细胞治疗产品上市后长期安全性监测的国际经验与中国优化路径

doi:10.19803/j.1672-8629.20260390

摘要/Abstract

摘要： 目的梳理国内外细胞治疗产品监管现状,针对性提出对我国目前细胞治疗产品监管现状的优化建议。方法分析美国、欧盟、日本、韩国、中国细胞治疗产品监管相关文献及政策文件,结合我国基本国情,探索可应用于细胞治疗产品长期安全性监测的优化方案。结果截至2026年3月,我国已批准上市8款CAR-T治疗产品和1款干细胞产品;美国食品药品监督管理局已批准上市细胞与基因治疗产品49款;欧洲药品管理局已批准上市28款细胞治疗产品。美国、欧盟、日本、韩国均设立专门的细胞治疗产品监管部门与风险管理制度;我国目前实施“双轨制”监管模式。结论建议我国完善顶层法规与风险分级制度、强化药品上市许可持有人与医疗机构双轨主体责任、推进人工智能驱动的精准药物警戒体系、健全长期随访知情同意与隐私保护四大优化方向,进一步完善我国细胞治疗产品上市后长期安全性监测。

关键词: 细胞治疗产品, 药品不良反应, 上市后监测, 药物警戒, 安全性监测

Abstract: Objective To investigate the way cell therapy products are currently regulated globally and to offer evidence-based recommendations for optimizing China's post-marketing long-term safety surveillance framework. Methods Related literature about and guidelines for the regulation of cell therapy products in the United States, the European Union, Japan, South Korea, and China were summarized. Based on the realities in China, feasible optimization strategies for long-term safety surveillance of cell therapy products were proposed. Results As of March 2026, eight chimeric antigen receptor T-cell (CAR-T) therapies and one stem cell product had been approved for marketing in China, compared with 49 cell and gene therapy products by the U.S. FDA and 28 cell therapy products by the European Medicines Agency (EMA). A comparative analysis of the current regulatory frameworks revealed that the United States, the European Union, Japan, and the Republic of Korea had all established specialized regulatory bodies and risk-based management systems, characterized by well-defined regulatory mechanisms and detailed guidelines. China had implemented a “dual-track” regulatory model. Conclusion We recommend a four-pronged strategic framework for optimizing China's post-marketing long-term safety surveillance system by updating the top legislation and establishing a risk-stratified monitoring system, reinforcing the dual-track responsibilities of marketing authorization holders (MAHs) and medical institutions, boosting an AI-empowered precision pharmacovigilance paradigm, and enhancing ethical governance frameworks for long-term follow-up informed consent and patient privacy protection. China needs to consolidate its long-term safety surveillance infrastructure for cell therapy products.

Key words: Cell Therapy Products, Adverse Drug Reaction, Post-Marketing Surveillance, Pharmacovigilance, Safety Surveillance

中图分类号:

R951
R994.11

冯思琪, 倪雨晨, 胡冯译, 刘玥, 叶小飞, 郭晓晶. 细胞治疗产品上市后长期安全性监测的国际经验与中国优化路径[J]. 中国药物警戒, 2026, 23(9): 975-981.

Feng Siqi, Ni Yuchen, Hu Fengyi, Liu Yue, Ye Xiaofei, Guo Xiaojing. Long-term post-marketing safety surveillance of cell therapy products: international experience and optimization pathways in China[J]. Chinese Journal of Pharmacovigilance, 2026, 23(9): 975-981.

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https://zgywjj.magtechjournal.com/CN/Y2026/V23/I9/975

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