Transverse myelitis 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

• There is currently no reliable, completed cost-effectiveness analysis (CEA) that identifies a most cost-effective acute or rehabilitative therapy for transverse myelitis (TM). TM is a clinical syndrome with multiple causes, and treatment selection depends on the disease-specific diagnosis, neurologic severity, expected benefit, safety, and patient circumstances.
• The principal published TM-specific health technology assessment (HTA), the United Kingdom randomiSed controlled TRial of IntraVEnous immunoglobulin (STRIVE), was designed to assess the clinical and economic effects of adding intravenous immunoglobulin (IVIG) to intravenous methylprednisolone (IVMP). Only 2 of the planned 170 participants were randomized; consequently, neither the treatment effect nor the health-economic effect could be determined.
• Clinical guidelines and consensus statements establish when a therapy may be clinically appropriate; they do not, by themselves, establish cost-effectiveness. The absence of a TM-specific economic estimate must not be interpreted as evidence that an indicated treatment is ineffective.

Clinical and economic bottom line
No acute treatment, relapse-prevention treatment, or rehabilitation strategy can presently be designated the single most cost-effective therapy for TM on the basis of reliable TM-specific evidence. Urgent clinically indicated treatment should not be delayed while awaiting an economic comparison.

Cost-Effectiveness of Therapy

Acute and rehabilitative therapy

TM-specific economic evidence

Therapy or strategy	Available economic evidence	Evidence-based interpretation
IVMP alone	IVMP was the standard-therapy comparator in STRIVE, but the trial did not recruit enough participants to estimate costs, outcomes, a quality-adjusted life-year (QALY), or an Incremental cost-effectiveness ratio (ICER).	It is not possible to assign a reliable TM-specific cost-effectiveness ratio.
IVMP plus IVIG	STRIVE planned a prospective economic evaluation using treatment and service-use costs, functional outcomes, and QALYs. Because only two participants were randomized, the clinical and health-economic effects of adding IVIG could not be determined.	IVIG cannot be described as cost-effective or not cost-effective for acute TM on the basis of STRIVE.
Therapeutic plasma exchange (TPE)	The 2026 American Society for Apheresis (ASFA) guideline provides clinical indication categories and evidence grades for disease-specific inflammatory demyelinating disorders, but it does not provide a TM-specific CEA or ICER.	A clinical recommendation for TPE must not be converted into a claim that TPE is the most cost-effective TM therapy.
Rehabilitation and symptom-directed care	No reliable TM-specific comparative CEA was identified for inpatient rehabilitation, outpatient physical therapy, occupational therapy, or symptom-directed multidisciplinary care in the cited guideline and HTA literature.	Services should be individualized according to functional impairment, goals, complications, and expected benefit; a cost-effectiveness ranking is not established.

When TM is caused by aquaporin-4 immunoglobulin G-positive neuromyelitis optica spectrum disorder

• Some patients present with TM as an attack of aquaporin-4 immunoglobulin G (AQP4-IgG)-positive neuromyelitis optica spectrum disorder (NMOSD). Economic evaluations of long-term relapse-prevention therapy in this population apply only after NMOSD has been established; they must not be extrapolated to idiopathic TM or to TM from another cause.
• The 2024 Neuromyelitis Optica Study Group (NEMOS) consensus states that the selection of long-term immunotherapy should consider efficacy, safety, comorbidities, patient preferences, adherence, clinical utility, availability, and costs. It also notes that high-level evidence does not establish the superiority of one available monoclonal antibody over another.

Selected formal HTA findings for AQP4-IgG-positive NMOSD

HTA source	Therapy	Published economic finding	Scope and interpretation
Canadian Agency for Drugs and Technologies in Health (CADTH; now Canada's Drug Agency), 2020	Eculizumab plus standard of care (SOC)	The CADTH reanalysis estimated an ICER of 1,508,152 Canadian dollars per QALY gained versus SOC alone and estimated that a 96% price reduction would be required to reach 50,000 Canadian dollars per QALY.	Results were highly dependent on extrapolated treatment effects. Comparative cost-effectiveness versus rituximab, IVIG, or other active therapies was unknown.
CADTH, 2021	Satralizumab	The CADTH reanalysis estimated 337,535 Canadian dollars per QALY for monotherapy versus no treatment and 752,179 Canadian dollars per QALY for satralizumab plus immunosuppressive therapy (IST) versus IST alone. Estimated price reductions were 80% and 89%, respectively, to reach 50,000 Canadian dollars per QALY.	The model depended strongly on the relapse definition and long-term extrapolation. Comparative cost-effectiveness versus active alternatives remained unknown.
CADTH, 2024	Inebilizumab	CADTH could not reliably estimate relative cost-effectiveness against available comparators because of limitations in the comparative evidence and economic model. Reimbursement was recommended only under conditions that included reducing the cost to no more than the least costly reimbursed comparator.	This is a conditional reimbursement conclusion, not evidence that inebilizumab is clinically equivalent or economically superior to every comparator.
CADTH, 2024	Ravulizumab	CADTH concluded that ravulizumab did not represent good value at the public list price. The submitted analysis estimated an ICER of 2,386,625 Canadian dollars per QALY versus satralizumab; a price reduction of at least 73% was required to reach 50,000 Canadian dollars per QALY, although CADTH could not produce a more reliable estimate because of model limitations.	The estimate is jurisdiction-, comparator-, price-, and model-specific; it is not a TM-wide treatment ranking.
National Institute for Health and Care Excellence (NICE), United Kingdom	Eculizumab, ravulizumab, and satralizumab	NICE terminated the relevant appraisals and was unable to make recommendations for use by the National Health Service (NHS) because manufacturers did not submit appraisal evidence. For ravulizumab and satralizumab, the published advice states that the manufacturers considered cost-effective use of NHS resources unlikely or inadequately supported for the appraised population.	A terminated appraisal is not a finding of clinical ineffectiveness and does not establish comparative cost-effectiveness.

Interpretation for clinicians and decision-makers

• Do not equate efficacy with cost-effectiveness. Clinical benefit, acquisition cost, administration, monitoring, adverse events, relapse-related hospitalization, rehabilitation, caregiver burden, and productivity losses may all affect value.
• Do not equate budget impact with cost-effectiveness. A therapy may increase or decrease annual spending without having a valid cost-per-QALY estimate.
• Do not use simple cost-minimization unless therapeutic equivalence is established. Current NMOSD consensus does not provide high-level evidence that the available monoclonal antibodies are therapeutically interchangeable.
• Apply economic findings only to the population, comparator, perspective, prices, and time horizon studied. Public list prices and confidential discounts change over time; results from Canada or the United Kingdom should not be transferred directly to another health system.
• Prioritize timely evidence-based care. Lack of TM-specific economic evidence should not delay acute treatment when it is clinically indicated. Cost and access should be considered through shared decision-making and local formulary or HTA processes after the clinical indication has been established.

Evidence gaps

• Adequately powered prospective trials of acute TM therapies with embedded resource-use, functional-outcome, health-utility, and caregiver-cost measurements are needed.
• Comparative economic evaluations should stratify patients by disease-specific cause, baseline severity, age, relapse risk, and treatment response rather than treating all TM as one homogeneous condition.
• Long-term studies should capture rehabilitation, assistive devices, bladder and bowel care, loss of employment, unpaid caregiving, and quality of life in addition to drug and hospitalization costs.