A Dolon Institute Report
Amyotrophic lateral sclerosis (ALS) is a rapidly progressive and life-threatening neurodegenerative disease, affecting around 32,000 people in Europe. People living with ALS typically experience an irreversible loss of muscle function and, for many, survival is only 2–5 years from symptom onset. Despite the severity and urgency of the disease, significant gaps remain across diagnosis, care and access to effective treatments.
This policy paper from the European ALS Coalition examines what it means to receive an ALS diagnosis and live with the disease across Europe. Bringing together clinicians, patient representatives, policymakers, payers, HTA experts and other stakeholders, it identifies the key challenges faced by people living with ALS (PLWALS), their families and caregivers, and sets out 10 actionable policy recommendations to improve outcomes.
A central theme is the need for earlier and more accurate diagnosis. Delays and misdiagnoses remain significant challenges, while limited disease understanding can make it difficult to identify and characterise ALS effectively. The Coalition calls for stronger capabilities among primary and frontline healthcare professionals, expert-led diagnosis from the point at which ALS is suspected, and ongoing assessment to better understand disease subtype and individual progression.
The paper also highlights major shortcomings in the current model of care. ALS is complex and changes rapidly, yet healthcare systems are often reactive rather than proactive. Access to multidisciplinary care varies considerably across Europe, leaving patients and caregivers to navigate fragmented services and make complex decisions with insufficient support. The Coalition advocates greater coordination between ALS and non-ALS specialists, stronger social care provision, faster access to assistive technologies and greater support for families and caregivers.
Finally, the paper considers the urgent need to improve prognosis through innovation. Despite increasing clinical research activity, the unmet need remains enormous, with only one treatment having been approved for ALS in Europe in more than 25 years at the time of publication. The Coalition calls for greater investment in research, improved access to clinical trials, closer alignment between researchers, clinicians, developers, regulators and payers, and faster routes to approval and reimbursement for new treatments.
The paper’s overarching message is that ALS requires a more proactive, integrated and patient-centred approach across the entire disease journey. Improving diagnosis, care and access to innovation could not only transform the lives of people living with ALS but also provide valuable lessons for other severe and complex neurological diseases.