Research on techniques of economic evaluations of drugs by using the real-world data_Chinese Journal of Evidence-Based Medicine

Authors：

 WU Jing ^1,2 ,  SUN Xin ^3,4 , ZHANG Jiahui ^1,2 , XIE Shitong ^1,2 , HE Xiaoning ^1,2 , WU Bin ⁵ , GUAN Xiaodong ^6,7 , REN Yan ^3,4 , GUO Wudong ⁸ , SUN Lihua ⁹ , ZHAO Kun ^10,11 , HU Shanlian ¹²

1. School of Pharmaceutical Science and Technology, Tianjin University, Tianjin 300072, P. R. China;
2. Center for Social Science Survey and Data, Tianjin University, Tianjin 300072, P. R. China;
3. Chinese Evidence-based Medicine Center, West China Hospital, Sichuan University, Chengdu 610041, P. R. China;
4. NMPA Key Laboratory for Real World Data Research and Evaluation in Hainan, Chengdu 610041, P. R. China;
5. Clinical Research Unit, Ren Ji Hospital, South Campus, School of Medicine, Shanghai Jiaotong University, Shanghai 200001, P. R. China;
6. Department of Pharmacy Administration and Clinical Pharmacy, School of Pharmaceutical Sciences, Peking University, Beijing 100191, P. R. China;
7. International Research Center for Medicinal Administration, Peking University, Beijing 100191, P. R. China;
8. China National Health Development Research Center, National Health Commission, Beijing 100044, P. R. China;
9. School of Business Administration, Shenyang Pharmaceutical University, Shenyang 110016, P. R. China;
10. Vanke School of Public Health, Tsinghua University, Beijing 100084, P. R. China;
11. China National Health Development Research Center National Health Commission, Beijing 100044, P. R. China;
12. School of Public Health, Fudan University, Shanghai 200032, P. R. China;

Corresponding author：

WU Jing, Email: jingwu@tju.edu.cn; SUN Xin, Email: sunxin@wchscu.cn

Keywords：

Economic value; Economic evaluation; Cost-effectiveness analysis; Real-world data

DOI：

10.7507/1672-2531.202401114

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The application of economic tools to evaluate the cost and health benefits and screen out more cost-effective drugs and technologies is an important measure to improve efficiency of medical resource allocation in China. Given the inherent differences between strict clinical trials and clinical routine practice, using trial-based economic evaluations to guide relevant medical decisions may lead to a certain risk of value deviation. Recent development of real-world data provides opportunities to assess the cost-effectiveness of drugs under the practical utilization, and has gradually become a new research hotspot. However, the complexity of the actual clinical environment also puts higher demands on researchers and decision makers to construct, understand and apply real-world evidence. In order to further prompt the normalization of economic evaluation based on real-world data and promote the scientific application of real-world evidence in medical and health decision-making, this project aims at the crucial issues including scope, research design and quality evaluation, to clarify the key considerations on the using of real-world evidence in medical decision-making. Combined with the international guidelines, the latest advancement of relevant research areas and the advice and opinions from multidisciplinary experts, we aim to provide technical references and guidance for researchers and decision makers, and to strengthen the evidence base of management policies.

Citation： WU Jing, SUN Xin, ZHANG Jiahui, XIE Shitong, HE Xiaoning, WU Bin, GUAN Xiaodong, REN Yan, GUO Wudong, SUN Lihua, ZHAO Kun, HU Shanlian. Research on techniques of economic evaluations of drugs by using the real-world data. Chinese Journal of Evidence-Based Medicine, 2024, 24(5): 529-535. doi: 10.7507/1672-2531.202401114 Copy

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4.	Caro JJ, Briggs AH, Siebert U, et al. Modeling good research practices--overview: a report of the ISPOR-SMDM Modeling Good Research Practices Task Force-1. Value Health, 2012, 15(6): 796-803.
5.	Thokala P, Dodd P, Baalbaki H, et al. Developing Markov models from real-world data: a case study of heart failure modeling using administrative data. Value Health, 2020, 23(6): 743-750.
6.	Xia C, Dong X, Li H, et al. Cancer statistics in China and United States, 2022: profiles, trends, and determinants. Chin Med J (Engl), 2022, 135(5): 584-590.
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8.	National University of Singapore. Use of real-world data and real-world evidence to support drug reimbursement decision-making in Aisa. 2021.
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11.	Chay J, Fenner BJ, Finkelstein EA, et al. Real-world cost-effectiveness of anti-VEGF monotherapy and combination therapy for the treatment of polypoidal choroidal vasculopathy. Eye (Lond), 2022, 36(12): 2265-2270.
12.	Hong SH, Cho JY, Kim TB, et al. Cost-effectiveness of tiotropium in elderly patients with severe asthma using real-world data. J Allergy Clin Immunol Pract, 2021, 9(5): 1939-1947.
13.	Aarnio E, Korhonen MJ, Huupponen R, et al. Cost-effectiveness of statin treatment for primary prevention in conditions of real-world adherence--estimates from the Finnish prescription register. Atherosclerosis, 2015, 239(1): 240-247.
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16.	National Institute for Health and Clinical Excellence. Technology appraisal guidance. 2022.
17.	Faleiros DR, Alvares-Teodoro J, Nunes da Silva E, et al. Budget impact analysis of medicines: estimated values versus real-world evidence and the implications. Expert Rev Pharmacoecon Outcomes Res, 2022, 22(2): 271-281.
18.	国家药品监督管理局药品审评中心. 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). 2021.
19.	U. S. Food & Drug Administration. Use of real-world evidence to support regulatory decision-making for medical devices.
20.	聂晓璐, 王胜锋, 姚晨, 等. 国内外真实世界数据/证据的相关药械监管政策法规及指导原则比较研究. 药物流行病学杂志, 2022, 31(1): 5-11.
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22.	国家药品监督管理局. 关于发布真实世界数据用于医疗器械临床评价技术指导原则(试行)的通告(2020年第77号). 2020.
23.	聂晓路, 彭晓霞. 使用常规收集卫生数据开展观察性研究的报告规范. 中国循证医学杂志, 2017, 17(4): 475-487.
24.	Bowrin K, Briere JB, Levy P, et al. Use of real-world evidence in meta-analyses and cost-effectiveness models. J Med Econ, 2020, 23(10): 1053-1060.
25.	Hampson G, Towse A, Dreitlein WB, et al. Real-world evidence for coverage decisions: opportunities and challenges. J Comp Eff Res, 2018, 7(12): 1133-1143.
26.	高培, 王杨, 罗剑锋, 等. 基于真实世界数据评价治疗结局研究的统计分析技术规范. 中国循证医学杂志, 2019, 19(7): 787-793.
27.	聂晓璐, 武泽昊, 赵厚宇, 等. 使用常规收集医疗卫生数据开展观察性研究的报告规范. 药物流行病学杂志, 2019(3): 190-198, 212.

1. 刘国恩. 中国药物经济学评价指南2020. 北京: 中国市场出版社, 2020.
2. 刘国恩. 中国药物经济学评价指南导读(2020). 北京: 中国市场出版社, 2022.
3. Lu ZK, Xiong X, Lee T, et al. Big data and real-world data based cost-effectiveness studies and decision-making models: a systematic review and analysis. Front Pharmacol, 2021, 12: 700012.
4. Caro JJ, Briggs AH, Siebert U, et al. Modeling good research practices--overview: a report of the ISPOR-SMDM Modeling Good Research Practices Task Force-1. Value Health, 2012, 15(6): 796-803.
5. Thokala P, Dodd P, Baalbaki H, et al. Developing Markov models from real-world data: a case study of heart failure modeling using administrative data. Value Health, 2020, 23(6): 743-750.
6. Xia C, Dong X, Li H, et al. Cancer statistics in China and United States, 2022: profiles, trends, and determinants. Chin Med J (Engl), 2022, 135(5): 584-590.
7. National Institute for Health and Clinical Excellence. NICE real-world evidence framework. 2022.
8. National University of Singapore. Use of real-world data and real-world evidence to support drug reimbursement decision-making in Aisa. 2021.
9. Timbie JW, Kim AY, Concannon TW. Use of real-world evidence for regulatory approval and coverage of medical devices: a landscape assessment. Value Health, 2021, 24(12): 1792-1798.
10. Lou J, Kc S, Toh KY, et al. Real-world data for health technology assessment for reimbursement decisions in Asia: current landscape and a way forward. Int J Technol Assess Health Care, 2020, 36(5): 474-480.
11. Chay J, Fenner BJ, Finkelstein EA, et al. Real-world cost-effectiveness of anti-VEGF monotherapy and combination therapy for the treatment of polypoidal choroidal vasculopathy. Eye (Lond), 2022, 36(12): 2265-2270.
12. Hong SH, Cho JY, Kim TB, et al. Cost-effectiveness of tiotropium in elderly patients with severe asthma using real-world data. J Allergy Clin Immunol Pract, 2021, 9(5): 1939-1947.
13. Aarnio E, Korhonen MJ, Huupponen R, et al. Cost-effectiveness of statin treatment for primary prevention in conditions of real-world adherence--estimates from the Finnish prescription register. Atherosclerosis, 2015, 239(1): 240-247.
14. Canadian Agency for Drugs and Technologies in Health. Guidance for reporting real-world evidence. 2023.
15. Schöttler MH, Coerts FB, Postma MJ, et al. The effect of the drug life cycle price on cost-effectiveness: case studies using real-world pricing data. Value Health, 2023, 26(1): 91-98.
16. National Institute for Health and Clinical Excellence. Technology appraisal guidance. 2022.
17. Faleiros DR, Alvares-Teodoro J, Nunes da Silva E, et al. Budget impact analysis of medicines: estimated values versus real-world evidence and the implications. Expert Rev Pharmacoecon Outcomes Res, 2022, 22(2): 271-281.
18. 国家药品监督管理局药品审评中心. 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). 2021.
19. U. S. Food & Drug Administration. Use of real-world evidence to support regulatory decision-making for medical devices.
20. 聂晓璐, 王胜锋, 姚晨, 等. 国内外真实世界数据/证据的相关药械监管政策法规及指导原则比较研究. 药物流行病学杂志, 2022, 31(1): 5-11.
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. 国家药品监督管理局. 关于发布真实世界数据用于医疗器械临床评价技术指导原则(试行)的通告(2020年第77号). 2020.
23. 聂晓路, 彭晓霞. 使用常规收集卫生数据开展观察性研究的报告规范. 中国循证医学杂志, 2017, 17(4): 475-487.
24. Bowrin K, Briere JB, Levy P, et al. Use of real-world evidence in meta-analyses and cost-effectiveness models. J Med Econ, 2020, 23(10): 1053-1060.
25. Hampson G, Towse A, Dreitlein WB, et al. Real-world evidence for coverage decisions: opportunities and challenges. J Comp Eff Res, 2018, 7(12): 1133-1143.
26. 高培, 王杨, 罗剑锋, 等. 基于真实世界数据评价治疗结局研究的统计分析技术规范. 中国循证医学杂志, 2019, 19(7): 787-793.
27. 聂晓璐, 武泽昊, 赵厚宇, 等. 使用常规收集医疗卫生数据开展观察性研究的报告规范. 药物流行病学杂志, 2019(3): 190-198, 212.

Chinese Journal of Evidence-Based Medicine

Research on techniques of economic evaluations of drugs by using the real-world data

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