Presentation (CTI)

OBJECTIVES: The high cost of orphan drugs challenges healthcare systems. This study aimed to quantify the preferences of rare disease patients and caregivers for orphan drug reimbursement criteria in China.
METHODS: A discrete choice experiment was conducted among rare disease patients and caregivers in China between October 2024 and September 2025. Six attributes were included: untreated life expectancy, untreated quality of life, health gain, catastrophic health expenditure, availability of alternative treatments, and increase in health insurance financing. Choice data were analyzed using conditional logit and mixed logit models. Relative importance and scenario analyses were performed to evaluate the contribution of each attribute. Preference heterogeneity was explored using latent class analysis and subgroup analyses.
RESULTS: A total of 312 respondents were included. The mixed logit model provided a better fit than the conditional logit model and revealed significant preference heterogeneity. Respondents preferred reimbursing treatments for diseases with greater reductions in life expectancy, poorer untreated quality of life, larger health gains, catastrophic expenditure risk, and no available alternative treatments. In contrast, increases in health insurance financing were not significantly associated with reimbursement preferences. Relative importance analysis showed that health gain was the most influential attribute (41.8%), followed by untreated life expectancy (18.7%), catastrophic health expenditure (14.0%), availability of alternative treatments (12.2%), and untreated quality of life (11.8%). Scenario analysis indicated that increasing health gain from 0.01 to 4 QALYs increased the predicted probability of reimbursement selection to 83.4%. Latent class analysis identified two distinct preference groups: one focused on treatment benefits, another on effectiveness, disease severity, unmet need, and financial burden. Preference heterogeneity was observed across risk attitude, respondent status and reimbursement status.
CONCLUSIONS: Patients and caregivers value multiple social considerations in orphan drug reimbursement. Significant preference heterogeneity supports incorporating diverse stakeholder perspectives into policy to enhance transparency and patient-centeredness in China.