The development status and path of rare disease treatment and orphan drugs in China / 中国药理学通报

ABSTRACT

Rare diseases are a group of diseases with low incidence, complex conditions, difficulty in diagnosis and poor treatment accessibility. With the growing attention on rare diseases in China in recent years, relevant policies are gradually improved, rapid progress have been made in the treatment of rare diseases and the development of orphan drugs, but there are still challenges. Based on the development status of rare disease treatment and orphan drugs, this paper elaborates on the development path of rare disease diagnosis, and provides reference for the rare disease treatment, orphan drug development and policy formation in China.