Analysis of the British “compassionate medicine use system” and its reference to China

SONG Yuanyuan; DU Xinyu; LI Yuchang; LIU Yuanli

doi:10.6039/j.issn.1001-0408.2024.09.01

您当前的位置：

首页 >

文章列表页 >

Analysis of the British “compassionate medicine use system” and its reference to China

更新时间：2025-09-25

- Analysis of the British “compassionate medicine use system” and its reference to China
- ZHONGGUO YAOFANG Vol. 35, Issue 9, Pages: 1029-1033(2024)
- 作者机构：
  
  中国医学科学院/北京协和医学院卫生健康管理政策学院，北京　100730
- 作者简介：
- 基金信息：
- DOI：10.6039/j.issn.1001-0408.2024.09.01
  CLC： R-013;R951
- Received：09 January 2024，
  
  Revised：2024-03-30，
  
  Accepted：01 April 2024，
  
  Published：15 May 2024
- 稿件说明：
移动端阅览
宋源源,杜昕昱,李雨畅,等.英国“同情用药制度”分析及对我国的借鉴[J].中国药房,2024,35(09):1029-1033.

SONG Yuanyuan,DU Xinyu,LI Yuchang,et al.Analysis of the British “compassionate medicine use system” and its reference to China[J].ZHONGGUO YAOFANG,2024,35(09):1029-1033.
宋源源,杜昕昱,李雨畅,等.英国“同情用药制度”分析及对我国的借鉴[J].中国药房,2024,35(09):1029-1033. DOI： 10.6039/j.issn.1001-0408.2024.09.01.

SONG Yuanyuan,DU Xinyu,LI Yuchang,et al.Analysis of the British “compassionate medicine use system” and its reference to China[J].ZHONGGUO YAOFANG,2024,35(09):1029-1033. DOI： 10.6039/j.issn.1001-0408.2024.09.01.

摘要

“同情用药制度”作为药品监管的灵活手段之一，已成为当前满足患者用药可及性的新路径。英国的“同情用药制度”，即早期药物可及性计划，通过不同政府部门间的通力合作，早期介入申请企业的药物开发过程，在保障患者及时获得创新药物的同时，也加快了药物的上市与支付报销流程。当前我国仅对“同情用药制度”作出了原则性规定，并未出台具体的实施细则。建议结合英国的制度经验尽快对我国“同情用药”的准入条件、不同主体的责任与义务、费用支付机制以及如何促进相关部门的合作等内容作出明确规定，以形成规范可操作的适合我国国情的“同情用药制度”。

Abstract

As one of the flexible means of drug supervision， the “compassionate medicine use system” has become a new way to meet the drug accessibility of patients at present. The “compassionate medicine use system” in Britain， that is， Early Access to Medicine Scheme， intervenes in the drug development process of the applicant enterprises at an early stage through the cooperation of different government departments， which not only ensures patients obtain innovative drugs in time， but also accelerates the drug listing and payment and reimbursement process. At present， China has only made principled provisions on the “compassionate medicine use system”， but has not issued specific implementation rules. It is suggested that the access conditions of “compassionate medicine use”， the responsibilities and obligations of different subjects， the payment mechanism of expenses and how to promote the cooperation of relevant departments should be clearly defined as soon as possible in combination with the system experience of Britain， so as to form a standardized and operable “compassionate medicine use system” suitable for China’s national conditions.

关键词

Keywords

references

刘晓 . 同情用药制度的国内外立法沿革及思考：兼论药品可及性相关制度的比较 [J ] . 中国食品药品监管， 2022 （ 12 ）： 32 - 39 .

LIU X . The domestic and foreign legislative evolution and reflection of compassionate use：the comparison of drug accessibility related systems [J ] . China Food Drug Adm Mag ， 2022 （ 12 ）： 32 - 39 .

BRAZIL R . Just out of reach：why the UK must rethink its barriers to novel medicines [J/OL ] . Pharm J ， 2020 [ 2023-07-10 ] . https：//pharmaceutical-journal.com/article/feature/just-out-of-reach-why-the-uk-must-rethink-its-barriers-to-novel-medicines https://pharmaceutical-journal.com/article/feature/just-out-of-reach-why-the-uk-must-rethink-its-barriers-to-novel-medicines .

O’CONNOR D J ， MCDONALD K ， LAM S P . Earlier patient access：the UK early access to medicines scheme （EAMS） [J ] . Med Access Point Care ， 2017 ， 1 ： 0000009 .

GOV ， UK . Apply for the early access to medicines scheme （EAMS） [EB/OL ] .（ 2016-05-10 ）[ 2023-07-15 ] . https：//www.gov.uk/guidance/apply-for-the-early-access-to-medi- cines-scheme-eams https://www.gov.uk/guidance/apply-for-the-early-access-to-medi-cines-scheme-eams .

GOV ， UK . Promising Innovative Medicine （PIM） desig- nation：step Ⅰ of early access to medicines scheme （EAMS） [EB/OL ] .（ 2014-04-18 ）[ 2023-07-15 ] . https：//assets.publishing.service.gov.uk/government/uploads/system/uploads/attachment_data/file/375327/PIM_designa-tion_guidance.pdf https://assets.publishing.service.gov.uk/government/uploads/system/uploads/attachment_data/file/375327/PIM_designa-tion_guidance.pdf .

袁妮，周娜，蒋蓉，等 . 探索我国罕见病患者药物可及的新途径：疫情下的同情用药制度 [J ] . 中国新药杂志， 2020 ， 29 （ 18 ）： 2041 - 2048 .

YUAN N ， ZHOU N ， JIANG R ， et al . Exploration of a new way of drug accessibility for patients with rare di- seases in China：the system of compassionate use under the epidemic situation [J ] . Chin J N Drugs ， 2020 ， 29 （ 18 ）： 2041 - 2048 .

GOV . UK .Guidance for applicants for the Early Access to Medicines Scheme （EAMS）：step Ⅱ[EB/OL ] .（ 2014-04-18 ）[ 2023-07-20 ] . https：//assets.publishing.service.gov.uk/government/uploads/system/uploads/attachment_data/file/375408/guidance_on_applying_for_a_scientific_opinion_ including_the_pre-submission_meeting.pdf https://assets.publishing.service.gov.uk/government/uploads/system/uploads/attachment_data/file/375408/guidance_on_applying_for_a_scientific_opinion_including_the_pre-submission_meeting.pdf .

GOV . UK .The Early Access to Medicines Scheme （EAMS）：operational guidance[EB/OL ] .（ 2016-05-10 ）[ 2023-07-15 ] . https：//assets.publishing.service.gov.uk/government/uploads/system/uploads/attachment_data/file/520967/eams-operational-guidance.pdf https://assets.publishing.service.gov.uk/government/uploads/system/uploads/attachment_data/file/520967/eams-operational-guidance.pdf .

Cancer Research UK . Safety measures and the Early Access to Medicines Scheme [EB/OL ] .（ 2019-11-08 ）[ 2023-10-17 ] . https：//www.cancerresearchuk.org/about-cancer/treatment/access-to-treatment/early-access-to-medicines-scheme/safety-measures https://www.cancerresearchuk.org/about-cancer/treatment/access-to-treatment/early-access-to-medicines-scheme/safety-measures .

GOV . UK .Early Access to Medicines Scheme （EAMS） scientific opinion：Nivolumab for treatment of adult patients with unresectable advanced，recurrent or metastatic oesophageal cancer after prior chemotherapy[EB/OL ] .（ 2022-04-18 ）[ 2023-07-20 ] . https：//www.gov.uk/government/publications/early-access-to-medicines-scheme-eams-scientific-opinion-nivolumab-for-treatment-of-adult-patients-with-unresectable-advanced-recurrent-or-metasta https://www.gov.uk/government/publications/early-access-to-medicines-scheme-eams-scientific-opinion-nivolumab-for-treatment-of-adult-patients-with-unresectable-advanced-recurrent-or-metasta .

HICKEY C . HPR22 eight years on，to what extent have patients benefitted from the UK’s Early Access to Medicines Scheme （EAMS）? [J ] . Value Health ， 2022 ， 25 （ 12 ）： S236 .

刘海燕 . 利益衡量视域下我国同情用药制度的完善 [J ] . 锦州医科大学学报（社会科学版）， 2023 ， 21 （ 6 ）： 10 - 15 .

LIU H Y . The improvement of China’s compassionate drug use system under the perspective of interest measurement [J ] . J Jinzhou Med Univ（Soc Sci Ed）， 2023 ， 21 （ 6 ）： 10 - 15 .

王如芳，邵文斌 . 中国罕见病药物可及性报告：2019 [R ] . 北京：中国罕见病发展中心， 2019 ： 13 .

WANG R F ， SHAO W B . China rare disease drug accessibility report：2019 [R ] . Beijing ： Chinese Organization for Rare Disorders ， 2019 ： 13 .

韩桑莹，姚军 . 美国欧盟的“同情用药” 制度及其对我国的启示 [J ] . 医学与法学， 2022 ， 14 （ 1 ）： 72 - 77 .

HAN S Y ， YAO J . Compassionate use system in the United States and the European Union and its enlightenment to China [J ] . Med Jurisprudence ， 2022 ， 14 （ 1 ）： 72 - 77 .

谭钰 . 浅议我国拓展性临床试验制度之建立 [J ] . 医学与法学， 2021 ， 13 （ 3 ）： 100 - 104，111 .

TAN Y . On the establishment of extended clinical trial system in China [J ] . Med Jurisprudence ， 2021 ， 13 （ 3 ）： 100 - 104，111 .

任磊，孙圆圆，舒丽芯，等 . 基于域外经验对我国同情用药制度的研究 [J ] . 实用药物与临床， 2022 ， 25 （ 9 ）： 860 - 864 .

REN L ， SUN Y Y ， SHU L X ， et al . Study on compassio- nate use system in China based on foreign experience [J ] . Pract Pharm Clin Remedies ， 2022 ， 25 （ 9 ）： 860 - 864 .

阮未艾，姚峥嵘，王艳翚，等 . 拓展性临床试验制度的中国实践及探索：以同情用药为视角 [J ] . 中国现代应用药学， 2021 ， 38 （ 18 ）： 2273 - 2277 .

RUAN W A ， YAO Z R ， WANG Y H ， et al . Practice and exploration of extended clinical trial system in China：from the perspective of compassionate use system [J ] . Chin J Mod Appl Pharm ， 2021 ， 38 （ 18 ）： 2273 - 2277 .

金秋晨，李梦颖，陈永法 . 英国NICE卫生技术评估公众参与机制及启示 [J ] . 中国药房， 2023 ， 34 （ 11 ）： 1302 - 1306 .

JIN Q C ， LI M Y ， CHEN Y F . Mechanism and enlightenment of public participation in the NICE health techno- logy assessment in England [J ] . China Pharm ， 2023 ， 34 （ 11 ）： 1302 - 1306 .

Views

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

Address：8th Floor, Sihuan Building, No. 129 Daping Main Street, Yuzhong District, Chongqing, China Postal code：400042
Technical support is provided by Beijing Founder Electronics co., LTD 渝ICP备15012710号公网安备50010302001817
It is recommended to read the content of this site in Chrome&IE9+. Please switch to extreme mode in browser 360.
Cookies We use cookies to help provide and enhance our service and tailor content. By continuing, you agree to the use of cookies.

⁰