中国药房2025，Vol.36Issue(17)：2097-2101,5.DOI:10.6039/j.issn.1001-0408.2025.17.03
我国罕见病药品进口现状及挑战的调研分析
Investigation and analysis of the current status and challenges in importing rare disease drugs in China
摘要
Abstract
OBJECTIVE To analyze the current status and challenges in importing rare disease drugs in China,providing references for optimizing the import process and improving relevant policies.METHODS Questionnaires and interviews were conducted with stakeholders involved in rare disease drug importation,including government departments,multinational pharmaceutical enterprises,healthcare institutions,and patient organizations.This explored the current situation and challenges encountered by each party.Expert opinions were synthesized to propose improvement suggestions.RESULTS A questionnaire survey of representatives from 25 multinational pharmaceutical companies in the rare disease field revealed that these companies had a strong willingness to import rare disease drugs,with 58.33%of them practicing diverse import models.However,significant challenges hindered this process,including unclear regulations(54.17%),complex approval procedures(45.83%),and excessively long approval cycles(41.67%),negatively impacting their motivation.Meanwhile,interviews with 13 experts from government departments,healthcare institutions,pharmaceutical enterprises,and patient organizations identified deficiencies in policy design,approval processes,sampling inspection costs,and communication efficiency with regulators.Additionally,the drug import model in special medical zones also required improvement.CONCLUSIONS The importation of rare disease drugs in China faces challenges such as incomplete policies,inflexible regulatory mechanisms,and insufficient communication channels.It is recommended to enhance the rare disease definition criteria,optimize import incentive policies,and refine regulatory models,so as to further optimize the import process of rare disease drugs and improve relevant policies.关键词
罕见病/药品进口/政策建议/中国Key words
rare disease/drug importation/policy recommendations/China分类
医药卫生引用本文复制引用
邬镜净,苏沁凝,陶雪怡,杨雨菲,茅宁莹..我国罕见病药品进口现状及挑战的调研分析[J].中国药房,2025,36(17):2097-2101,5.基金项目
国家社会科学基金重大项目(No.15ZDB167) （No.15ZDB167）
江苏省社会科学基金一般专项资助项目(No.22JZB002) （No.22JZB002）
2024年江苏省研究生科研创新计划项目(No.KYCX24_1050) （No.KYCX24_1050）
