The Enlightenment of Foreign Drug Security System for Rare Diseases to China / 中国药学杂志

ABSTRACT

OBJECTIVE: To explore the suggestions for the development of the medical security system and the medication for rare diseases (orphan drugs) in China and analyze the current progress of orphan drugs in China as well as other developed countries. METHODS: Based on literature search, the policies related with orphan drugs well as the medical security policies for patients with rare diseases in China and abroad were analyzed and summarized. RESULTS: Through analysis, it was found that the definition, regulation, and the research and development for orphan drugs started early in the developed countries and had made good progress. At the same time, they have relatively sound medical security system for rare diseases. At present,China has not established a complete policy, regulation and medical security system for rare disease, nor has an official definition of rare diseases. However, in recent years, China's policies on the research and approval of rare diseases and as well as medical serurity are improving day by day. CONCLUSION: Although China has not yet established a complete and perfect policy system for rare diseases, it has already made a series of policies according to our specific national conditions, which narrowed the gap between China and other developed countries. Our country should learn from foreign experience and consider the actual situation of our country, further complete the policies for rare diseases, and improve the diagnosis and treatment of patients with rare diseases.