Eurordis - Rare Disease Europe

We are particularly concerned by the absence of a standalone successor to the EU4Health programme. Strong, solidarity-based health systems are a core European value and a strategic advantage. Absorbing health under a general competitiveness agenda risks diluting the EU’s focus on long-term healthcare resilience, access, and equity. Although the proposal includes €22.6 billion for health, biotechnology, bioeconomy, and agriculture combined, the lack of a dedicated envelope for health leaves its future impact uncertain and raises questions about the political priority given to building the European Health Union. A clear, visible health strand is urgently needed – one that addresses prevention, innovation, and persistent inequalities across Member States. Ultimately, we call for a budget that guarantees EU solidarity with the most vulnerable citizens, including people living with rare diseases and their families. 

We continue to call for a strategic EU framework on rare diseases – not necessarily backed by drastically increased spending, but focused on making better use of existing investments, coordinating efforts, and delivering measurable outcomes for people living with rare conditions. Such a framework would build on what works while helping to close persistent gaps. 

One clear success is the European Reference Networks. Continued and predictable funding for ERNs – including their registries, virtual consultations, and integration into the European Health Data Space – will be essential for ensuring access to specialised care and driving innovation. However, it is important to recognise that ERNs are not the be-all and end-all; they represent a vital component of a broader strategy to improve rare disease care, but must be supported by complementary initiatives at both national and European levels. 

We also strongly emphasise the need to sustain operating grants for civil society and patient organisations. These groups are not merely project collaborators – they are catalysts for meaningful real-world change and essential drivers of inclusive policymaking who often fulfil roles mandated by EU legislation to engage in regulatory and legislative processes.

We welcome the decision to maintain FP10 as a standalone research and innovation programme, nearly doubling its budget from Horizon Europe to €175 billion and linking it more closely to the new European Competitiveness Fund. If designed with health and care systems in mind, this structure could help bridge the gap between fundamental science and real-world application. 

To accelerate innovation, it is essential to preserve strong support for basic research and to secure the funding needed to deliver on the EU’s Life Sciences Strategy. Undervaluing fundamental research today risks stalling tomorrow’s medical breakthroughs. 

Rare disease research must remain a visible priority within FP10, covering all stages from basic to clinical science. Simplified access for patient-led consortia and stronger pathways from research to care – including HTA integration and access planning – are vital. 

We also call on the Commission to ensure the Competitiveness Fund addresses societal as well as industrial priorities. Europe’s innovation strength lies in tackling real-world challenges – especially unmet medical needs.