Key considerations for using real-world data to evaluate the clinical and economic value of drugs_Chinese Journal of Evidence-Based Medicine

Authors：

 TAN Jing ^1,2 , XIONG Yiquan ^1,2 , HUANG Shiyao ^1,2 , REN Yan ^1,2 , GAO Pei ³ , CHEN Yingyao ⁴ , MAO Chen ⁵ , PENG Xiaoxia ⁶ , ZHAO Shaoyang ⁷ , WU Hongyan ⁸ , ZHANG Tiantian ⁹ , GUAN Haijing ¹⁰ ,  SUN Xin ^1,2

1. Chinese Evidence-based Medicine Center, West China Hospital, Sichuan University, Chengdu 610041, P. R. China;
2. NMPA Key Laboratory for Real World Data Research and Evaluation in Hainan, Chengdu 610041, P. R. China;
3. Department of Epidemiology and Biostatistics, School of Public Health, Peking University, Beijing 100191, P. R. China;
4. School of Public Health, Fudan University, Shanghai 200433, P. R. China;
5. Department of Epidemiology, School of Public Health, Southern Medical University, Guangzhou 510515, P. R. China;
6. National Center for Children’s Health, China; Clinical Epidemiology and Evidence-based Medicine Center, Beijing Children’s Hospital, Capital Medical University, Beijing 100045, P. R. China;
7. School of economics, Sichuan University, Chengdu 610041, P. R. China;
8. School of Medicine and Health Management, Guizhou Medical University, Guiyang 550000, P. R. China;
9. College of Pharmacy, Jinan University, Guangzhou 510515, P. R. China;
10. Department of Pharmacy, Beijing Tiantan Hospital, Capital Medical University, Beijing 100045, P. R. China;

Corresponding author：

TAN Jing, Email: tanjing_wch@scu.edu.cn; SUN Xin, Email: sunxin@wchscu.cn

Keywords：

Real-world data; Real-world evidence; Clinical value of drugs; Economic value of drugs

DOI：

10.7507/1672-2531.202401081

Video：

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With the acceleration of global innovative drug development, selecting safe, effective, and cost-effective products from numerous drugs has posed new challenges for the decision-making process of medical insurance drug access and dynamic updating of insurance directory. Real-world data (RWD) provides a new perspective for evaluation of clinical and economic value of drugs, but there are still uncertainties regarding the scope, quality standards, and evidence categories of RWD that can be used. Based on the current status of domestic and international RWD supporting the assessment of the clinical and economic value of drugs, this paper, in collaboration with national RWD and healthcare experts, has developed the key considerations for using real-world data to evaluate the clinical and economic value of drugs. This paper first clarifies the scope of RWD that can be used to evaluate the clinical and economic value of drugs evaluate; secondly, provides specific requirements and guidance on data attribution, data governance, and quality standards for RWD; finally, summarizes the evidence categories of RWD supporting evaluate the clinical and economic value of drugs evaluate.

Citation： TAN Jing, XIONG Yiquan, HUANG Shiyao, REN Yan, GAO Pei, CHEN Yingyao, MAO Chen, PENG Xiaoxia, ZHAO Shaoyang, WU Hongyan, ZHANG Tiantian, GUAN Haijing, SUN Xin. Key considerations for using real-world data to evaluate the clinical and economic value of drugs. Chinese Journal of Evidence-Based Medicine, 2024, 24(5): 516-522. doi: 10.7507/1672-2531.202401081 Copy

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2.	国家卫生健康委办公厅. 药品临床综合评价管理指南(2021年版试行)(国卫办药政发〔2021〕16号). 2021.
3.	Centers for Medicare & Medicaid Services (CMS). Coverage with evidence development. 2006.
4.	NICE. Real-world evidence framework. 2022.
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8.	孙鑫, 谭婧, 王雯, 等. 真实世界证据助推药械评价与监管决策. 中国循证医学杂志, 2019, 19(5): 521-526.
9.	孙鑫, 谭婧, 唐立, 等. 基于真实世界证据的上市后药品评价技术框架体系: 思考与建议. 中国循证医学杂志, 2018, 18(4): 277-283.
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13.	王雯, 谭婧, 任燕, 等. 重新认识真实世界数据研究: 更新与展望. 中国循证医学杂志, 2020, 20(11): 1241-1246.
14.	孙鑫, 谭婧, 唐立, 等. 重新认识真实世界研究. 中国循证医学杂志, 2017, 17(2): 126-130.
15.	孙鑫, 谭婧, 王雯, 等. 建立真实世界数据与研究技术规范, 促进中国真实世界证据的生产与使用. 中国循证医学杂志, 2019, 19(7): 755-762.
16.	高富平. 论医疗数据权利配置─医疗数据开放利用法律框架. 现代法学, 2020, 42(4): 52-68.
17.	张博妮, 陈昱洁, 毛湛, 等. 人工智能医疗数据应用的法律问题及对策探析. 法制博览, 2021, (8): 18-21.
18.	訾明杰, 白楠, 蒋辉, 等. 泛化知情同意的临床操作及思考. 中国医学伦理学, 2020, 33(3): 315-319.
19.	王雯, 高培, 吴晶, 等. 构建基于既有健康医疗数据的研究型数据库技术规范. 中国循证医学杂志, 2019, 19(7): 763-770.
20.	谭婧, 彭晓霞, 舒啸尘, 等. 患者登记数据库构建技术规范. 中国循证医学杂志, 2019, 19(7): 771-778.
21.	国家药品监督管理局. 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). 2020.
22.	FDA. Real-world data: assessing electronic health records and medical claims data to support regulatory decision-making for drug and biological products. 2021.
23.	AMCP. Guidance on submission of pre-approval and post-approval clinical and economic information and evidence, version 4.1. 2023.
24.	PHARMAC. Prescription for pharmacoeconomic analysis: methods for cost-utility analysis version 2.2. 2020.
25.	HAS. Choices in methods for economic evaluation. 2020.
26.	IQWiG. General methods for the assessment of the relation of benefits to costs. 2009.

1. 宣建伟, 李幼平. 真实世界研究与创新药物价值. 中国循证医学杂志, 2018, 18(4): 273-276.
2. 国家卫生健康委办公厅. 药品临床综合评价管理指南(2021年版试行)(国卫办药政发〔2021〕16号). 2021.
3. Centers for Medicare & Medicaid Services (CMS). Coverage with evidence development. 2006.
4. NICE. Real-world evidence framework. 2022.
5. CADTH. Guidelines for the economic evaluation of health technologies: Canada (4th edition). 2021.
6. Garrison LP, Neumann PJ, Erickson P, et al. Using real-world data for coverage and payment decisions: the ISPOR Real-World Data Task Force report. Value Health, 2007, 10(5): 326-335.
7. Oortwijn W, Sampietro-Colom L, Trowman R. How to deal with the inevitable: generating real-world data and using real-world evidence for HTA purposes - from theory to action. Int J Technol Assess Health Care, 2019, 35(4): 346-350.
8. 孙鑫, 谭婧, 王雯, 等. 真实世界证据助推药械评价与监管决策. 中国循证医学杂志, 2019, 19(5): 521-526.
9. 孙鑫, 谭婧, 唐立, 等. 基于真实世界证据的上市后药品评价技术框架体系: 思考与建议. 中国循证医学杂志, 2018, 18(4): 277-283.
10. FDA. Framework for FDA’s real-world evidence program. 2018.
11. ICER. Real world evidence for coverage decisions: opportunities and challenges. 2018.
12. Makady A, de Boer A, Hillege H, et al. What is real-world data. A review of definitions based on literature and stakeholder interviews. Value Health, 2017, 20(7): 858-865.
13. 王雯, 谭婧, 任燕, 等. 重新认识真实世界数据研究: 更新与展望. 中国循证医学杂志, 2020, 20(11): 1241-1246.
14. 孙鑫, 谭婧, 唐立, 等. 重新认识真实世界研究. 中国循证医学杂志, 2017, 17(2): 126-130.
15. 孙鑫, 谭婧, 王雯, 等. 建立真实世界数据与研究技术规范, 促进中国真实世界证据的生产与使用. 中国循证医学杂志, 2019, 19(7): 755-762.
16. 高富平. 论医疗数据权利配置─医疗数据开放利用法律框架. 现代法学, 2020, 42(4): 52-68.
17. 张博妮, 陈昱洁, 毛湛, 等. 人工智能医疗数据应用的法律问题及对策探析. 法制博览, 2021, (8): 18-21.
18. 訾明杰, 白楠, 蒋辉, 等. 泛化知情同意的临床操作及思考. 中国医学伦理学, 2020, 33(3): 315-319.
19. 王雯, 高培, 吴晶, 等. 构建基于既有健康医疗数据的研究型数据库技术规范. 中国循证医学杂志, 2019, 19(7): 763-770.
20. 谭婧, 彭晓霞, 舒啸尘, 等. 患者登记数据库构建技术规范. 中国循证医学杂志, 2019, 19(7): 771-778.
21. 国家药品监督管理局. 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). 2020.
22. FDA. Real-world data: assessing electronic health records and medical claims data to support regulatory decision-making for drug and biological products. 2021.
23. AMCP. Guidance on submission of pre-approval and post-approval clinical and economic information and evidence, version 4.1. 2023.
24. PHARMAC. Prescription for pharmacoeconomic analysis: methods for cost-utility analysis version 2.2. 2020.
25. HAS. Choices in methods for economic evaluation. 2020.
26. IQWiG. General methods for the assessment of the relation of benefits to costs. 2009.

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