OBJECTIVES: The aim of this review is to identify information in the literature on burden of illness and treatment of amyotrophic lateral sclerosis (ALS) and to highlight disparities in the disease and management.
METHODS: A structured review of articles published between 9 May 2019 and 4 July 2024 was conducted, focusing on disease description, epidemiology, humanistic and economic burden, treatment guidelines, treatment patterns, and health disparities.
RESULTS: ALS is a progressive neurodegenerative disorder for which there is no cure, and survival post-diagnosis is typically 2-4 years. Significant diagnostic delays are common and have been reported to be longer for Black versus non-Hispanic White patients. Clinical and economic burden and health-related quality of life (HRQOL) worsen with disease progression, and caregivers experience psychological burden and productivity losses. Current treatment options are very limited. In Europe, riluzole and tofersen (ALS with SOD1 mutations) are the only approved pharmacological products, and in the United States (US), riluzole, edaravone, and tofersen have been approved by the Food and Drug Administration. In a US retrospective study, all racial and ethnic groups had lower odds of receiving riluzole (P = 0.003) than non-Hispanic White patients. None of the available treatments can cure ALS or stop disease progression, and they only have modest effects on prolonging median survival or reducing the rate of disease progression.
CONCLUSIONS: ALS remains a disease with critical unmet needs. There is an immediate need for a new treatment that can help reduce the rate of progression of ALS, increase survival, and allow HRQOL to be maintained for longer durations. Addressing diagnostic delays and ensuring equitable access to emerging new and innovative therapies are essential steps toward reducing health disparities. Improved care and innovative treatments would benefit not only patients with ALS but also their caregivers and healthcare systems.
