Abstract: According to the status-quo of orphan drug access in China, this paper constructedan index evaluation system so as to provide referable foundationfor establishing and improving orphan drug authorization system. Study results indicated that the less-improved orphan drug marketing authorization system, poorer orphan drug availability, supply capacity and affordability compared with developed countries and regions severely affected patients′ timely medication and hindered orphan drug industry development. With reference to foreign countries, it is urgent to improve orphan drugs marketing authorization system for the purpose of encouraging orphan drug R&D and improving the health benefits of patients.