Analysis of medical insurance access policy for rare disease drugs in the United Kingdom and enlightenment to China

DENG Taoyi; LIU Deyang; ZHOU Jing; WU Zhiang; HU Ming

doi:10.6039/j.issn.1001-0408.2023.13.03

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Analysis of medical insurance access policy for rare disease drugs in the United Kingdom and enlightenment to China

更新时间：2025-09-25

- Analysis of medical insurance access policy for rare disease drugs in the United Kingdom and enlightenment to China
- ZHONGGUO YAOFANG Vol. 34, Issue 13, Pages: 1555-1561(2023)
- 作者机构：
  
  四川大学华西药学院，成都　610041
- 作者简介：
- 基金信息：
- DOI：10.6039/j.issn.1001-0408.2023.13.03
  CLC： R951
- Received：12 December 2022，
  
  Revised：2023-05-14，
  
  Accepted：15 May 2023，
  
  Published：15 July 2023
- 稿件说明：
移动端阅览
邓韬毅,刘德阳,周静,等.英国罕见病用药医保准入政策分析及对我国的启示[J].中国药房,2023,34(13):1555-1561.

DENG Taoyi,LIU Deyang,ZHOU Jing,et al.Analysis of medical insurance access policy for rare disease drugs in the United Kingdom and enlightenment to China[J].ZHONGGUO YAOFANG,2023,34(13):1555-1561.
邓韬毅,刘德阳,周静,等.英国罕见病用药医保准入政策分析及对我国的启示[J].中国药房,2023,34(13):1555-1561. DOI： 10.6039/j.issn.1001-0408.2023.13.03.

DENG Taoyi,LIU Deyang,ZHOU Jing,et al.Analysis of medical insurance access policy for rare disease drugs in the United Kingdom and enlightenment to China[J].ZHONGGUO YAOFANG,2023,34(13):1555-1561. DOI： 10.6039/j.issn.1001-0408.2023.13.03.

摘要

目的

通过分析英国的罕见病用药医保准入政策，基于已有的准入途径及框架为我国罕见病用药医保准入提供参考依据。

方法

收集英国的罕见病用药相关指南、政策文件等，使用内容分析法对罕见病用药评估机制、报销决策标准、利益相关者参与程度、处理不确定性及风险的应对策略、政策实施效果等进行分析，提炼英国罕见病用药医保准入的关键点，为我国建立完善罕见病用药医保准入制度提供可参考建议。结果与

结论

从准入途径来看，英国采取了单独的路径和明确的考量标准对罕见病用药进行评估和报销。从评估机制来看，英国有医生、患者、申请人等多方利益相关者参与决策流程。英国通过收集更充分的临床证据，利用患者可及计划来处理不确定性和风险。英国在实施罕见病用药相关政策之后，在罕见病用药资金投入、药品申请报销通过率、受资助患者人数等方面卓有成效。建议我国在建立和完善罕见病用药评估报销制度过程中，通过建立单独的罕见病用药准入评估路径，再加上更多利益相关的参与，实现我国罕见病用药可及性的提高。

Abstract

OBJECTIVE

To provide reference for the access to medical insurance for rare diseases in China based on the existing access pathway and framework by analyzing the access policy of medical insurance for rare diseases in the United Kingdom （UK）.

METHODS

After collecting relevant guidelines and policy documents related to drug use for rare diseases in the UK， content analysis method was used to analyze the evaluation mechanism of drug use for rare diseases， reimbursement decision-making standards， stakeholder participation， coping strategies for dealing with uncertainties and risks， and policy implementation effects， and extract the key points of medical insurance access for drug use for rare diseases in the UK， to provide some suggestions for the establishment of medical insurance access system for rare diseases in China. RESULTS &

CONCLUSIONS

From the perspective of access， the UK had adopted a separate approach and clear criteria to assess and reimburse drugs for rare diseases. From the perspective of evaluation mechanism， multi-stakeholders such as doctors， patients and applicants participated in the decision-making process in the UK. The UK addressed uncertainty and risk by gathering better clinical evidence and using the patient access programme. After the implementation of the policy related to drug use for rare diseases， the UK had achieved remarkable results in terms of funding for drug use for rare diseases， the reimbursement rate of drug application， and the number of funded patients. It is suggested that in the process of establishing and improving the evaluation and reimbursement system for rare diseases drugs in China， the availability of rare diseases drugs should be improved by establishing a separate access assessment path for rare diseases drugs and involving more stakeholders.

关键词

Keywords

references

RHEE T G . Policymaking for orphan drugs and its challenges [J ] . AMA J Ethics ， 2015 ， 17 （ 8 ）： 776 - 779 .

Scottish Medicines . PACE （Patient & Clinician Engagement） overview document [EB/OL ] . [ 2022-08-22 ] . https：//www.scottishmedicines.org.uk/media/2782/pace-overview-document.pdf https://www.scottishmedicines.org.uk/media/2782/pace-overview-document.pdf .

Organisation for Economic Co-operation and Development （OECD） . Health expenditure and financing [EB/OL ] . [ 2022-08-24 ] . https：//stats.oecd.org/Index.aspx?DatasetCode=HEALTH_STAT https://stats.oecd.org/Index.aspx?DatasetCode=HEALTH_STAT .

National statistics . Healthcare expenditure，UK Health [EB/OL ] . [ 2022-08-25 ] . https：//www.ons.gov.uk/peoplepopulationandcommunity/healthandsocialcare/healthcaresystem/bulletins/ukhealthaccounts/2017#how-healthcare-in-the-uk-is-financed https://www.ons.gov.uk/peoplepopulationandcommunity/healthandsocialcare/healthcaresystem/bulletins/ukhealthaccounts/2017#how-healthcare-in-the-uk-is-financed .

Department of Health . The NHS constitution for England [EB/OL ] . [ 2022-08-26 ] . https：//www.gov.uk/government/publications/thenhs-constitution-for-england/the-nhs-constitution-for-england https://www.gov.uk/government/publications/thenhs-constitution-for-england/the-nhs-constitution-for-england .

National Statistics . Prescriptions dispensed in the community [EB/OL ] . [ 2022-09-10 ] . https：//files.digital.nhs.uk/publication/s/o/pres-disp-com-eng-2006-16-rep.pdf https://files.digital.nhs.uk/publication/s/o/pres-disp-com-eng-2006-16-rep.pdf .

袁妮，田婷婷，张海军，等 . 英国罕见病用药纳入报销体系的主要途径及对我国的启示 [J ] . 中国药科大学学报， 2019 ， 50 （ 1 ）： 113 - 119 .

张海军，梁云，袁妮 . 英国NICE罕见病用药卫生技术评估流程介绍及其启示 [J ] . 中国新药杂志， 2018 ， 27 （ 14 ）： 1587 - 1594 .

宣建伟，孙巧 . 中国罕见病药物经济学评估适用模型与支付阈值参考标准探讨 [J ] . 国际药学研究杂志， 2019 ， 46 （ 9 ）： 659 - 665 .

ADRIAN T ， MARTINA G . Appraising ultra-orphan drugs：is Cost-Per-QALY appropriate? a review of the evidence[EB/OL ] .[ 2022-08-26 ] . https：//ideas.repec.org/cgi-bin/htsearch?form=extended&wm=wrd&dt=range&ul=&q=appraising+ultra-orphan+drugs&cmd=Search%21&wf=4BFF&s=R&db=&de= https://ideas.repec.org/cgi-bin/htsearch?form=extended&wm=wrd&dt=range&ul=&q=appraising+ultra-orphan+drugs&cmd=Search%21&wf=4BFF&s=R&db=&de= .

National Institute for Health and Care Excellence（NICE） . Interim process and methods of the Highly Specialised Technologies programme [EB/OL ] . [ 2022-09-12 ] . https：//www.nice.org.uk/Media/Default/About/what-we-do/NICE-guidance/NICE-highly-specialised-technologies-guidance/HST-interim-methods-process-guide-may-17.pdf https://www.nice.org.uk/Media/Default/About/what-we-do/NICE-guidance/NICE-highly-specialised-technologies-guidance/HST-interim-methods-process-guide-may-17.pdf .

National Institute for Health and Care Excellence（NICE） . Guidance and advice list [EB/OL ] . [ 2022-09-15 ] . https：//www.nice.org.uk/guidance/published?type=hst https://www.nice.org.uk/guidance/published?type=hst .

National Health Service . Specialized services [EB/OL ] . [ 2022-09-15 ] . https：//www.england.nhs.uk/commissioning/spec-services/key-docs/#scog https://www.england.nhs.uk/commissioning/spec-services/key-docs/#scog .

National Health Service . Spotlight on specialised services [EB/OL ] . [ 2022-09-18 ] . https：//www.england.nhs.uk/wp-content/uploads/2018/09/spotlight-on-specialised-services-second-edition.pdf https://www.england.nhs.uk/wp-content/uploads/2018/09/spotlight-on-specialised-services-second-edition.pdf .

NICOD E ， ANNEMANS L ， BUCSICS A ， et al . HTA programme response to the challenges of dealing with orphan medicinal products：process evaluation in selected European countries [J ] Health Policy ， 2019 ， 123 （ 2 ）： 140 - 151 .

Scottish Medicines . Patient and Clinician Engagement （PACE） process [EB/OL ] . [ 2022-09-18 ] . https：//www.scottishmedicines.org.uk/how-we-decide/pace/ https://www.scottishmedicines.org.uk/how-we-decide/pace/ .

Scottish Government . Review of access to new medicines [EB/OL ] . [ 2022-09-22 ] . https：//www.gov.scot/publications/review-access-new-medicines/pages/11/ https://www.gov.scot/publications/review-access-new-medicines/pages/11/ .

Scottish Government . Treatments for rare conditions [EB/OL ] .[ 2022-09-22 ] . https：//news.gov.scot/news/treatments-for-rare-conditions https://news.gov.scot/news/treatments-for-rare-conditions .

Scottish Medicines . Revised process ultra-orphan medicines for extremely rare conditions [EB/OL ] . [ 2022-09-25 ] . https：//www.scottishmedicines.org.uk/how-we-decide/revised-process-ultra-orphan-medicines-for-extremely-rare- conditions/ https://www.scottishmedicines.org.uk/how-we-decide/revised-process-ultra-orphan-medicines-for-extremely-rare-conditions/ .

Scottish Medicines . Guidance to submitting companies for completion of New Product Assessment Form （NPAF）：supplement for medicines for extremely rare conditions （ultra-orphan medicines） [EB/OL ] . [ 2022-09-25 ] . https：//www.scottishmedicines.org.uk/media/4155/guidance-supplement-ultra-orphan.pdf https://www.scottishmedicines.org.uk/media/4155/guidance-supplement-ultra-orphan.pdf .

李乐乐，何晓彤，陈湘妤，等 . 我国罕见病用药保障现状及优化路径 [J ] . 中国医疗保险， 2022 （ 9 ）： 116 - 119 .

袁帅，吴应宇 . 我国罕见病用药的医疗保险准入评价指标体系构建 [J ] . 中国卫生资源， 2021 ， 24 （ 6 ）： 646 - 649 .

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