《药品管理法》颁布四十年来我国罕见病用药审评审批的改革成效  

Effectiveness of Drug Review and Approval for Rare Diseases in China Over Four Decades Since the Promulgation of Drug Administration Law

作　　者：邵蓉[1] 谢金平 SHAO Rong;XIE Jinping(NMPA Key Laboratory for Drug Regulatory Innovation and Evaluation,Institute of Regulatory Science for Medical Products,China Pharmaceutical University,Nanjing 211198,China)

机构地区：[1]中国药科大学药品监管科学研究院,国家药品监督管理局药品监管创新与评价重点实验室,南京211198

出　　处：《中国药学杂志》2024年第18期1679-1685,共7页Chinese Pharmaceutical Journal

摘　　要：自1984年我国颁布第一部《中华人民共和国药品管理法》(以下简称《药品管理法》)以来,我国药品管理进入了法治化、规范化阶段。药品监管部门持续加强药品监管法律法规体系建设,不断优化药品审评审批制度。罕见病用药可及性是全球面临的一项重大挑战。为满足罕见病患者的用药需求,我国出台了一系列激励政策,全面支持罕见病用药研发、加快罕见病用药审评审批。本文系统回顾了我国罕见病用药审评审批的历史演变、发展进程、改革成效,并展望未来,以期进一步优化我国罕见病用药审评审批政策,更好地满足患者的用药需求。Since the enactment of China′s first“Drug Administration Law of the People′s Republic of China”in 1984,China has entered a phase of legal and standardized drug management.The drug regulatory authorities have continuously strengthened the construction of the legal and regulatory framework for drug supervision and have optimized the drug review and approval system.The accessibility of drugs for rare diseases is a significant challenge worldwide.To meet the medication needs of patients with rare diseases,China has introduced a series of incentive policies to fully support the development of drugs for rare diseases and accelerate their review and approval.This paper systematically reviews the historical evolution,development process,and reform achievements of China′s rare disease drug review and approval system,and looks ahead to the future,aiming to further optimize the system to better meet the medication needs of patients.

关 键 词：罕见病用药 审评审批 改革成效 药品监管 药品管理法 

分 类 号：R951[医药卫生—药学]