Abstract

Neuromyelitis optica spectrum disorder (NMOSD) is an inflammatory condition of the central nervous system. The extent of disability depends on the severity of the disease and the number of relapses. Although azathioprine is currently the main treatment for patients with NMOSD in Thailand, patients often relapse during its use. Hence, it is argued that there are other drugs that would be more effective. The purpose of this study is to evaluate, from a societal perspective and from the economic impact on Thailand's healthcare system, the cost utility of treatment with mycophenolate mofetil (MMF) and rituximab in patients resistant to azathioprine. The Markov model with a one-year cycle length was applied to predict the health and cost outcomes in patients with NMOSD over a lifetime. The results showed that rituximab exhibited the highest quality-adjusted life year (QALY) gains among all the options. Among the rituximab-based treatments, the administration of a rituximab biosimilar with CD27+ memory B cell monitoring proved to be the most cost-effective option. At the willingness-to-pay threshold of 160,000 Thai baht (THB), or 5,289 US dollar (USD), per QALY gained, the treatment exhibited the highest probability of being cost effective (48%). A sensitivity analysis based on the adjusted price of a generic MMF determined that the treatment was cost effective, exhibiting an incremental cost-effectiveness ratio of -164,653 THB (-5,443 USD) and a 32% probability of being cost effective. The calculated budget impact of treating patients resistant to conventional therapy was 1-6 million THB (33,000-198,000 USD) for the first three years, while after the third year, the budget impact stabilized at 3-4 million THB (99,000-132,000 USD). These data indicate that, in Thailand, treatment of drug resistant NMOSD with a rituximab biosimilar with CD27+ memory B cell monitoring or treatment with a generic MMF would be cost effective and would result in a low budget impact. Therefore, the inclusion of both the rituximab biosimilar and a generic MMF in the National Drug List of Essential Medicine for the treatment of NMOSD may be appropriate.

Highlights

  • Neuromyelitis optica spectrum disorder (NMOSD) is a devastating central nervous system (CNS) inflammatory demyelinating disease that is caused by autoantibodies targeting aquaporin-4 immunoglobulin G (AQP4-IgG) [1]

  • The results showed that rituximab exhibited the highest quality-adjusted life year (QALY) gains among all the options

  • The outcomes were estimated based on qualityadjusted life years (QALYs) gained, and the incremental cost-effectiveness ratio (ICER) was expressed in Thai baht (THB) per QALY gained

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Summary

Introduction

Neuromyelitis optica spectrum disorder (NMOSD) is a devastating central nervous system (CNS) inflammatory demyelinating disease that is caused by autoantibodies targeting aquaporin-4 immunoglobulin G (AQP4-IgG) [1]. Severe attacks are typically managed by treatment with high dose steroids followed by a plasma exchange to rescue neurological function [3]. The cost of treatment is higher in patients with acute severe attacks compared to those with mild attacks for whom high dose steroid therapy is usually sufficient. Used drugs for the prevention of NMOSD relapse include prednisolone, azathioprine, mycophenolate mofetil (MMF), and rituximab [6]. Due to the high cost of rituximab and MMF, azathioprine is the only drug included on the National Drug List of Essential Medicine (NLEM) for the prevention of NMOSD relapses in Thailand. The main objective of this study was to evaluate the cost effectiveness of rituximab and MMF in the treatment of NMOSD patients.

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