Neuromyelitis optica spectrum disorder cost-effectiveness of therapy
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1] Associate Editor(s)-in-Chief: Julinka Auta Fernandes
Overview
Cost-effectiveness of therapy for neuromyelitis optica spectrum disorder (NMOSD) depends on treatment costs, prevention of attacks, and the consequences of disability. A 2025 nationwide study illustrates why both medical expenditure and disability outcomes need consideration when evaluating treatment value.[1]
Published economic findings must be interpreted within the healthcare setting, comparators, and price assumptions used in each analysis.[2]
Cost-Effectiveness of Therapy
Neuromyelitis optica spectrum disorder cost-utility analyses report quality-adjusted life-years (QALYs). The incremental cost-effectiveness ratio (ICER) expresses additional cost per additional health benefit.[2]
Rituximab and Oral Immunosuppressants
Neuromyelitis optica spectrum disorder economic studies have compared rituximab with azathioprine and mycophenolate mofetil.
- South Korea, 2025: A nationwide observational study of 899 patients associated rituximab with fewer relapses but higher overall medical expenditure than oral immunosuppressants. Disability-related costs influenced the authors' assessment of treatment value. The study did not calculate a QALY-based ICER; it should be interpreted as observational cost-and-outcome evidence.[1]
- Thailand, 2020: In a lifetime model for azathioprine-resistant NMOSD, a memory B-cell-guided rituximab biosimilar regimen had a 48% probability of being cost-effective at 160,000 Thai baht (THB) per QALY. A reduced-price generic mycophenolate mofetil scenario also supported cost-effectiveness. Results used 2019 prices.[2]
Targeted Biologic Therapies
Neuromyelitis optica spectrum disorder therapies have been assessed by the Canadian Agency for Drugs and Technologies in Health (CADTH). The reviews below concern aquaporin-4 antibody-positive disease. Monetary results are in Canadian dollars (CAD) and reflect the prices and assumptions reviewed.
| Therapy | Review | Findings and limitations |
|---|---|---|
| Satralizumab | Canada, 2021 | CADTH reanalysis estimated an ICER of CAD 337,535 per QALY for monotherapy versus no treatment, and CAD 752,179 per QALY for addition to conventional immunosuppressants versus immunosuppressants alone. Both exceeded the CAD 50,000 per QALY threshold used in the review. Comparative cost-effectiveness against other active options remained uncertain.[3] |
| Ravulizumab | Canada, 2024 | The sponsor's model estimated CAD 2,386,625 per QALY versus satralizumab. CADTH could not produce a more reliable estimate because of uncertainty in comparative efficacy and long-term extrapolation. A price reduction of at least 73% was estimated to be necessary in that modeled comparison at a CAD 50,000 per QALY threshold.[4] |
| Inebilizumab | Canada, 2024 | CADTH could not reliably assess relative cost-effectiveness because of structural problems in the submitted economic model and limitations in comparative efficacy inputs. A dependable ICER against the available alternatives could not be established.[5] |
| Eculizumab | Comparator in the Canadian ravulizumab review, 2024 | Eculizumab was included as a comparator. Substantial uncertainty in the indirect clinical comparison limits conclusions about comparative treatment value between eculizumab and ravulizumab.[4] |
These historical model results are not current negotiated prices or estimates of an individual patient's out-of-pocket expenses.
Interpretation for Clinical Practice
Neuromyelitis optica spectrum disorder treatment selection should integrate efficacy, safety, disease severity, comorbidities, patient preferences, availability, and costs, as emphasized by the Neuromyelitis Optica Study Group (NEMOS).[6]
- Local assessment: Include local acquisition prices, biosimilar availability, administration costs, monitoring, and the costs of attacks and disability.[5]
- Model uncertainty: Projected lifetime benefits depend on assumptions about comparative efficacy, disability, mortality, and treatment duration.[4]
- Affordability: Assess budget impact alongside cost-effectiveness; a favorable cost-effectiveness result does not establish affordability for every health system.[2]
For treatment regimens, see medical therapy.
References
- ↑ 1.0 1.1 Lee HL, Kang M, Seok JM, et al. (2025). "Comparison of treatment efficacy and cost-effectiveness of rituximab and oral agents among patients with neuromyelitis optica spectrum disorders: a population-based cohort study". Therapeutic Advances in Neurological Disorders. 18: 17562864251314020. doi:10.1177/17562864251314020. PMC 11851748 Check
|pmc=value (help). PMID 40008136 Check|pmid=value (help). - ↑ 2.0 2.1 2.2 2.3 Aungsumart S, Apiwattanakul M (2020). "Cost effectiveness of rituximab and mycophenolate mofetil for neuromyelitis optica spectrum disorder in Thailand: Economic evaluation and budget impact analysis". PLoS ONE. 15 (2): e0229028. doi:10.1371/journal.pone.0229028. PMC 7015451 Check
|pmc=value (help). PMID 32050011 Check|pmid=value (help). - ↑ Canadian Agency for Drugs and Technologies in Health (2021). "Pharmacoeconomic Report: Satralizumab (Enspryng): Executive Summary". National Center for Biotechnology Information (NCBI) Bookshelf. Retrieved 2026-10-30.
- ↑ 4.0 4.1 4.2 Canadian Agency for Drugs and Technologies in Health (2024). "Ravulizumab (Ultomiris): CADTH Reimbursement Review: Pharmacoeconomic Review". NCBI Bookshelf. Retrieved 2026-10-30.
- ↑ 5.0 5.1 Canadian Agency for Drugs and Technologies in Health (2024). "Inebilizumab (Uplizna): CADTH Reimbursement Review: Pharmacoeconomic Review". NCBI Bookshelf. Retrieved 2026-10-30.
- ↑ Kümpfel T, Giglhuber K, Aktas O, et al. (2024). "Update on the diagnosis and treatment of neuromyelitis optica spectrum disorders (NMOSD) – revised recommendations of the Neuromyelitis Optica Study Group (NEMOS). Part II: Attack therapy and long-term management". Journal of Neurology. 271 (1): 141–176. doi:10.1007/s00415-023-11910-z. PMC 10770020 Check
|pmc=value (help). PMID 37676297 Check|pmid=value (help).