CHARACTERISTICS AND TREATMENT PATTERNS OF PATIENTS WITH SPINAL MUSCULAR ATROPHY IN THE UNITED STATES
Author(s)
Crystal Watson, MS1, Daymelis Santiesteban, MPP2, Robert Sedgley, BS2, Thomas Brown, PhD1, Taylor Ryan, MHI2, Curtis Griffith, PharmD1, Andrej Marich, PhD, PT1, Mouhamed Gueye, PharmD, MBA1, Jessamine Winer-Jones, PhD2, Mac Bonafede, MPH, PhD2.
1Scholar Rock, Cambridge, MA, USA, 2Veradigm, Raleigh, NC, USA.
1Scholar Rock, Cambridge, MA, USA, 2Veradigm, Raleigh, NC, USA.
OBJECTIVES: Spinal muscular atrophy (SMA) is a rare neuromuscular disease characterized by motor neuron degeneration and progressive muscle atrophy. This study examined patient characteristics and treatment patterns among treated patients with SMA in the United States.
METHODS: This retrospective analysis identified patients in the Veradigm Network EHR linked to Komodo open claims with ≥1 claim for nusinersen, risdiplam, or onasemnogene abeparvovec (OA) between 01/01/2022 and 07/31/2025. Index date was the earliest qualifying event with ≥6 months of prior claims activity. Patients were required to have ≥6 months of post-index claims activity (variable-length follow-up). Patients with missing age, missing sex, or post-index evidence of misdiagnoses/rule-out diagnoses were excluded. Patients were stratified by treatment: nusinersen (follow-up), OA (anytime), risdiplam (follow-up). Patient characteristics and treatment patterns were captured.
RESULTS: Of 1,057 patients meeting the inclusion criteria, 490 (46.4%) received nusinersen, 482 (45.6%) risdiplam, and 85 (8.0%) OA. Among all patients, mean (SD) age was 22.1 (17.4) years and 52.1% were female: 24.7 (17.1) years and 50.2% for nusinersen, 23.2 (16.9) years and 55.0% for risdiplam, and 1.7 (1.4) years and 47.1% for OA. During a mean (SD) follow-up of 3.5 (0.8) years, 9.8% of the nusinersen cohort and 4.6% of the risdiplam cohort switched treatments; 27.1% of the OA cohort added nusinersen or risdiplam in the follow-up. During follow-up, 57.6% of patients received prescription pain medication and 41.5% received opioids: 65.3% and 49.6% nusinersen, 55.0% and 38.4% risdiplam, and 28.2% and 12.9% OA, respectively.
CONCLUSIONS: This analysis suggests there continues to be a persistent and high burden of disease and a need for additional SMA treatment options as 1 in 4 patients who received OA initiated an alternative treatment afterwards, and over half of patients treated with nusinersen or risdiplam received prescription pain medication in the follow-up period.
METHODS: This retrospective analysis identified patients in the Veradigm Network EHR linked to Komodo open claims with ≥1 claim for nusinersen, risdiplam, or onasemnogene abeparvovec (OA) between 01/01/2022 and 07/31/2025. Index date was the earliest qualifying event with ≥6 months of prior claims activity. Patients were required to have ≥6 months of post-index claims activity (variable-length follow-up). Patients with missing age, missing sex, or post-index evidence of misdiagnoses/rule-out diagnoses were excluded. Patients were stratified by treatment: nusinersen (follow-up), OA (anytime), risdiplam (follow-up). Patient characteristics and treatment patterns were captured.
RESULTS: Of 1,057 patients meeting the inclusion criteria, 490 (46.4%) received nusinersen, 482 (45.6%) risdiplam, and 85 (8.0%) OA. Among all patients, mean (SD) age was 22.1 (17.4) years and 52.1% were female: 24.7 (17.1) years and 50.2% for nusinersen, 23.2 (16.9) years and 55.0% for risdiplam, and 1.7 (1.4) years and 47.1% for OA. During a mean (SD) follow-up of 3.5 (0.8) years, 9.8% of the nusinersen cohort and 4.6% of the risdiplam cohort switched treatments; 27.1% of the OA cohort added nusinersen or risdiplam in the follow-up. During follow-up, 57.6% of patients received prescription pain medication and 41.5% received opioids: 65.3% and 49.6% nusinersen, 55.0% and 38.4% risdiplam, and 28.2% and 12.9% OA, respectively.
CONCLUSIONS: This analysis suggests there continues to be a persistent and high burden of disease and a need for additional SMA treatment options as 1 in 4 patients who received OA initiated an alternative treatment afterwards, and over half of patients treated with nusinersen or risdiplam received prescription pain medication in the follow-up period.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EPH87
Topic
Epidemiology & Public Health, Health Service Delivery & Process of Care
Disease
Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal), Rare & Orphan Diseases