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Patient preferences and willingness-to-pay for therapy in generalized myasthenia gravis: a large-scale discrete
Yafang Xu1, Ling Feng2, Mingjie Zhang1
1Huashan Rare Disease Center and Department of Nursing, Huashan Hospital, National Center for Neurological Disorders, Fudan University, Shanghai, China.
Background:
Generalized myasthenia gravis (gMG) is a rare, chronic autoimmune disorder that imposes a substantial disease burden in China. As novel therapeutic options emerge, understanding patient preferences has become essential for treatment decision-making. This study aimed to assess treatment preferences and willingness to pay (WTP) among Chinese gMG patients.
Methods:
This multicenter, cross-sectional survey was conducted from March to August 2025 among patients with gMG in China. A discrete choice experiment was used to quantify patient preferences by presenting hypothetical treatment scenarios defined by eight key attributes. A mixed Logit regression model was used to identify preference drivers, and WTP was estimated to reflect the monetary value assigned to changes in treatment attributes.
Results:
Among the 909 analyzed patients, the mean age was 48.9 ± 13.27 years, and 61.8% were female. Within the selected attribute sets, safety was a key determinant of patient preferences, with consistently favored for lower risks of adverse drug reactions (ADRs) on metabolic diseases, infection, myelosuppression, and liver and kidney function impairment; WTP increased as risk levels decreased. Patients preferred oral administration, followed by subcutaneous injection and intravenous infusion. Infrequent dosing, once every-6-month, weekly or daily, was preferred over 2-3 times daily. Faster onset time of action (≤2 weeks) was favored, and higher out-of-pocket costs were disfavored. Subgroup analyses revealed young patients valued once weekly dosing, subcutaneous injection, and low risk of liver and kidney function impairment than old patients. Patients with high disease burden showed similar preferences for low and moderate ADRs risk. High-income patients favored low ADRs risk, infrequent dosing, subcutaneous injection, and were less sensitive to cost than to low- and medium-income patients.
Conclusion:
This large-scale study was the first to investigated treatment preferences and WTP among Chinese patients with gMG. Within the selected attribute sets, patients prioritized safety, infrequent and convenient administration route, affordability, and rapid onset of action. Patients were willing to invest financially in therapies that align with these preferences. Incorporating patient preferences in clinical decision-making and reimbursement policy may improve adherence, reduce disease burden, and enhance quality of life for individuals with gMG in China.
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