我国罕见病药品审评审批现状分析
Current situation and analysis of the review and approval for orphan drugs in China
曹永芳 1唐湘燕 1武铮 1周刚1
作者信息
- 1. 国家药品监督管理局药品审评中心,北京 100076
- 折叠
摘要
罕见病是全人类面临的重大医学挑战.目前全球范围内已知的罕见病有7000～10000 种,中国罕见病患者数量众多,大多数患者面临着无药可治或者境外有药、境内无药的问题.药品审评审批制度改革以来,我国从政策文件、法律规范、技术标准等维度规范了罕见病范围、罕见病药品加快审评途径、技术标准等内容,加快罕见病药品上市,以求不断满足罕见病患者用药需求.通过对我国罕见病药品审评审批制度的分析,建议从罕见病药品认定标准、设立专门机构、建立市场独占期制度、加大研发指导等方面完善罕见病药品审评审批制度.
Abstract
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.
关键词
罕见病/罕见病药品/审评审批制度改革Key words
rare diseases/orphan drugs/reform of the review and approval system引用本文复制引用
出版年
2024
NETL
NSTL
万方数据