SHIFTS IN TREATMENT PATTERNS, HEALTHCARE RESOURCE UTILIZATION, AND COSTS IN NMOSD FOLLOWING INTRODUCTION OF APPROVED THERAPIES POST-2019
Author(s)
Stefanie Rodenbeck, MD1, Kavita V. Nair, PhD2, Chris Ho Ching Yeung, PhD3, Chelsea McKibbon, MSc3, Dustin Cavida, PharmD4, Moushmi Singh, MSc5.
1Indiana University Health, Indianapolis, IN, USA, 2University of Colorado Anschutz Medical Campus, Aurora, CO, USA, 3Cytel Inc., Cambridge, MA, USA, 4Amgen Inc., Thousand Oaks, CA, USA, 5Amgen Ltd., Uxbridge, United Kingdom.
1Indiana University Health, Indianapolis, IN, USA, 2University of Colorado Anschutz Medical Campus, Aurora, CO, USA, 3Cytel Inc., Cambridge, MA, USA, 4Amgen Inc., Thousand Oaks, CA, USA, 5Amgen Ltd., Uxbridge, United Kingdom.
OBJECTIVES: Neuromyelitis optica spectrum disorder (NMOSD) is a rare autoimmune disease affecting the optic nerves and spinal cord, leading to blindness, paralysis, and death. Since 2019, multiple targeted therapies received regulatory approval. This study described real-world treatment patterns, healthcare resource utilization (HCRU), and costs among US NMOSD patients receiving ≥1 treatment, pre‑ and post‑2019 eras.
METHODS: This retrospective observational study used Merative MarketScan claims data (January 1, 2016-December 31, 2023). Adult (≥18 years) with a qualified NMOSD diagnosis (index date), ≥1 year pre-index and ≥6 months post-index continuous enrollment, and taking ≥1 NMOSD treatment (both FDA and non-FDA approved) were included. Outcomes included disease specific medication and HCRU treatment patterns and duration, and all-cause and NMOSD-related HCRU costs (total, inpatient, outpatient, emergency room, pharmacy). AQP4-IgG status was not available.
RESULTS: Among 295 treated patients (mean [SD] age 46.7 [13.5] years; 76.6% female; 93.2% commercially insured), first-line treatment before 2019 was predominantly off-label immunosuppressants, including mycophenolate mofetil (45.4%) and azathioprine (31.2%). After 2019, treatment availability increased with emergence of FDA-approved therapies (13.0%), while off-label use remained common (rituximab: 22.7%, mycophenolate mofetil: 22.1%). Compared with pre-2019, the post-2019 era was associated with lower NMOSD-related HCRU (overall mean [SD]: 6.9 [7.8] vs 4.7 [6.8]; inpatient: 0.7 [2.7] vs 0.3 [0.8] visit per-patient-per-year), suggesting improved disease management following the availability of targeted therapies. Although outpatient and pharmacy-related costs increased post-2019, these may reflect greater use of biologics in outpatient and a shift away from inpatient care. Collectively, these findings suggest approved therapies may improve disease control and reduce relapse-related burden.
CONCLUSIONS: Introduction of approved NMOSD therapies after 2019 was associated with meaningful changes in HCRU treatment patterns, and reductions in NMOSD-related HCRU. While healthcare changes during COVID-19 during 2020-2021 may have influenced utilization, these findings show early signals supporting the potential real-world benefits of approved NMOSD therapies.
METHODS: This retrospective observational study used Merative MarketScan claims data (January 1, 2016-December 31, 2023). Adult (≥18 years) with a qualified NMOSD diagnosis (index date), ≥1 year pre-index and ≥6 months post-index continuous enrollment, and taking ≥1 NMOSD treatment (both FDA and non-FDA approved) were included. Outcomes included disease specific medication and HCRU treatment patterns and duration, and all-cause and NMOSD-related HCRU costs (total, inpatient, outpatient, emergency room, pharmacy). AQP4-IgG status was not available.
RESULTS: Among 295 treated patients (mean [SD] age 46.7 [13.5] years; 76.6% female; 93.2% commercially insured), first-line treatment before 2019 was predominantly off-label immunosuppressants, including mycophenolate mofetil (45.4%) and azathioprine (31.2%). After 2019, treatment availability increased with emergence of FDA-approved therapies (13.0%), while off-label use remained common (rituximab: 22.7%, mycophenolate mofetil: 22.1%). Compared with pre-2019, the post-2019 era was associated with lower NMOSD-related HCRU (overall mean [SD]: 6.9 [7.8] vs 4.7 [6.8]; inpatient: 0.7 [2.7] vs 0.3 [0.8] visit per-patient-per-year), suggesting improved disease management following the availability of targeted therapies. Although outpatient and pharmacy-related costs increased post-2019, these may reflect greater use of biologics in outpatient and a shift away from inpatient care. Collectively, these findings suggest approved therapies may improve disease control and reduce relapse-related burden.
CONCLUSIONS: Introduction of approved NMOSD therapies after 2019 was associated with meaningful changes in HCRU treatment patterns, and reductions in NMOSD-related HCRU. While healthcare changes during COVID-19 during 2020-2021 may have influenced utilization, these findings show early signals supporting the potential real-world benefits of approved NMOSD therapies.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EPH20
Topic
Economic Evaluation, Epidemiology & Public Health, Health Service Delivery & Process of Care
Disease
Neurological Disorders