According to the World Health Organization, more than 300 million people suffer from rare diseases across the globe and 50 percent of them are children. China is home to some 20 million people diagnosed with rare diseases. Because of the low incidence rate to population, there is little clinical research, and due to the lack of effective diagnosis and treatment, rare diseases have become a chronic problem. In recent years, China has drafted a catalog of rare diseases, established a coordination network for diagnosis and treatment, adjusted the medicine supply mechanism, simplified imported drug approvals, and encouraged R&D at domestic pharmaceutical enterprises. More rare disease medications have been added to the list of medical insurance reimbursement so patients can live in dignity.