我国干细胞药物注册临床试验进展与安全风险管理

Progress and safety risk management of registration-based clinical trials of stem cell drugs in China

摘要

目的: 本文旨在为干细胞药物临床试验的动态监管及提升药物警戒工作水平提供参考。方法: 基于国家药品监督管理局药品审评中心的药物临床试验登记与信息公示平台与药物警戒数据库,对国内干细胞药物注册临床试验进展和可疑且非预期严重不良反应(suspected unexpected serious adverse reaction,SUSAR)个例安全性报告进行回顾性分析。结果: 截至2025年6月30日,国内共批准干细胞新药临床试验申请(IND)134项,登记平台有113项注册临床试验。2021年1月—2025年6月,共收到29例境内SUSAR报告,不良反应以全身性疾病及给药部位反应、呼吸相关疾病等为主。结论: 我国干细胞药物注册临床试验近年来呈现稳步发展态势,持续关注干细胞长期潜在安全风险,为临床试验的安全性保驾护航。

Abstract

Objective: This article aims to provide a reference for the dynamic regulatory oversight of clinical trials of stem cell drugs and the enhancement of pharmacovigilance practices. Methods: Using data from the Drug Clinical Trial Registration and Information Disclosure Platform and the Pharmacovigilance Database of the Center for Drug Evaluation of the National Medical Products Administration, we conducted a retrospective analysis of the progress of domestic registered clinical trials for stem cell drugs and of individual case safety reports on suspected unexpected serious adverse reactions (SUSAR). Results: As of June 30, 2025, a total of 134 Investigational New Drug (IND) applications for stem cell drugs have been approved in China, and 113 registered clinical trials are recorded on the registration platform. From January 2021 to June 2025, a total of 29 domestic SUSAR reports were received, with adverse reactions mainly involving systemic disorders and administration site reactions, as well as respiratory disorders. Conclusion: Registered clinical trials of stem cell drugs in China have shown a steady development trend in recent years. Sustained attention must be paid to the long-term potential safety risks of stem cells to safeguard the safety of clinical trials.

关键词

干细胞 / 药物注册临床试验 / 可疑且非预期严重不良反应 / 安全风险

Key words

stem cells / drug registration clinical trials / suspected unexpected serious adverse reactions / safety risk

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