Myasthenia gravis cost-effectiveness of therapy
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1] Associate Editor(s)-in-Chief: Hafiz M. Ahmed, M.D.[2]
Overview
Formal cost-effectiveness data for myasthenia gravis (MG) therapy are limited. Treatment selection nonetheless incorporates cost considerations, particularly in positioning newer, high-cost biologic agents relative to established, lower-cost immunotherapies.
Cost-Effectiveness of Therapy
Established immunosuppressants and thymectomy remain the foundation of chronic MG management. Older steroid-sparing agents such as azathioprine, mycophenolate mofetil, and methotrexate are relatively inexpensive, and their corticosteroid-sparing effect can lower the long-term costs linked to chronic steroid toxicity. Thymectomy likewise reduces immunotherapy requirements and hospitalizations for exacerbations over time.
Newer biologic agents are substantially more expensive and are generally reserved for refractory disease:
- Eculizumab, a complement C5 inhibitor, is effective in severe, refractory acetylcholine receptor (AChR) antibody–positive generalized MG but is a high-cost therapy best reserved for patients in whom other immunotherapies have failed to achieve treatment goals. Its use adds further expense for mandatory meningococcal vaccination and, when treatment cannot be delayed, antibiotic prophylaxis.
- Rituximab is a reasonable early option in MuSK antibody–positive disease that responds poorly to first-line therapy, but its benefit in refractory AChR antibody–positive MG is uncertain, a consideration relevant when weighing its cost against expected clinical benefit.
A stepwise approach is generally favored, in which lower-cost, better-established therapies are used first and higher-cost biologics are reserved for refractory disease until stronger comparative cost-effectiveness evidence is available.[1]
References
- ↑ Narayanaswami, P., Sanders, D. B., Wolfe, G., Benatar, M., Cea, G., Evoli, A., Gilhus, N. E., Illa, I., Kuntz, N. L., Massey, J., Melms, A., Murai, H., Nicolle, M., Palace, J., Richman, D., & Verschuuren, J. (2021). International consensus guidance for management of myasthenia gravis: 2020 update: 2020 Update. Neurology, 96(3), 114–122. https://doi.org/10.1212/WNL.0000000000011124