欧美细胞治疗产品上市后研究的经验及对我国的启示

Post-market research of cell therapy products in Europe and America and their implications for China

细胞治疗产品作为一种活细胞药物,不仅制备技术复杂,在体内的作用具有一定不可控性,而且某些作用机制尚未研究透彻,所以在疗效显著的同时也存在很大的风险,因此上市后研究十分重要。本文就美国和欧盟对细胞治疗产品上市后研究的一般性要求和特殊性要求进行介绍,并以一款嵌合抗原受体T细胞(CAR-T)细胞治疗产品为例,分析其上市后研究的实施情况,再结合我国现状,总结出欧美先进管理经验对我国的启示。建议在完善药品上市后研究的一般性管理框架的基础之上,注重细胞治疗产品的特殊性,增加具有针对性的要求,并贯彻风险管理的要求,实行分级分类管理,加强细胞治疗产品上市后的持续管理。

As a living cell medicine,cell therapy products have not only complicated preparation technology,but also some uncontrollable effect in the body,whose mechanism of action remains unclear.Despite its outstanding curative effect,possible risks exist.Therefore,post-market research is very important.This article introduced both the general requirements and specific requirements for the post-market research of cell therapy products in the United States and the European Union,and used a chimeric antigen receptor T cell(CAR-T)product as an example to analyze the implementation of its post-market research.Referring to the current situation in China,we summed up management experience of Europe and America.Besides improving the general management framework for postmarket research of drugs,we should pay attention to the particularity of cell therapy products and targeted requirements.Hierarchical and classified risk management is needed to strengthen the postmarket research of cell therapy products.