The European Parliament Magazine’s Rare Diseases Forum, held in Brussels on 21 April 2026, brought together policymakers, patient advocates, researchers, healthcare professionals and industry representatives to discuss the future of rare disease policy in Europe. Across discussions on innovation, access, research, regulation and healthcare delivery, a clear message emerged: while significant progress has been made in advancing rare disease research and treatment, Europe must now take a more coordinated approach if it is to translate scientific advances into meaningful outcomes for patients.

For the rare neurology community, this message is particularly significant. Rare neurological conditions represent one of the most complex and challenging areas within the wider rare disease landscape. Patients and families frequently face lengthy diagnostic journeys, limited treatment options, fragmented care pathways, and significant inequalities in access to expertise and innovation across Europe. As policymakers consider the next phase of rare disease policy, it is essential that the specific needs of people living with rare neurological conditions are fully reflected within future European initiatives.

A Growing Consensus Around European Action

One of the strongest themes emerging from the Forum was the increasing consensus around the need for a coordinated European Rare Disease Action Plan. Contributions from Members of the European Parliament, including Nicolas González Casares and Tomislav Sokol, highlighted the limitations of fragmented national approaches and the importance of strengthening European cooperation in diagnosis, treatment, research and care.

The call for a comprehensive Action Plan reflects a growing recognition that rare diseases cannot be effectively addressed through isolated national policies alone. While healthcare systems remain the responsibility of Member States, many of the challenges facing rare disease communities transcend borders. Expertise is often concentrated in a small number of specialist centres, patient populations are dispersed across multiple countries, and access to innovation varies considerably depending on geography.

For rare neurological conditions, these challenges are particularly acute. Patients frequently require highly specialised multidisciplinary care, access to centres of excellence, and participation in international research efforts. A coordinated European framework therefore has the potential to improve outcomes not only by supporting research and innovation, but also by strengthening access to expertise and reducing inequalities across Member States.

 

Innovation Must Translate Into Access

A second recurring theme throughout the Forum was the need to ensure that scientific innovation is translated into equitable patient access.

Contributions from Biogen, Chiesi, Amgen, Sanofi, Alexion, PTC and others highlighted the remarkable progress achieved in rare disease research over recent decades. Advances in genomics, precision medicine, gene therapies and other innovative technologies are transforming the possibilities for patients living with previously untreatable conditions.

Yet despite these advances, many patients continue to face substantial barriers in accessing approved therapies. Delays in reimbursement decisions, fragmented access pathways, differences between national systems and unequal availability of treatments continue to create significant disparities across Europe.

For people living with rare neurological conditions, access delays can have profound consequences. Many conditions are progressive, meaning that delays in diagnosis, treatment initiation or access to specialist care may result in irreversible loss of function and reduced quality of life. The challenge for Europe is therefore no longer simply to support innovation, but to ensure that innovation reaches patients in a timely and equitable manner.

As highlighted throughout the Forum, achieving this goal will require greater coordination between European and national decision-makers, stronger collaboration between stakeholders, and a shared commitment to reducing inequalities in access regardless of where a patient lives.

Strengthening Europe’s Rare Disease Infrastructure

The discussions also reinforced the importance of strengthening the infrastructure that supports rare disease diagnosis, treatment and care.

European Reference Networks (ERNs) were repeatedly highlighted as one of Europe’s most successful examples of cross-border healthcare collaboration. By connecting specialist expertise across countries, ERNs play a critical role in supporting diagnosis, facilitating knowledge exchange and improving access to expert care for patients with complex and rare conditions.

Similarly, discussions around the European Health Data Space (EHDS) highlighted the potential for better data sharing and interoperability to support research, accelerate diagnosis and enable more personalised approaches to care.

For the rare neurology community, these initiatives represent important opportunities. Neurological conditions often require highly specialised expertise that may not be available within every healthcare system. Strengthening ERNs, improving data sharing and supporting cross-border collaboration can help ensure that patients have access to the expertise they need, regardless of national boundaries.

The Missing Piece: Understanding the Lived Experience of Rare Neurological Conditions

While the Forum devoted significant attention to innovation, regulation, research and access, it also highlighted the need for a deeper understanding of the realities faced by patients and families living with rare diseases.

Policy decisions are often informed by clinical evidence, regulatory assessments and health economic evaluations. While these are essential, they do not always capture the full impact of rare neurological conditions on everyday life. The burden of delayed diagnosis, treatment uncertainty, mental health challenges, social isolation, caregiver responsibilities, financial pressures and barriers to employment can remain under-recognised within traditional healthcare datasets.

As Europe advances discussions around a Rare Disease Action Plan, there is an opportunity to ensure that lived experience evidence becomes a more central component of policymaking.

This is where patient-generated evidence has an increasingly important role to play.

Through initiatives such as NeuroLytics, EFNA is helping to strengthen the evidence base for neurological patient advocacy by capturing insights directly from people living with neurological conditions. By generating robust data on patient experiences, unmet needs, quality of life, treatment burden and access challenges, NeuroLytics can help ensure that policy discussions are informed not only by clinical outcomes but also by the realities of living with neurological conditions.

Such evidence has the potential to support policymakers, researchers, healthcare professionals and industry partners in identifying priorities, evaluating interventions and developing more patient-centred approaches to care.

Turning Momentum Into Meaningful Change

The Rare Diseases Forum demonstrated that momentum is building behind a stronger European rare disease agenda. The discussions reflected growing political support for coordinated action, renewed commitment to research and innovation, and increasing recognition of the importance of patient involvement in shaping future policy.

However, as several contributors observed, science alone is not enough. Europe has already demonstrated its ability to support innovation. The challenge now is to ensure that scientific progress translates into earlier diagnosis, equitable access, coordinated care and improved quality of life for patients and families.

For the rare neurology community, this means ensuring that future policy frameworks address not only innovation, but also access, healthcare system readiness, specialist workforce capacity, patient involvement and the generation of meaningful lived experience evidence.

As a leading voice for neurological patient organisations across Europe, EFNA welcomes the growing ambition demonstrated throughout the Forum and remains committed to supporting the development of solutions that deliver real-world impact for patients. Working alongside policymakers, researchers, healthcare professionals, industry partners and patient communities, EFNA will continue to advocate for a coordinated European approach that recognises the unique challenges facing people living with rare neurological conditions.

Europe has shown that it can drive scientific progress in rare diseases. The next challenge is ensuring that this progress reaches every patient who needs it. Through collaboration, innovation and meaningful patient partnership, Europe has an opportunity to build a future where no person living with a rare neurological condition is left behind.