基因治疗药物监管模式的国际经验与中国探索  

International experience and Chinese exploration of regulatory model of gene therapy drugs

作　　者：关晓岩 常翰玉 王美琪 梁广盛 袁妮[1] GUAN Xiao-yan;CHANG Han-yu;WANG Mei-qi;LIANG Guang-sheng;YUAN Ni(School of Public Health,Dalian Medical University,Dalian LIAONING 116044,China;Department of Medical Insurance,the First Hospital of Lanzhou University,Lanzhou GANSU 730013,China;The International Peace Maternity and Child Health Hospital,SHANGHAI 200030,China)

机构地区：[1]大连医科大学公共卫生学院,辽宁大连116044 [2]兰州大学第一医院医保管理处,甘肃兰州730013 [3]中国福利会国际和平妇幼保健院,上海200030

出　　处：《中国新药与临床杂志》2024年第7期523-528,共6页Chinese Journal of New Drugs and Clinical Remedies

基　　金：2022年度辽宁省教育厅基本科研项目(LJKMR20221296)。

摘　　要：作为一种新的治疗方式,基因治疗药物(GTDs)在发挥治疗作用的同时也存在诸多风险,这给GTDs的监管带来了挑战。对此,美国和欧盟均在监管主体、监管依据和上市监管方面建立了比较完善的体系,值得我国借鉴。我国对于GTDs的监管还存在着监管依据层次不够细致、监管内容不够成熟透明、监管执行主体和责任主体还不够清晰等问题。建议我国可借鉴美国和欧盟的先进经验,对GTDs的风险进行划分和控制,组建专家技术团队,成立咨询委员会,加强国际沟通和交流,从而完善我国GTDs监管体系,提高此类药物的安全性。As a new therapeutic modality,gene therapy drugs(GTDs)play a therapeutic role and also have many risks,which brings challenges to the regulation of GTDs.The United States(US)and the European Union(EU)have established a relatively perfect system in terms of the regulatory body,regulatory basis and listing regulation,which is worthy of China's reference.China's regulation of GTDs still exists in the regulatory basis level is not detailed,the regulatory content is not mature and transparent,the main body of the regulatory implementation and the body of the responsibility is not clear enough and other issues.It is suggested that our country can learn from the advanced experience of the US and the EU,divide and control the risk of GTDs,set up an expert technical team,establish an advisory committee,and strengthen international communication and exchange,so as to improve the regulatory system of GTDs and enhance the safety of such drugs in China.

关 键 词：基因治疗 监管 罕见病 生物制品 新药临床试验申请 

分 类 号：R95[医药卫生—药学]