目的: 本研究基于英国国家卫生与服务优化研究院（NICE）的真实世界数据（RWD）应用案例,分析药品医保准入中RWD的角色与挑战,为我国政策制定提供借鉴。方法: 采用案例研究法,分析NICE体系下药品准入与管理准入计划（MAA）的实施细节。结果: 英国医保体系通过NICE实施的MAA,利用RWD评估药品疗效与成本效益,促进了创新药品快速进入国家医疗服务体系（NHS）。结论: 我国可借鉴英国经验,完善RWD相关政策与标准,加强国际合作,推动RWD在医保体系的广泛应用与深入发展,为医疗资源的优化配置与患者的医疗保障提供更加坚实的支撑。
Abstract
Objective: Based on the real-world data (RWD) application case study of the National Institute for Health and Care Excellence (NICE) in the UK, this study analyzes the roles and challenges of RWD in medical insurance access of drugs, which can provide a reference for the policy making in China. Methods: A case study method was used to analyze the implementation details of the Managed Access Agreement (MAA) under the NICE system. Results: The UK healthcare system has facilitated the rapid entry of innovative medicines into the National Health Service (NHS) by utilizing RWD to assess the efficacy and cost-effectiveness of medicines through the MAA implemented by NICE. Conclusion: China can learn from the UK, improve RWD-related policies and standards, strengthen international cooperation, and promote the wide application and in-depth development of RWD in the medical insurance system, so as to provide a more solid support for the optimal allocation of healthcare resources and patients' healthcare security.
关键词
真实世界数据（RWD） /
英国国家卫生与服务优化研究院（NICE） /
医保准入
Key words
real-world data (RWD) /
National Institute for Health and Care Excellence (NICE) /
medical insurance access
{{custom_sec.title}}
{{custom_sec.title}}
{{custom_sec.content}}
参考文献
[1] 国家药品监督管理局药品审评中心.国家药监局关于发布真实世界证据支持药物研发与审评的指导原则(试行)的通告(2020年第1号)[EB/OL]. (2020-01-07)[2024-07-22].https://www.nmpa.gov.cn/directory/web/nmpa/xxgk/ggtg/ypggtg/ypqtggtg/20200107151901190.html.
[2] 国家药品监督管理局药品审评中心.国家药监局药审中心关于发布《用于产生真实世界证据的真实世界数据指导原则(试行)》的通告(2021年第27号)[EB/OL].(2021-04-15)[2024-07-22].https://www.cde.org.cn/main/news/viewInfoCommon/2a1c437ed54e7b838a7e86f4ac21c539.
[3] 黄涛,李灿,黄先琴,等.真实世界数据用于药品医保决策的国内外政策分析[J].中国循证医学杂志,2024,24(05):510-515.
[4] 周菁菁,黄梦琴,孙志明,等.附条件批准上市药品纳入基本医疗保险药品目录的政策风险分析[J].医学与社会,2023,36(01):99-104.
[5] NHS England.NHS commissioning[EB/OL].[2024-07-22].https://www.england.nhs.uk/commissioning/.
[6] NHS England. NHS commercial framework for new medicines[EB/OL]. (2022-07-07)[2024-07-22]. https://www.england.nhs.uk/publication/nhs-commercial-framework-for-new-medicines/.
[7] ZHANG X, CHEN L, BRACCO OL.Regulator-requested non-interventional postauthorization safety and effectiveness studies for oncology drugs: a systematic review[J]. Clinical pharmacology and therapeutics, 2022, 111(1):155-167.
[8] 徐嘉悦,何俏,刘艳梅,等.真实世界数据用于药品临床价值评价的关键技术考量[J].中国循证医学杂志,2024,24(05):523-528.
[9] Gemeinsamer Bundesausschuss. Genehmigung eines langfristigen Heilmittelbedarfs[EB/OL].(2021-11-18)[2024-07-22]. https://www.g-ba.de/downloads/17-98-5254/2024-07-22_AM-RL_Onasemnogen-Abeparvovec_abD_Studienprotokoll.pdf.
[10] HUTTON J, TRUEMAN P, HENSHALL C.Coverage with evidence development: an examination of conceptual and policy issues[J]. International journal of technology assessment in health care, 2007, 23(4):425-432.
[11] NHS England.Standards and collections[EB/OL].[2024-07-22].https://digital.nhs.uk/data-and-information/information-standards/information-standards-and-data-collections-including-extractions/publications-and-notifications/standards-and-collections.
[12] MORRELL L, WORDSWORTH S, SCHUH A.Will the reformed Cancer Drugs Fund address the most common types of uncertainty? an analysis of NICE cancer drug appraisals[J]. BMC health services research, 2018, 18(1):393.
[13] KANG J, CAIRNS J.Protocol for data extraction: how real-world data have been used in the National Institute for Health and Care Excellence appraisals of cancer therapy[J]. BMJ open, 2022, 12(1):e055985.
[14] TRIGG L A, BARNISH M S, HAYWARD S.An analysis of uncertainties and data collection agreements in the cancer drugs fund[J]. Pharmacoecon open, 2024, 8(2):303-311.
[15] KANG J, CAIRNS J."Don't think twice, it's all right": using additional data to reduce uncertainty regarding oncologic drugs provided through managed access agreements in England[J]. Pharmacoecon open, 2023, 7(1):77-91.
[16] DAYER V W, DRUMMOND M F, DABB OUS O.Real-world evidence for coverage determination of treatments for rare diseases[J]. Orphanet journal of rare diseases, 2024, 19(1):47.
[17] BERGER M L, DREYER N, ANDERSON F.Prospective observational studies to assess comparative effectiveness: the ISPOR good research practices task force report[J]. Value in health, 2012, 15(2):217-230.
[18] 谭婧,彭晓霞,舒啸尘,等.患者登记数据库构建技术规范[J].中国循证医学杂志,2019,19(07):771-778.
[19] LI M, CHEN S, LAI Y.Integrating real-world evidence in the regulatory decision-making process: a systematic analysis of experiences in the US, EU, and China using a logic model[J]. Frontiers in medicine, 2021, 8:669509.
基金
2022年度辽宁省教育厅基本科研项目“基于中断时间序列分析辽宁省儿童医疗保障情况的真实世界研究”（LJKMR20221296）
