Time to Event Milestones in Patients With Amyotrophic Lateral Sclerosis Treated With IV Edaravone: Results From a US Administrative Claims Analysis
Melissa Hagan1, Malgorzata Ciepielewska1, Antoinette Harrison1, Ying Liu2, Jeffrey Zhang2, Stephen Apple1
1Mitsubishi Tanabe Pharma America, Inc, 2Princeton Pharmatech
To estimate the time to progression milestones for insured patients who initiated intravenous (IV) edaravone for amyotrophic lateral sclerosis (ALS).
IV edaravone was approved by the United States Food and Drug Administration for ALS in May 2017 and became available to US healthcare providers in August 2017. Information on real-world outcomes of IV edaravone is limited. 
A retrospective cohort study was conducted using Optum’s de-identified Clinformatics® Data Mart Database (CDM). Patients with a diagnostic code for ALS were selected if they had at least 1 claim for IV edaravone (between August 2017 and December 2018) and at least 6 months of pre- and 12 months of post-index continuous enrollment in the CDM database. The start date of IV edaravone was the index date. Milestones were defined according to the Healthcare Common Procedure Coding System and included the use of canes/walkers/wheelchairs (M1); artificial nutrition (M2); noninvasive ventilation (M3); invasive ventilation/speech-generating devices (M4); and hospice (M5). All variables were assessed descriptively.
In total, 79 patients treated with IV edaravone were analyzed; mean age was 64.2 years (standard deviation: 10.3); 47 (59.5%) were male; 66 (83.5%) were also treated with riluzole; median (interquartile range [IQR]) disease duration before the start of IV edaravone was 163 days (75, 355); 31 (39.2%) had commercial insurance and 48 (60.8%) were covered by Medicare Advantage plans. In the post-index period, the average time to milestones was 9.6, 8.9, 8.1, 8.0, and 11.0 months for M1, M2, M3, M4, and M5, respectively. Median (IQR) treatment duration was 404.0 days (210, 557).
This analysis describes the time to milestones in ALS patients treated with IV edaravone in a real-world setting. This information may be useful to clinicians in optimizing use of IV edaravone for their patients.