目的:提出完善我国细胞和基因治疗(cell and gene therapy,CGT)产品监管创新路径的具体建议.方法:基于文献调研,对CGT产品的研发特点、监管需求以及我国、美国、欧盟、日本4 个国家/地区的监管创新路径进行研究;基于数据统计分析,对我国、美国、欧盟、日本 4 个国家/地区的CGT产品监管创新路径的实施效果进行对比.结果:与美国、欧盟和日本相比,我国CGT产品的监管创新路径尚需完善;我国上市的CGT产品品种较少且治疗领域单一,整体研发时间最短,但审评时间长于美国和日本.结论:建议我国明晰CGT产品的审批路径和审批标准、成立专门的CGT产品监管和审评部门、加强我国CGT产品开发的证据体系建设、建立针对性的CGT产品加快上市审批通道、引进CGT产品资格认定机制、完善我国CGT产品的上市后监管.
Research on innovative regulatory pathways for cell and gene therapy products
Objective:To propose targeted recommendations for enhancing the regulatory innovation pathways for cell and gene therapy(CGT)products in China.Methods:This study employed a comprehensive literature review to examine the research and development characteristics,regulatory requirements,and innovation pathways for CGT products in China,the United States,Europe,and Japan.Additionally,a statistical analysis was conducted to compare the implementation outcomes of regulatory innovation pathways for these products across the aforementioned regions.Results:In comparison to the United States,the European Union,and Japan,the regulatory pathways for CGT products in China requires further enhancement.The diversity of CGT products available in China is relatively limited,and the therapeutic application range is narrow.Although the overall development timeline is the shortest,the review period is longer in China than that observed in the United States and Japan.Conclusion:It is recommended that China should elucidate the approval pathways and standards for CGT products,establish a specialized department for the supervision and evaluation of CGT products,enhance the construction of an evidence-based system for CGT products development,create targeted expedited market approval programs for CGT products,implement a qualification recognition mechanism for CGT products,and strengthen post-market surveillance of CGT products in China.
cell and gene therapy productsregulatory pathwaysinnovation
NETL
NSTL
万方数据
