Advancing Care Pathways for Ataxia and Rare Neurological Diseases in Europe

8th of September 2026 | European Parliament

Rare neurological diseases represent a complex and under-recognised challenge for European health systems. These conditions are chronic, progressive and highly heterogeneous, requiring timely diagnosis, specialist expertise and long-term multidisciplinary care. Yet across Europe, patients still face diagnostic delays, fragmented referral pathways, uneven access to specialist centres and insufficient coordination between medical, rehabilitative and supportive services.

The European Brain Council’s Value of Treatment (VOT) project on ataxia, a rare neurological disorder that causes a loss of control of voluntary muscle coordination, has generated an important evidence base by demonstrating that patient care pathways matter. These studies found that patients attending specialist ataxia centres reported better understanding of their condition, more tailored care, better coordinated referrals and better satisfaction trends than those in non-specialist settings. The accompanying cost analysis showed that, within countries, resource use and total costs were broadly similar between specialist and non-specialist settings, while inpatient stays remained a major contributor to overall costs. The innovation of this work is therefore not only clinical, but also organisational and policy-relevant: it shows that better coordinated specialist care can improve patient experience and pathway quality without clear evidence of a systematic cost penalty. At the same time, innovation in ataxia is entering a new phase. Advances in genomics, biomarkers and emerging therapeutic approaches — including gene replacement, gene editing, oligonucleotide and protein-based strategies — are creating new opportunities in hereditary ataxias, even if major translational and access challenges remain. This makes the need for structured care pathways, expert centres and coordinated research-ready systems even more urgent, so that patients can benefit from both current best care and future therapeutic innovation.

Within EBC’s Rare Brain Disease Ecosystem (RBDE), the ataxia case study forms part of a broader project portfolio aimed at bridging neuroscience, clinical innovation and patient needs across rare brain conditions. In that sense, ataxia serves as a strategic entry point for addressing wider challenges shared across rare neurological diseases, including inequalities in access, fragmented care, emerging needs in genomics and health data, and the need to translate expertise into more coordinated and patient-centred pathways. The challenge is no longer only to document unmet needs, but to build shared priorities for implementation: how to improve earlier diagnosis, organise multidisciplinary follow-up, connect national systems with European expertise, and ensure that innovation translates into tangible improvements in patients’ lives. Such a meeting is essential to move from evidence generation to actionable policy recommendations and to ensure that future rare neurological disease strategies are informed by clinical expertise, lived experience and health system realities alike.

Meeting objective

The meeting aims to translate the European Brain Council’s Value of Treatment findings on ataxia into concrete scientific and policy priorities for Europe, while positioning ataxia within the broader Rare Brain Disease Ecosystem. Using ataxia as a strategic case study, the event will bring together experts, patient association representatives and policymakers to identify how earlier diagnosis, specialist multidisciplinary care, rehabilitation, patient-centred outcomes and cross-border expertise can be better integrated into care pathways for ataxia and other rare neurological diseases. The goal is to support more coordinated, equitable and forward-looking care models and to inform the next phase of European action on rare brain diseases.

Invitations will be sent by email. If you believe you or your organisation would benefit from taking part, we’d be happy to hear from you — please don’t hesitate to reach out to us at research@braincouncil.eu.