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·药事管理·

欧盟先进疗法医药产品的医院豁免条款研究及对我国的启示
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王艺霏 ，谢金平 ，邵 蓉（中国药科大学药品监管科学研究院/国家药品监督管理局药品监管创新与评价重点
实验室，南京 211198）

中图分类号 R95 文献标志码 A 文章编号 1001-0408（2024）11-1285-06
DOI 10.6039/j.issn.1001-0408.2024.11.01

摘 要 目的 研究欧盟先进疗法医药产品的医院豁免条款特点，为完善我国细胞和基因治疗产品的监管体系提供启示与建议。
方法 通过文献研究及检索欧盟各成员国官网，从“非常规”定义、生产质量标准、药物警戒要求方面比较各成员国适用和实施医院
豁免条款的差异，分析医院豁免条款在实践中可能存在的问题，并结合我国细胞和基因治疗产品的监管现状提出政策建议。结果
与结论 欧盟医院豁免条款为罕见病、缺乏有效治疗或更好治疗方案的患者提供了获得新治疗方法的机会，有效提高了患者用药
的可及性。但欧盟各成员国针对医院豁免条款的规定存在一定差异，譬如部分成员国未明确界定“非常规”情形，各个成员国在生
产质量标准及药物警戒要求方面不同。医院豁免条款在实施过程中也存在信息透明度不高、缺乏一定的限制条件等问题。结合
我国细胞和基因治疗产业的发展及监管现状，建议我国在“非常规”情形下探索低风险细胞和基因治疗方法的临床转化应用，并从
生产质量标准、药物警戒要求等方面加强对临床转化应用疗法的管理；应当进一步规范研究者发起的临床研究，并注意平衡临床
应用与药品注册上市的关系，引导我国细胞和基因治疗产业持续健康发展。
关键词 先进疗法医药产品；医院豁免条款；细胞和基因治疗；研究者发起的临床研究

Study on hospital exemption clause of advanced therapy medicinal products in EU and its enlightenment to
China
WANG Yifei，XIE Jinping，SHAO Rong（Institute of Regulatory Science for Medical Products， China
Pharmaceutical University/NMPA Key Laboratory for Drug Regulatory Innovation and Evaluation， Nanjing
211198， China）

ABSTRACT OBJECTIVE To study the hospital exemption clause of advanced therapy medicinal products in the EU， and to
provide policy recommendations for improving the regulatory system of cell and gene therapy （CGT） products in China.
METHODS Through literature review and investigation of the official websites of EU member states， this study compared the
differences in the application and implementation of the hospital exemption clause among member states from the perspectives of
“non-conventional” definition， manufacturing standards， and pharmacovigilance requirements； the potential issues of hospital
exemption clauses in practice were analyzed to propose policy recommendations based on the regulatory status of CGT in China.
RESULTS & CONCLUSIONS EU has provided patients with rare diseases， who lack effective treatment or better therapy plans，
with the opportunity to obtain new treatments through the hospital exemption clause， which has effectively improved the
accessibility of medicines for patients. However， there still are certain disparities in the provisions of hospital exemption clause
among EU member states. For instance， some member states have not explicitly defined “unconventional” circumstances； each
member state has different requirements regarding production quality standards and pharmacovigilance requirement. Additionally， in
the practical implementation of hospital exemption clause， issues such as poor transparency of information and a lack of certain
restrictive conditions persist. Therefore， considering the current landscape and regulation of China’s CGT， it is recommended that
China explore the clinical translational application of low-risk CGT in “unconventional” situations， strengthen the management of
clinical translational application in terms of production quality standards and pharmacovigilance requirement. At the same time， it is
necessary to further standardize the investigator initiated trials， and pay attention to the balance between clinical application and
drug registration and marketing， thereby guiding the sustained and healthy development of China’s CGT.
KEYWORDS advanced therapy medicinal products； hospital exemption clause； cell and gene therapy； investigator initiated trials

Δ 基金项目 中国药品监督管理研究会研究课题（No.2023-Y-
近年来，细胞和基因治疗（cell and gene therapy，
Y-001）
CGT）已成为生命科学研究的前沿方向，是未来医学的
＊第一作者 硕士研究生。研究方向：医药政策与法规。E-mail：
wyf18041292276@163.com 发展趋势，且CGT产品具有技术迭代快、创新潜力巨大、
[1]
产品作用机制复杂等特点 。目前，全球已有多款 CGT
# 通信作者 讲师，硕士生导师，博士。研究方向：医药政策与法
规。E-mail：15151870834@163.com 产品上市，有关 CGT 的临床研究数量也呈爆发式增长。

中国药房 2024年第35卷第11期 China Pharmacy 2024 Vol. 35 No. 11 · 1285 ·