本文基于英国国家卫生与临床优化研究所（National Institute for Health and Care Excellence, NICE）发布的《真实世界证据框架》,探讨其方法论创新及对我国医保准入决策的启示。在全球医保治理迈向价值驱动的背景下,真实世界证据（Real World Evidence, RWE）已成为弥补随机对照试验局限、支撑医保精准购买的关键工具,其科学生成与规范应用至关重要。NICE框架以透明性、数据适用性与方法严谨性为三大支柱,系统构建了RWE的应用体系：通过倡导“目标试验模拟”来增强观察性研究的因果推断效力,并整合定性证据,以全面评估患者体验与临床实施路径。实践表明,该框架在癌症、数字疗法及罕见病等领域的评估中成效显著。我国正处于真实世界医保价值评价探索期,鉴于此,可重点从夯实数据治理基础、规范方法学标准、明晰实施路径三个维度着手,构建符合国情的本土化RWE应用体系。
Abstract
Based on the Real-World Evidence Framework published by the United Kingdom's National Institute for Health and Care Excellence (NICE), this paper explores its methodological innovations and discusses the instructive value for healthcare insurance reimbursement decision-making in China. Against the backdrop of a global shift toward value-driven healthcare governance, real-world evidence (RWE) has become a key tool for addressing the limitations of randomized controlled trials and supporting strategic purchasing decisions by payers. The scientific generation and standardized application of RWE are therefore of paramount importance. The NICE framework is built upon three pillars: transparency, data suitability, and methodological rigor. It systematically constructs an application system for RWE. It improves the validity of causal inference in observational studies by advocating the "target trial emulation" approach and incorporates qualitative evidence to comprehensively assess patient experience and clinical implementation pathways. Practice has demonstrated the significant effectiveness of this framework for evaluating technologies in domains such as oncology, digital therapeutics, and rare diseases. Considering this, China, in the exploratory phase of leveraging RWE for the comprehensive value assessment of health technologies within its healthcare insurance decision-making, can focus on three key dimensions: strengthening data governance foundations, standardizing methodological norms, and clarifying implementation pathways, to build a localized RWE application system suited to the national context.
关键词
真实世界证据 /
真实世界数据 /
NICE框架 /
医保决策 /
卫生技术评估
Key words
real-world evidence /
real-world data /
NICE framework /
healthcare insurance decision-making /
health technology assessment
{{custom_sec.title}}
{{custom_sec.title}}
{{custom_sec.content}}
参考文献
[1] CANDORE G, MARTIN C, MILLS MJ, et al.FRAME: framework for real-world evidence assessment to mitigate evidence uncertainties for efficacy/effectiveness - an evaluation of regulatory and health technology assessment decision making[J]. Clin Pharmacol Ther. 2025,118(3):649-661.
[2] MOSPAN AR, MORRIS HL, FRIED MW. Real-world evidence in hepatocellular carcinoma[J]. Liver Int.2021,41 Suppl 1:61-67.
[3] TADROUS M, AVES T, FAHIM C, et al.Development of a Canadian guidance for reporting real-world evidence for regulatory and health-technology assessment (HTA) decision-making[J]. J Clin Epidemiol. 2024, 176:111545.
[4] WAURE C, CALABRO GE, RICCIARDI W.Value(s) of Vaccination Project Steering Committee. Recommendations to drive a value-based decision-making on vaccination[J]. Expert Rev Vaccines. 2022,21(3):289-296.
[5] 国家医疗保障局办公室.关于开展真实世界医保综合价值评价试点工作的通知[EB/OL]. (2025-09-23)[2025-12-4] https://www.nhsa.gov.cn/art/2025/9/23/art_109_17984.html.
[6] National Institute for Health and Care Excellence. NICE real-world evidence framework [EB/OL]. (2022-06-23)[2025-12-04]. https://www.nice.org.uk/corporate/ecd9.
[7] KO AH, OH DY, LAU J, et al.Investigational use of real-world data as a hybrid control in pancreatic ductal adenocarcinoma from the randomized Phase Ib/II MORPHEUS Trial[J]. Clin Pharmacol Ther, 2025,117(4):1021-1029.
[8] ARENA PJ, SUN Y, JAKSA A, et al.Information bias in electronic health records and administrative claims data: a targeted review of the recent literature[J]. Clin Pharmacol Ther, 2026, 119(2):362-367.
[9] WEBERPALS J, WANG SV.External controls to study treatment effects in rare diseases: challenges and future directions[J]. Clin Pharmacol Ther, 2024,116(6):1521-1524.
[10] RUSSEK M, PELTNER J, HAENISCH B.Supplementing single-arm trials with external control arms-evaluation of German real-world data[J]. Clin Pharmacol Ther, 2025,118(6):1443-1450.
[11] PAPADAKIS G, IBOK A, KOLOVOS S, et al. Guidance for real-world evidence generation for the joint clinical assessment process and its implications for country-specific HTA body acceptability[EB/OL]. (2025-11-14)[2025-12-04]. https://www.ispor.org/heor-resources/presentations-database/presentation-cti/ispor-europe-2025/poster-session-3-2/guidance-for-real-world-evidence-generation-for-the-joint-clinical-assessment-process-and-its-implications-for-country-specific-hta-body-acceptability.
[12] CUSCHIERI S. The STROBE guidelines[J]. Saudi J Anaesth, 2019 Apr,13(Suppl 1):S31-S34.
[13] BENCHIMOL EI, SMEETH L, GUTTMANN A, et al.RECORD Working Committee, the reporting of studies conducted using observational routinely-collected health data (RECORD) statement[J]. PLoS Med, 2015,12(10):e1001885.
[14] LANGAN SM, SCHMIDT SA, WING K, et al.The reporting of studies conducted using observational routinely collected health data statement for pharmacoepidemiology (RECORD-PE)[J]. BMJ, 2018,363:k3532.
[15] WANG SV, PINHEIRO S, HUA W, et al.STaRT-RWE: structured template for planning and reporting on the implementation of real world evidence studies[J]. BMJ, 2021,372:m4856.
[16] BULLEMENT A, PODKONJAK T, ROBINSON MJ, et al.Real-world evidence use in assessments of cancer drugs by NICE[J]. International journal of technology assessment in health care, 2020,36(4):388-94.
[17] National Institute for Health and Care Excellence. Digitally enabled therapies for adults with anxiety disorders: early value assessment[EB/OL]. (2023-12-14)[2025-12-23]. https://www.nice.org.uk/guidance/htg676.
[18] National Institute for Health and Care Excellence. Artificial intelligence (AI)-derived software to analyse chest X-rays for suspected lung cancer in primary care referrals: early value assessment[EB/OL]. (2025-02-10)[2025-12-23]. https://www.nice.org.uk/guidance/htg696.
[19] National Institute for Health and Care Excellence. Elosulfase alfa for treating mucopolysaccharidosis type 4A[EB/OL]. (2022-04-20)[2025-12-04]. https://www.nice.org.uk/guidance/hst19.
[20] National Institute for Health and Care Excellence. Nusinersen for treating spinal muscular atrophy[EB/OL]. (2019-07-24)[2025-12-04]. https://www.nice.org.uk/guidance/ta588.
