关键词: 细胞和基因治疗, 主动监测, 医院集中监测, 精准药物警戒, 嵌合抗原受体 T 细胞

Abstract: Objective To analyze the current monitoring practices at home and abroad,and to explore new approaches to pharmacovigilance for cell and gene therapy products. Methods By taking CAR-T cell therapy products, the domestic and overseas data on monitoring of these products, information about safety, and related technological guidelines were collected to assess the safety profile of the related drugs. The strengths, weaknesses and characteristics of passive monitoring and active monitoring, spontaneous reporting, and of intensive hospital monitoring were elaborated. Novel approaches to pharmacovigilance that targeted cell and gene therapy products were explored. Results Cell and gene therapy products had distinct features in terms of mechanisms of action, in vivo metabolism, therapeutic effects, and adverse reactions, which is why specific pharmacovigilance was required. Conclusion This study has explored a precision pharmacovigilance model that can not only combine passive monitoring with active monitoring and spontaneous reporting of adverse reactions with intensive hospital monitoring, but also identify risks of products. This study is expected to find a new means for pharmacovigilance for post-marketing monitoring of safety of cell and gene therapy products in China.

Key words: Cell and Gene Therapy, Active Monitoring, Intensive Hospital Monitoring, Precision Pharmacovigilance, Chimeric Antigen Receptor T Cells(CAR-T)