Across Europe, conversations about shrinking civic spaces and the “war on NGOs” are growing louder. Yet a quieter struggle with real consequences for over 30 million Europeans living with rare diseases is unfolding: patient organisations are being pushed out of European Union-funded rare disease research by ever-stricter funding requirements.
As healthcare professionals, we have seen firsthand how indispensable these organisations are in shaping research and innovation that deliver better outcomes. Paradoxically, patients are being hailed as being at the heart of innovation, yet their voices are becoming harder to hear. As policymakers, we cannot allow this misstep to stand.
Patient organisations deserve a seat at the table
The issue lies in how the EU defines who gets to participate in health research programmes. Vague and inconsistent eligibility criteria are being too rigidly interpreted by funding bodies and agencies, excluding patient organisations who rely on multiple funding sources, including private industry support.
Yes, independence matters, and transparency must be a guiding principle, but these highly subjective terms are too easily abused. Properly defining these concepts would prevent the exclusion of the unique and irreplaceable contributions of rare disease patient organisations to clinical trial design and development, patient recruitment and dissemination of findings with affected communities.
Shrinking public health budgets and limited financial support for patient-led initiatives compound the issue. Many patient organisations operate under complex funding realities, often drawing from a mix of sources to sustain their work. Penalising them for this undermines their ability to represent the very communities EU programmes are intended to serve.
We risk creating asymmetric conditions where the communities EU programmes are supposed to support are unable to make their voices heard. This situation defies the reality in which patient organisations operate and weakens the legitimacy of our collective research efforts.
EU leadership through inclusive, patient-driven research
As policymakers, we will continue to advocate for stronger support and more ambitious EU funding for rare disease research, but funding alone is not enough. The right voices must be included in shaping how these resources are used.
The creation of the European Reference Networks (ERNs), which bring together hospital centres of expertise, the launch of the European Rare Diseases Research Alliance (ERDERA) and the strategic vision of initiatives like EU4Health and Horizon Europe have significantly advanced our collective ability to respond to rare diseases.
However, in the European Parliament’s committee on Public Health (SANT) and the Rare Disease Intergroup, we see worrying signs that neither health nor rare diseases are a political priority.
This is in stark contrast to what EU citizens are telling us. Repeated Eurobarometer surveys confirm that health is a top concern. Among young Europeans, public health is ranked higher than employment or migration. The message is clear: Europeans care about health, and so must we.
A holistic research and funding approach that involves patient organisations in every phase of the research process, from setting the agenda to disseminating results must be a priority.
Choosing a better path for rare disease patients
Rigid interpretation of funding rules that shrink the role and recognition of patient organisations could stall rare disease progress. Reaffirming Europe’s commitment to patient-centred research could break down the barriers that stand in the way of rare disease research.
In the face of diminishing resources and political support, the continued involvement of patient communities is critical. Patients hold essential expertise in clinical trial design, patient recruitment, and the effective dissemination of results. Their involvement in research programmes is vital to ensure that outcomes are relevant, inclusive, and grounded in real-world needs.
Failing to include patient organisations that meaningfully contribute to research processes means missing out on crucial insights and limiting the impact of scientific work. We risk developing solutions that do not meet the needs of those they are meant to serve.
The European Medicines Agency (EMA) offers a strong example to follow. Its eligibility criteria recognises patient organisations as equal partners by establishing clear, fair and consistent conditions grounded in transparency and governance. Rare disease patient communities, and even those with very rare diseases, face greater difficulties organising themselves and often rely on multiple sources of funding.
While transparency is key to this reality, the fact is that without multiple forms of funding, many of these communities would not exist. Public authorities must support associations and community participation in healthcare.
Citizen participation moves away from a Weberian view of administration and brings the scientific community, healthcare authorities, and patients closer together in a productive exchange, characteristic of the systems that a deliberative democracy should establish.
Patient involvement is not a luxury, but a necessity. It would be pointless to pursue rare disease research and innovation without involving them. For the millions of Europeans with rare diseases, we must fight for a future where their participation and recognition of their value and input in EU-funded projects is secure.
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