欧盟罕见病用药审评审批分析及其对我国的启示.

This study systematically summarizes the drug policy for rare diseases in the European Union, statistically analyzes the review and approval of drugs for rare diseases under the regulatory framework of the European Medicines Agency, so as to provide reference for the research and development of drugs for rare diseases and the formulation of related policy in China by comparing with the relevant situation in China. At present, there are 141 kinds of drugs for rare disease approved for market use in the European Union, covering 107 rare disease indications. The marketing approval of drugs for rare diseases in the European Union takes a long time and is cautious, with an average of 5. 67 years for the identification and marketing of drugs. Compared with the European Union, there is still a big gap in the definition and coverage of the catalog of rare diseases, the policy to encourage research and development and marketing, the total number of priority review and approval of drugs for rare diseases and the coverage of indications in China, especially in the innovative research and development of drugs for rare diseases. It is necessary to further improve relevant policies, establish an innovation support system, strengthen the protection of the rights and interests of researchers and developers, promote the innovative development of the drug industry for rare diseases, and better meet the drug protection needs of patients with rare diseases in China. [ABSTRACT FROM AUTHOR]