Eurordis - Rare Disease Europe

In the EU, we estimate that around 30 million people live with a rare disease. So while rare diseases are rare, patients living with them are not. This is why the Commission has been active in this field for many years. A strong European Health Union helps improve the health of all our citizens—no matter where they live, no matter their disease or complex condition.

Our work on rare diseases is underpinned by a solid EU framework, based on the Commission Communication on Rare Diseases, the Council Recommendation on action in the field of rare diseases, and the Cross-Border Healthcare Directive.

On this strong foundation, the Commission is supporting Member States through targeted and concrete actions that can make a real difference for rare disease patients.

The EU can do a lot in this area, but we do not have a comprehensive plan for these diseases. It’s time to change that. We need a European rare diseases plan with clear objectives, defined deadlines, benchmarks, and finance.

Vytenis Andriukaitis MEP (S&D, Lithuania), and former European Commissioner for Health and Food Safety (2014-2019)

Why do we not pool our efforts more? We should work more closely together in the EU share experience, data, and best practices. We need cross-border cooperation to ensure that the best researchers, doctors, and solutions are available to those patients that most urgently need it. Commissioner Várhelyi, thank you for making a start here – we expect more.

Stine Bosse MEP (Renew, Denmark)

President, Commissioner, for me there are around 30 million reasons why we need to take bold action now. Thirty million children and adults across Europe have to fight every day. The fight is mostly very unequal, as diagnosis takes years. Patients have limited treatment options, specialists may not be anywhere nearby, and treatments are very expensive. They have to fight because they live with rare diseases – most of which are genetic and affect children. Rare diseases are not rare if we look at them all together. That is exactly where the EU’s added value lies. This is why a European Action Plan on Rare Diseases is needed – and is needed now.

Tilly Metz MEP (Greens/EFA, Luxembourg)

We need public-private partnerships to promote research, innovation, and the transfer of technological expertise. We need a regulatory framework that supports research. We need a European strategy that helps us overcome the fragmented and piecemeal approach we currently have – one that ensures all patients can access treatment, regardless of which Member State they come from.

Letizia Moratti MEP (EPP, Italy)

We need to build on the European Reference Networks, speed up research and drug development, train specialists, build centres, and allow cross-border treatment. To do this, we need an Action Plan. We need commitment from the European Parliament. And we need Member States to work together. This is necessary so that in the future, we can talk not about how many children we’ve lost – but about how many lives we’ve saved.

András Tivadar Kulja MEP (EPP, Hungary)

Families are being abandoned. We urgently need a plan, including measures to tackle rare diseases, which allows access to treatment in other countries where there is the necessary experience – because Europe can do what many nation states at national level are not able to do. So, we call on the Commission and the Council to cooperate with the Parliament to establish this plan, because the European Union is not just freedom of movement – it’s also freedom to benefit from adequate treatment everywhere. And cross-border medical care is important.

Vlad Vasile-Voiculescu MEP (Renew, Romania)

The EU initiatives referenced by Commissioner Várhelyi – ERNs, ERDERA, the general pharmaceutical reforms, and the Critical Medicines Act – are all significant and valued. But the guiding policy instruments – the Commission Communication and Council Recommendation – are over a decade old. They are no longer fit for today’s realities. They are not equipped to keep pace with scientific advances, the pressing unmet needs of people living with rare diseases in the EU, and the many policy developments that have taken place since their adoption.

If we continue with only these outdated tools, we will fail to tackle the challenges our community faces in a coherent, forward-looking way. Inequities in care and health outcomes will persist. Diagnostic delays will remain a reality for many. The social and mental health impacts of rare diseases will continue to be overlooked. Research and innovation will stay fragmented. And even if fully integrated into national systems, the ERNs will not reach their full potential without a unifying EU vision.

What we need now is a comprehensive European Action Plan that connects the dots, sets measurable goals, and delivers tangible results.

Thanks to efforts at both national and EU levels, many essential building blocks are already in place. What’s missing is the mortar – a strategy that binds these elements into a durable, impactful whole. Only with the ‘strategic mortar’ of a European Action Plan can we mould these disparate components into coordinated and transformative change.