Study on hospital exemption clause of advanced therapy medicinal products in EU and its enlightenment to China

WANG Yifei; XIE Jinping; SHAO Rong

doi:10.6039/j.issn.1001-0408.2024.11.01

您当前的位置：

首页 >

文章列表页 >

Study on hospital exemption clause of advanced therapy medicinal products in EU and its enlightenment to China

更新时间：2025-09-26

- Study on hospital exemption clause of advanced therapy medicinal products in EU and its enlightenment to China
- ZHONGGUO YAOFANG Vol. 35, Issue 11, Pages: 1285-1290(2024)
- 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：
- 基金信息：
- DOI：10.6039/j.issn.1001-0408.2024.11.01
  CLC： R95
- Received：05 January 2024，
  
  Revised：2024-04-30，
  
  Accepted：05 May 2024，
  
  Published：15 June 2024
- 稿件说明：
移动端阅览
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[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[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

Keywords

references

高建超，韦薇，张旻，等 . 细胞和基因治疗产品监管科学研究进展和展望 [J ] . 中国新药杂志， 2022 ， 31 （ 2 ）： 105 - 108 .

GAO J C ， WEI W ， ZHANG M ， et al . Progress and pro- spect of regulatory science in cell and gene therapy pro- ducts [J ] . Chin J N Drugs ， 2022 ， 31 （ 2 ）： 105 - 108 .

虞淦军，吴艳峰，汪珂，等 . 国际细胞和基因治疗制品监管比较及对我国的启示 [J ] . 中国食品药品监管， 2019 （ 8 ）： 4 - 19 .

YU G J ， WU Y F ， WANG K ， et al . Comparison of international supervision of cell and gene therapy products and its enlightenment to China [J ] . China Food Drug Adm Mag ， 2019 （ 8 ）： 4 - 19 .

陈云，邹宜諠，邵蓉，等 . 美国干细胞产业发展政策与监管及对我国的启示 [J ] . 中国医药工业杂志， 2018 ， 49 （ 12 ）： 1733 - 1741 .

CHEN Y ， ZOU Y X ， SHAO R ， et al . The research and enlightenment about development policy and regulation of American stem cell industry [J ] . Chin J Pharm ， 2018 ， 49 （ 12 ）： 1733 - 1741 .

王晶，鲁爽 . 美国和欧洲细胞和基因治疗产品加快审评审批政策概况及对我国的启示 [J ] . 中国新药杂志， 2023 ， 32 （ 24 ）： 2441 - 2446 .

WANG J ， LU S . Overview of accelerated assessment and approval policies for cell and gene therapy products in the United States and Europe and its implications for China [J ] . Chin J N Drugs ， 2023 ， 32 （ 24 ）： 2441 - 2446 .

European Medicines Agency . How the committees work [EB/OL ] .[ 2023-12-20 ] . https：//www.ema.europa.eu/en/committees/how-committees-work https://www.ema.europa.eu/en/committees/how-committees-work .

PIGNATTI F ， ARONSSON B ， GATE N ， et al . The review of drug applications submitted to the European Medicines Evaluation Agency：frequently raised objections，and outcome [J ] . Eur J Clin Pharmacol ， 2002 ， 58 （ 9 ）： 573 - 580 .

任晓星，史录文 . 中美欧新药上市加快审评审批政策研究 [J ] . 中国新药杂志， 2020 ， 29 （ 9 ）： 961 - 971 .

REN X X ， SHI L W . A policy study on NDA/BLA expedited pathways in China，US and EU [J ] . Chin J N Drugs ， 2020 ， 29 （ 9 ）： 961 - 971 .

Cuende N ， Ciccocioppo R ， Forte M ， et al . Patient access to and ethical considerations of the application of the European Union hospital exemption rule for advanced therapy medicinal products [J ] . Cytotherapy ， 2022 ， 24 （ 7 ）： 686 - 690 .

Coppens D G M ， Gardarsdottir H ， Bruin M L D ， et al . Regulating advanced therapy medicinal products through the hospital exemption：an analysis of regulatory approaches in nine EU countries [J ] . Regen Med ， 2020 ， 15 （ 8 ）： 2015 - 2028 .

Hills A ， Awigena-Cook J ， Genenz K ， et al . An assessment of the hospital exemption landscape across European Member States：regulatory frameworks，use and impact [J ] . Cytotherapy ， 2020 ， 22 （ 12 ）： 772 - 779.e1 .

European Parliament ， Council of the European Union . Regulation （EC） No 726/2004 of the European Parliament and of the Council of 31 March 2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency [EB/OL ] .（ 2022-01-28 ）[ 2023-12-20 ] . https：//eur-lex.europa.eu/eli/reg/2004/726/oj https://eur-lex.europa.eu/eli/reg/2004/726/oj .

陈云，李珊珊，邵蓉，等 . 欧洲前沿疗法药物的监管、审批及其启示 [J ] . 中国新药杂志， 2020 ， 29 （ 15 ）： 1681 - 1690 .

CHEN Y ， LI S S ， SHAO R ， et al . Supervision and approval of advanced therapy medicinal products in European and its inspirations [J ] . Chin J N Drugs ， 2020 ， 29 （ 15 ）： 1681 - 1690 .

AEMPS . Autorizaciones de uso de medicamentos de terapia avanzada [EB/OL ] .[ 2023-12-20 ] . https：//www.aemps.gob.es/medicamentos-de-uso-humano/terapias-avanzadas/autorizaciones-de-uso-de-medicamentos-de-terapia-avan-zada/?lang=en#aemps https://www.aemps.gob.es/medicamentos-de-uso-humano/terapias-avanzadas/autorizaciones-de-uso-de-medicamentos-de-terapia-avan-zada/?lang=en#aemps .

王广基，王越，李洁，等 . 国外细胞治疗产品监管体系介绍及对我国的启示 [J ] . 中国食品药品监管， 2023 （ 9 ）： 6 - 13 ， 166 - 167 .

WANG G J ， WANG Y ， LI J ， et al . Introduction to the regulatory system of cell therapy products abroad and its enlightenment for China [J ] . China Food Drug Adm Mag ， 2023 （ 9 ）： 6 - 13 ， 166 - 167 .

白桦，张抒扬 . 研究者发起的临床研究用于支持新药研发面临的挑战 [J ] . 中国肺癌杂志， 2022 ， 25 （ 7 ）： 511 - 516 .

BAI H ， ZHANG S Y . Challenges of investigator-initiated clinical trials to support the new drug development [J ] . Chin J Lung Cancer ， 2022 ， 25 （ 7 ）： 511 - 516 .

李会娟，苑杰，武阳丰 . 研究者发起的临床研究中常见伦理问题及监管考量 [J ] . 医学与哲学， 2022 ， 43 （ 7 ）： 6 - 10 .

LI H J ， YUAN J ， WU Y F . Common ethical issues in investigator-initiated clinical studies and regulatory considerationst [J ] . Medicine & Philosophy ， 2022 ， 43 （ 7 ）： 6 - 10 .

Views

380

下载量

CSCD

Alert me when the article has been cited

提交

Tools

Publicity Resources

No data

Related Author

No data

Related Institution

No data

⁰