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

王艺霏; 谢金平; 邵蓉

doi:10.6039/j.issn.1001-0408.2024.11.01

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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

药事管理 | 更新时间：2024-06-11

- 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
- Study on hospital exemption clause of advanced therapy medicinal products in EU and its enlightenment to China
- 中国药房 2024年35卷第11期页码：1285-1290
- 作者机构：
  
  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
- 作者简介：
  
  硕士研究生。研究方向：医药政策与法规。E-mail：wyf18041292276@163.com
  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
- 基金信息：
  
  中国药品监督管理研究会研究课题(2023-Y-Y-001)
- DOI：10.6039/j.issn.1001-0408.2024.11.01
  中图分类号： R95
- 纸质出版日期：2024-06-15，
  
  收稿日期：2024-01-05，
  
  修回日期：2024-04-30，
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王艺霏,谢金平,邵蓉.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 ^Δ[J].中国药房,2024,35(11):1285-1290.

WANG Yifei,XIE Jinping,SHAO Rong.Study on hospital exemption clause of advanced therapy medicinal products in EU and its enlightenment to China[J].ZHONGGUO YAOFANG,2024,35(11):1285-1290.
王艺霏,谢金平,邵蓉.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 ^Δ[J].中国药房,2024,35(11):1285-1290. DOI： 10.6039/j.issn.1001-0408.2024.11.01.

WANG Yifei,XIE Jinping,SHAO Rong.Study on hospital exemption clause of advanced therapy medicinal products in EU and its enlightenment to China[J].ZHONGGUO YAOFANG,2024,35(11):1285-1290. 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。

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.

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

Keywords

hospital exemption clausecell and gene therapyinvestigator initiated trials

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