Background: Patients with amyotrophic lateral sclerosis (ALS) have several US Food and Drug Administration (FDA)-approved treatment options. Riluzole was the first FDA-approved treatment for ALS in December 1995. In 2017, intravenous (IV) edaravone was approved by the FDA after being shown in clinical trials to slow the rate of physical functional decline in patients with ALS. Additionally, oral edaravone and sodium phenylbutyrate, taurursodiol (PB-TURSO), and tofersen were FDA approved for use in patients with ALS in May 2022, September 2022, and April 2023 respectively.
Objectives: To describe preliminary data on demographics, characteristics, and treatment combinations in patients with ALS in this real-world, observational, US-based administrative claims analysis.
Results: Patients with ALS who were continuously enrolled in Optum’s de-identified Clinformatics® Data Mart (CDM) from August 1, 2015, through March 31, 2023, were included and grouped based on use of riluzole (n=2662), edaravone (n=109), PB-TURSO (n=31), riluzole+edaravone (n=442), edaravone+PB-TURSO (n=24), riluzole+PB-TURSO (n=102), or riluzole+edaravone+PB-TURSO (n=149). The index date was the date of ALS diagnosis. Patients were closely balanced between male and female with almost all groups including slightly more males (48.4%-56.1%). For most groups, more patients were aged 60 to 69 years (32.9%-41.9%) than any other age group. Patients were predominantly White (70.6%-87.1%) and covered by Medicare (54.2%-74.5%) rather than commercial insurance. Pre-index disease progression milestones were noted, including use of canes/walkers/wheelchairs, artificial nutrition, non-invasive ventilation, invasive ventilation, hospitalization, and gastrostomy tube placement.
Conclusions: Additional results are expected for these preliminary analyses of real-world data that may help clinicians and payers better understand the demographics, clinical characteristics, and current treatment combinations in patients with ALS, including those treated with edaravone.
Sponsorship: This study was sponsored by Mitsubishi Tanabe Pharma America, Inc.