美国《药物开发平台技术认定计划行业指南草案》及其对我国细胞和基因治疗监管的启示  

The Platform Technology Designation Program for Drug Development Guidance for Industry of the United States and its implications for the regulation of cell and gene therapy in China

作　　者：麻巧宁 孙潭霖 王闻雅 MA Qiao-ning;SUN Tan-lin;WANG Wen-ya(National Medical Products Administration Institute of Executive Development,Beijing 100073,China;Beijing Tsinghua Changgung Hospital,Beijing 102218,China)

机构地区：[1]国家药品监督管理局高级研修学院,北京100073 [2]清华大学附属北京清华长庚医院,北京102218

出　　处：《中国新药杂志》2025年第4期337-340,共4页Chinese Journal of New Drugs

基　　金：药品监管科学体系建设重点项目(RS2024G002);国家药品监督管理局高级研修学院院内基金资助项目(NMPAIED102024001)。

摘　　要：美国FDA最新发布的《药物开发平台技术认定计划行业指南草案》为药品开发、制造和审查流程提供了新的效率提升途径。该指南详细阐述了平台技术认定的申请条件、潜在好处、数据利用、里程碑会议讨论、申请内容和审查时间表等关键要素。该指南对中国细胞和基因治疗(cellular and gene therapy,CGT)监管具有重要启示。中国监管机构可借鉴美国经验,建立类似的平台技术认定框架,加强监管机构与企业的沟通交流,促进国际经验交流,以推动CGT行业的创新和规范发展,提高患者对先进医疗产品的可及性。The US FDA has recently issued the Platform Technology Designation Program for Drug Development Guidance for Industry,providing a new avenue for improving the efficiency of drug development,manufacturing,and review processes.The guidance elaborates on key elements such as the application conditions for platform technology designation,potential benefits,data utilization,discussions in milestone meetings,application content,and the review schedule.This guidance offers important insights for the regulation of cell and gene therapy(CGT)in China.Chinese regulatory agencies can draw on American experience to establish a similar framework for the designation of platform technology,strengthen communication between regulatory agencies and enterprises,and promote international exchange of experiences,in order to advance innovation and standardized development in the CGT industry,and enhance patient access to advanced medical products.

关 键 词：美国FDA 平台技术认定 药物开发 监管科学 细胞和基因治疗 

分 类 号：R95[医药卫生—药学]