Suggestions on the development of rare disease drugs in China / 国际药学研究杂志

ABSTRACT

The accessibility of orphan drugs has been the focus of many countries. Many countries and regions have established special laws and regulations to promote the development of orphan drugs since 1980’s. The development of orphan drugs has now become to be an important strategy for the drug development of many large pharmaceutical companies. At present, China has not yet defined the rare diseases, and the orphan drugs used in China are almost dependent on imports. The domestic situation that the research and development, introduction, production and sales of orphan drugs lack the policy support has been the bottleneck restricting the development of related industries in China. This paper reviews the present status of worldwide development of the rare diseases and their therapeutic drugs, and put forward related suggestions for the development of orphan drugs in China.