Europe's rare disease landscape was already fragile in more stable times. Today, the outlook is worsening. Companies and investors are looking elsewhere. Clinical trials are moving to the U.S. and China, advanced therapies are being developed outside the European Union and patients are waiting years for diagnosis and treatment.
The EU's system of 27 different regulatory frameworks, with 27 different pricing and reimbursement pathways, is killing research and innovation in the field.
More than 2,000 stakeholders, including coordinators of European Reference Networks — cross-border structures connecting hospital centers and specialized units across the bloc to support patients affected by rare diseases — as well as EU and national policymakers and researchers, have signed a joint declaration. It outlines the assets and policy instruments needed to unify and strengthen the ecosystem.
Around 36 million people in the EU are affected by rare diseases. If Europe intends to present itself as a global leader in the study for the prevention of rare diseases, it must create a 28th regime for life sciences. That means reinforcing the strategic role of ERNs, boosting long-term investment, better integrating European medical infrastructure and cutting red tape for treatment of critically ill and terminal patients.
Moving into high gear
Today, 24 ERNs connect 1,606 centers across more than 350 hospitals. They are the only patient-centered, cross-border clinical networks of their kind globally. Yet they still lack the resources to expand their reach and train the next generation of specialists.
The EU must act quickly, by immediately committing to ringfenced funding for ERNs in the 2028-2034 European long-term budget.
The new European Competitiveness Fund is set to become the primary financial engine for health in the next Multiannual Financial Framework. However, it remains unclear how it would enhance pan-EU infrastructure, data connectivity and public-private partnerships.
ERNs would, in fact, benefit from deeper integration with other relevant assets and infrastructures, such as biobanks, AI-powered analytics and the 1+ Million Genomes initiative — a European project launched to provide access to clinical data across member states.
Moreover, ERNs can be strengthened by promoting public-private partnerships and programs, including the European Rare Diseases Research Alliance and the Joint Action on Integration of ERNs into national healthcare systems. Bundling research, diagnostics and care delivery will not only better serve the needs of people living with rare diseases, but will also bolster broader health systems.
The EU must also reduce excessive legal complexity to accelerate access to innovative treatment for those who need them most.
The existing drug approval pathway is a barrier for patients facing life-threatening conditions without available treatment. Among the most urgent priorities, Brussels must encourage multi-country clinical studies by simplifying regulatory requirements, harmonizing ethical standards and reducing approval times.
A 28th regime for life sciences
The recently proposed EU Inc. regulation — aimed at harmonizing rules for company formation — is a crucial starting point. However, to create a 28th regime for the life sciences sector, EU Inc. must be combined with the European Commission's proposal for a Biotech Act, which would help bridge the gap between scientific discovery and the capacity to scale.
Additional policy instruments are needed: comprehensive EU pharmaceutical legislation; the European Health Data Space regulation, which establishes common rules for the exchange of electronic data across the Union; and a reformed medical devices regulation to reduce administrative burdens.
This framework would allow EU companies to scale across a unified European market and enable patients in smaller countries to have access to innovative treatments on the same terms as those in larger ones.
Through smart legislation and immediate, substantial funding, the EU can translate innovation "from bench to bedside," ensuring that research findings turn into concrete therapies that reach patients faster, more efficiently and more equitably.
Sign up to The Parliament's weekly newsletter
Every Friday our editorial team goes behind the headlines to offer insight and analysis on the key stories driving the EU agenda. Subscribe for free here.