Real-world data studies: update and future development_Chinese Journal of Evidence-Based Medicine

Authors：

WANG Wen ¹ , TAN Jing ¹ , REN Yan ¹ , LI Ling ¹ , SHEN Chuanyong ² , TIAN Chunhua ² , SONG Haibo ² , WANG Tao ² , GAO Pei ³ , PENG Xiaoxia ⁴ , WEN Zehuai ⁵ , SHU Xiaochen ⁶ , WANG Li ⁷ , LIU Mei ¹ , HE Qiao ¹ , ZOU Kang ¹ ,  SUN Xin ¹

1. Chinese Evidence-based Medicine Center, West China Hospital, Sichuan University, Chengdu 610041, P.R.China;
2. Center for Drug Reevaluation, National Medical Products Administration, Beijing 100000, P.R.China;
3. Department of Epidemiology and Biostatistics, Peking University Health Science Center, Beijing 100191, P.R.China;
4. Center for Clinical Epidemiology and Evidence-based Medicine, Beijing Children’s Hospital, Capital Medical University, Beijing 100045, P.R.China;
5. Key Unit of Methodology in Clinical Research, Guangdong Provincial Hospital of Chinese Medicine, Guangzhou 510120, P.R.China;
6. Department of Epidemiology and Biostatistics, School of Public Health, Medical College of Soochow University, Suzhou 215123, P.R.China;
7. Institute of Basic Medical Sciences, Chinese Academy of Medical Sciences, Beijing 100005, P.R.China;

Corresponding author：

SUN Xin, Email: sunx79@hotmail.com

Keywords：

Real-world data and studies; Research data infrastructure; Standardization

DOI：

10.7507/1672-2531.202006183

Video：

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Real-world data studies have experienced rapid development in recent years, however, misunderstandings persist. This paper aims to improve practice and promote standardization by updating the categorization of real-world data, proposing two conceptual frameworks for conducting real-world data studies, developing the concepts of research data infrastructure and clarifying the misconceptions on registry database, and discussing future development.

Citation： WANG Wen, TAN Jing, REN Yan, LI Ling, SHEN Chuanyong, TIAN Chunhua, SONG Haibo, WANG Tao, GAO Pei, PENG Xiaoxia, WEN Zehuai, SHU Xiaochen, WANG Li, LIU Mei, HE Qiao, ZOU Kang, SUN Xin. Real-world data studies: update and future development. Chinese Journal of Evidence-Based Medicine, 2020, 20(11): 1241-1246. doi: 10.7507/1672-2531.202006183 Copy

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2.	U.S. Food & Drug Administration. Use of real-world evidence to support regulatory decision-making for medical devices. Available from: https://www.fda.gov/media/99447/download.
3.	Sherman RE, Anderson SA, Dal Pan GJ, et al. Real-world evidence - what is it and what can it tell us? N Engl J Med, 2016, 375(23): 2293-2297.
4.	U.S. Food & Drug Administration. Best practices for conducting and reporting pharmacoepidemiologic safety studies using electronic healthcare data. Available from: https://www.fda.gov/media/79922/download.
5.	Sun X, Tan J, Tang L, et al. Real world evidence: experience and lessons from China. BMJ, 2018, 360: j5262.
6.	国家药品监督管理局. 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). Available from: https://www.nmpa.gov.cn/yaopin/ypggtg/ypqtgg/20200107151901190.html.
7.	国家药品监督管理局医疗器械技术审评中心. 关于公开征求《真实世界数据用于医疗器械临床评价技术指导原则(征求意见稿)》意见的通知. Available from: https://www.cmde.org.cn/CL0101/20139.html.
8.	Ponce Guevara LV, Laffond Yges E, Gracia Bara MT, et al. Adherence to anaphylaxis guidelines: Real-world data from the emergency department of a tertiary hospital. J Investig Allergol Clin Immunol, 2018, 28(4): 246-252.
9.	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.
10.	王雯, 刘艳梅, 谭婧, 等. 回顾性数据库研究的概念、策划与研究数据库构建. 中国循证医学杂志, 2018, 18(2): 230-237.
11.	谭婧, 程亮亮, 王雯, 等. 患者登记研究的策划与患者登记数据库构建: 基于观察性设计的真实世界研究. 中国循证医学杂志, 2017, 17(12): 1365-1372.
12.	唐立, 康德英, 喻佳洁, 等. 实效性随机对照试验: 真实世界研究的重要设计. 中国循证医学杂志, 2017, 217(17): 999-1004.
13.	Agency for Healthcare Research and Quality. Registries for evaluating patient outcomes: a user's guide (3rd edition). AHRQ Publication, 2014. Available from: https://effectivehealthcare.ahrq.gov/products/registries-guide-3rd-edition/research.
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). BMJ, 2018, 363: k3532.
15.	ISPOR Connection. Real-life data: a growing need. Available from: http://www.ispor.org/News/articles/Oct07/RLD.asp.
16.	国家卫生计生委办公厅, 国家中医药管理局办公室. 关于印发电子病历应用管理规范(试行)的通知. Available from: http://www.nhc.gov.cn/mohwsbwstjxxzx/s8553/201702/fb49f9487d884645b7247218b764bba3.shtml.
17.	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.
18.	Benchimol EI, Smeeth L, Guttmann A, et al. The reporting of studies conducted using observational routinely-collected health data (RECORD) statement. PLoS Med, 2015, 12(10): e1001885.
19.	Wang W, Zhu S, He Q, et al. Developing a registry of healthcare-associated infections at intensive care units in west china: study rationale and patient characteristics. Clin Epidemiol, 2019, 11: 1035-1045.
20.	Wang W, Liu Y, Yu C, et al. Cefoperazone-sulbactam and risk of coagulation disorders or bleeding: a retrospective cohort study. Expert Opin Drug Saf, 2020, 19(3): 339-347.
21.	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.
22.	Schmidt M, Schmidt SA, Sandegaard JL, et al. The Danish national patient registry: a review of content, data quality, and research potential. Clin Epidemiol, 2015, 7: 449-490.
23.	彭晓霞, 舒啸尘, 谭婧, 等. 基于真实世界数据评价治疗结局的观察性研究设计技术规范. 中国循证医学杂志, 2019, 19(7): 779-786.
24.	温泽淮, 李玲, 刘艳梅, 等. 实效性随机对照试验的技术规范. 中国循证医学杂志, 2019, 19(7): 794-802.
25.	Hudson M, Avina-Zubieta A, Lacaille D, et al. The validity of administrative data to identify hip fractures is high - a systematic review. J Clin Epidemiol, 2013, 66(3): 278-285.
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.

1. U.S. Food & Drug Administration. Framework for FDA’s real-world evidence progarm. Available from: https://www.fda.gov/media/120060/download.
2. U.S. Food & Drug Administration. Use of real-world evidence to support regulatory decision-making for medical devices. Available from: https://www.fda.gov/media/99447/download.
3. Sherman RE, Anderson SA, Dal Pan GJ, et al. Real-world evidence - what is it and what can it tell us? N Engl J Med, 2016, 375(23): 2293-2297.
4. U.S. Food & Drug Administration. Best practices for conducting and reporting pharmacoepidemiologic safety studies using electronic healthcare data. Available from: https://www.fda.gov/media/79922/download.
5. Sun X, Tan J, Tang L, et al. Real world evidence: experience and lessons from China. BMJ, 2018, 360: j5262.
6. 国家药品监督管理局. 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). Available from: https://www.nmpa.gov.cn/yaopin/ypggtg/ypqtgg/20200107151901190.html.
7. 国家药品监督管理局医疗器械技术审评中心. 关于公开征求《真实世界数据用于医疗器械临床评价技术指导原则(征求意见稿)》意见的通知. Available from: https://www.cmde.org.cn/CL0101/20139.html.
8. Ponce Guevara LV, Laffond Yges E, Gracia Bara MT, et al. Adherence to anaphylaxis guidelines: Real-world data from the emergency department of a tertiary hospital. J Investig Allergol Clin Immunol, 2018, 28(4): 246-252.
9. 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.
10. 王雯, 刘艳梅, 谭婧, 等. 回顾性数据库研究的概念、策划与研究数据库构建. 中国循证医学杂志, 2018, 18(2): 230-237.
11. 谭婧, 程亮亮, 王雯, 等. 患者登记研究的策划与患者登记数据库构建: 基于观察性设计的真实世界研究. 中国循证医学杂志, 2017, 17(12): 1365-1372.
12. 唐立, 康德英, 喻佳洁, 等. 实效性随机对照试验: 真实世界研究的重要设计. 中国循证医学杂志, 2017, 217(17): 999-1004.
13. Agency for Healthcare Research and Quality. Registries for evaluating patient outcomes: a user's guide (3rd edition). AHRQ Publication, 2014. Available from: https://effectivehealthcare.ahrq.gov/products/registries-guide-3rd-edition/research.
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). BMJ, 2018, 363: k3532.
15. ISPOR Connection. Real-life data: a growing need. Available from: http://www.ispor.org/News/articles/Oct07/RLD.asp.
16. 国家卫生计生委办公厅, 国家中医药管理局办公室. 关于印发电子病历应用管理规范(试行)的通知. Available from: http://www.nhc.gov.cn/mohwsbwstjxxzx/s8553/201702/fb49f9487d884645b7247218b764bba3.shtml.
17. 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.
18. Benchimol EI, Smeeth L, Guttmann A, et al. The reporting of studies conducted using observational routinely-collected health data (RECORD) statement. PLoS Med, 2015, 12(10): e1001885.
19. Wang W, Zhu S, He Q, et al. Developing a registry of healthcare-associated infections at intensive care units in west china: study rationale and patient characteristics. Clin Epidemiol, 2019, 11: 1035-1045.
20. Wang W, Liu Y, Yu C, et al. Cefoperazone-sulbactam and risk of coagulation disorders or bleeding: a retrospective cohort study. Expert Opin Drug Saf, 2020, 19(3): 339-347.
21. 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.
22. Schmidt M, Schmidt SA, Sandegaard JL, et al. The Danish national patient registry: a review of content, data quality, and research potential. Clin Epidemiol, 2015, 7: 449-490.
23. 彭晓霞, 舒啸尘, 谭婧, 等. 基于真实世界数据评价治疗结局的观察性研究设计技术规范. 中国循证医学杂志, 2019, 19(7): 779-786.
24. 温泽淮, 李玲, 刘艳梅, 等. 实效性随机对照试验的技术规范. 中国循证医学杂志, 2019, 19(7): 794-802.
25. Hudson M, Avina-Zubieta A, Lacaille D, et al. The validity of administrative data to identify hip fractures is high - a systematic review. J Clin Epidemiol, 2013, 66(3): 278-285.
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.

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