This literature review aims to illustrate our current understanding of the societal costs of ALS healthcare, including costs borne by patients, health systems, and payors.

The chronic, progressive nature of ALS places a large economic burden on patients and the healthcare system; a 2014 retrospective cost-of-illness study reports a total national cost of $246-433 million. True costs may be higher given that studies tend not to focus on patient costs, particularly non-medical costs, and philanthropy.

A PubMed search using keywords (ALS, cost-of-illness, economic evaluation, cost study/analysis) yielded 654 articles published between 2000-2022. 496 references were excluded after title and abstract review. Full-text review eliminated 146 articles that were performed outside of the US (n=70), did not address costs (n=43) or ALS (n=31), or were duplicates (n=2).

The twelve papers included in the review revealed a fragmented understanding of ALS-associated costs, limited to medical costs derived from hospital records and insurance databases and omitting patient out-of-pocket expenses (i.e., copays/coinsurance, home/vehicle modifications, appointment-related travel), which are believed to be large cost drivers. Notably, one of the few cost-of-illness studies that measured non-medical costs found that, on average annually, patients and families spent $17,889 on non-medical out-of-pocket costs and lost $14,682 in foregone income.

There remains a need for prospective longitudinal studies estimating ALS-related expenses in relation to disease course, including medical and non-medical patient out-of-pocket costs, and cost-effectiveness studies to determine how to best allocate funds and resources. Additional work is also required to understand the economic impact of novel therapies that slow disease progression. Economic evaluations of ALS can address the urgent need for more accessible evidence-based cost information, which may point to strategies to alleviate the burden of sizeable out-of-pocket expenses concentrated over a short period of time and equip patients to make fully informed decisions about their care.