(C-190) Disease burden and unmet needs of patients with amyotrophic lateral sclerosis in the United States

Background: Amyotrophic lateral sclerosis (ALS) is a rare, progressive neurodegenerative disorder that causes significant disability. ALS diagnosis delays are common, potentially leading to a greater disease burden.

Objectives: To examine characteristics, disease progression milestones, treatments, and annualized healthcare resource utilization (HCRU) and HCRU-associated costs in incident United States-based patients with ALS.

Methods: Patients aged ≥18 years with incident ALS diagnosed between January 2016 and December 2022 were identified from the Inovalon closed claims database. Patients were followed from index date until disenrollment or study end date (August 31, 2023).

Results: Among 12,864 patients with ALS, 23.2% received ≥1 FDA-approved treatment, while 9910 (76.8%) received none. Most patients were aged 50–64 (45.2%), and male (55.1%). The mean (SD) time (days) from diagnosis to treatment was highest for edaravone (303.9 [351.2]), followed by riluzole (125.4 [230.3]). The mean (SD) duration of treatment was 384.3 (384.0) days for edaravone and 391.2 (399.5) days for riluzole. Among the entire ALS cohort, the following proportion of disease progression milestones were reported: mobility aids=34.1%, speech-generating devices=8.3%, noninvasive ventilation=21.3%, invasive ventilation=6.3%, artificial nutrition=26.3%, and hospice care=10.1%. Across all patients with ALS, the annualized mean (SD) number of outpatient visits=18 (20.7), physician office visits=11.5 (15.3), emergency department admissions=1.5 (3.9), ICU admissions=0.2 (0.8), and hospitalizations=0.5 (1.1), with a mean (SD) hospital stay of 4.8 (20.4) days. The following annualized mean (SD) costs for patients with ALS were estimated: total reimbursement ($25,099 [118,886]), outpatient visit(s)=$14,114 ($202,378), hospitalization(s)=$9,626 ($50,595), pharmacy-related=$5,342 ($20,558), and ER visit(s)=$1,226 ($3,413).

Conclusions: This study highlights the substantial healthcare burden and unmet needs of patients with ALS, with the majority being untreated individuals, including prolonged diagnosis to treatment period, high proportion of patients progressing, and high HCRU/associated costs. More research to assess treatment effectiveness and address gaps in ALS care, including approved ALS treatment access, is recommended.