Patient Access to Medical Insurance Covered Orphan Drugs in China: Real-World Evidence From Patient Survey

•This is the first study that focused on the accessibility of orphan drugs after listing in the reimbursement formulary of China, using a unique standpoint of patients to investigate the approachability, availability, and affordability of orphan drugs.•The study provided a snapshot of the status quo of orphan drug utilization and identified the issues associated with the low availability and affordability of high-cost orphan drugs, which could provide insights to healthcare stakeholders to improve care for patients with rare disease.