Current situation and analysis of the review and approval for orphan drugs in China
Rare diseases are a major medical challenge for humanity.Currently,there are 7000 to 10 000 known rare diseases worldwide.In China,there are a large number of patients with rare diseases,most of whom face the problem of limited medicine to use.Since the reform of the drug review and approval system,China has regulated the scope of rare diseases,accelerated review pathways for rare disease drugs,technical standards and other aspects through policy documents,legal norms and technical standards.This has accelerated the marketing of drugs treating rare diseases and continuously met the drug needs of patients with rare diseases.By analyzing China's review and approval system of rare disease drug,it is suggested to improve the system from the aspects such as the identification criteria for drugs treating rare disease,the establishment of specialized institutions,the building of a market exclusivity period system and increased research and development guidance.
rare diseasesorphan drugsreform of the review and approval system
NETL
NSTL
万方数据
