NeuroRare Forum

Rare neurological diseases (RNDs) encompass a diverse group of conditions affecting the central and peripheral nervous systems. In Europe, a disease is classified as rare if it affects fewer than 1 in 2,000 individuals. Collectively, rare diseases impact approximately 30 million Europeans, with a significant proportion presenting neurological manifestations. (1)

Rare diseases affect around 30 million people in Europe. Notably, 70% of these conditions begin in childhood, and approximately 75% present with neurological symptoms, highlighting the significant burden of rare neurological diseases across the lifespan. (2)

Access to specialized care varies significantly across Europe. While there are expert centers and European Reference Networks (ERNs) for RNDs, less than 50% of patients currently receive care at these specialized centers (2). Limited awareness among general practitioners and healthcare professionals further hampers timely diagnosis and referral. Treatment options are often scarce, with many conditions having no cure. Multidisciplinary care, including physical therapy and psychological support, is essential but not uniformly available. (3)

The burden of RNDs extends beyond medical challenges, significantly affecting patients, caregivers, and healthcare systems. The chronic and progressive nature of many RNDs results in long-term disability, limiting patients' ability to work and engage in daily activities. (4)

The economic impact is substantial, with costs including direct medical expenses, assistive devices, long-term care, and lost productivity. Families frequently face financial hardship due to high out-of-pocket costs and the need for one or more caregivers to reduce their working hours or leave employment entirely. (5)

In April 2025, the first EAN Industry Round Table on Rare Neurological Diseases (RNDs) was held, bringing together representatives from several pharmaceutical companies and the European Academy of Neurology. This dedicated platform fosters strategic dialogue between industry and EAN, aiming to address shared challenges and opportunities in the RND field. Please contact industry(at)ean.org or click here for more information on EAN’s collaboration with industry.