue, Stella Kyriakides promised, will be reflected in the forthcoming proposal on the European Health Data Space (see EUROPE 12712/27). (Original version in French by Agathe Cherki)