Abstract
BACKGROUND: Neuromyelitis optica spectrum disorder (NMOSD) is a rare and debilitating disease that has become more widely recognized in China. Legislative measures have been implemented by the government to improve treatment access for rare diseases. OBJECTIVES: To investigate the diagnostic journey, treatment status, treatment accessibility, and treatment satisfaction of the NMOSD patients on disease-modifying therapies (DMTs) in China. DESIGN: A patient online survey. METHODS: This cross-sectional online survey was conducted between November 2022 and January 2023. Patients over 18 years old and diagnosed with NMOSD were included. The questionnaire consisted of five sections covering demographics, diagnostic and treatment experiences, DMTs availability, cost and affordability, and treatment satisfaction using the Treatment Satisfaction Questionnaire for Medication (version II). Patient opinions and demands were also collected at the end of the survey. RESULTS: A total of 375 patients diagnosed with NMOSD were recruited, of which 321 patients used DMTs. It required 1.22 ± 3.22 years and 3.58 ± 4.24 hospital visits for a definitive diagnosis. One-third of the patients still needed to travel for over 2 h to access DMTs. The total treatment expenditure was estimated to be CNY 59,827.00 (USD 8315.95) a year. Drug expenses alone accounted for 52.22% of the average annual household income. The most common challenges perceived were the inability to afford treatment and a lack of effective options. No significant difference was found in treatment satisfaction among DMTs, except that rituximab scored lowest in convenience compared to other DMTs. Patients' age and travel time required to obtain medications were negatively associated with global treatment satisfaction. CONCLUSION: In China, patients with NMOSD face challenges in obtaining proper treatment due to diagnostic difficulties, distant medication access, and high costs. Policies should prioritize improving disease education and alleviating financial burdens for the patients.