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

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.	國家藥品監督管理局. 國家藥監局關于發布真實世界證據支持藥物研發與審評的指導原則(試行)的通告(2020 年第 1 號). Available from: https://www.nmpa.gov.cn/yaopin/ypggtg/ypqtgg/20200107151901190.html.
7.	國家藥品監督管理局醫療器械技術審評中心. 關于公開征求《真實世界數據用于醫療器械臨床評價技術指導原則(征求意見稿)》意見的通知. 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.	孫鑫, 譚婧, 唐立, 等. 重新認識真實世界研究. 中國循證醫學雜志, 2017, 17(2): 126-130.
10.	王雯, 劉艷梅, 譚婧, 等. 回顧性數據庫研究的概念、策劃與研究數據庫構建. 中國循證醫學雜志, 2018, 18(2): 230-237.
11.	譚婧, 程亮亮, 王雯, 等. 患者登記研究的策劃與患者登記數據庫構建: 基于觀察性設計的真實世界研究. 中國循證醫學雜志, 2017, 17(12): 1365-1372.
12.	唐立, 康德英, 喻佳潔, 等. 實效性隨機對照試驗: 真實世界研究的重要設計. 中國循證醫學雜志, 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.	國家衛生計生委辦公廳, 國家中醫藥管理局辦公室. 關于印發電子病歷應用管理規范(試行)的通知. Available from: http://www.nhc.gov.cn/mohwsbwstjxxzx/s8553/201702/fb49f9487d884645b7247218b764bba3.shtml.
17.	王雯, 高培, 吳晶, 等. 構建基于既有健康醫療數據的研究型數據庫技術規范. 中國循證醫學雜志, 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.	譚婧, 彭曉霞, 舒嘯塵, 等. 患者登記數據庫構建技術規范. 中國循證醫學雜志, 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.	彭曉霞, 舒嘯塵, 譚婧, 等. 基于真實世界數據評價治療結局的觀察性研究設計技術規范. 中國循證醫學雜志, 2019, 19(7): 779-786.
24.	溫澤淮, 李玲, 劉艷梅, 等. 實效性隨機對照試驗的技術規范. 中國循證醫學雜志, 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, 王楊, 羅劍鋒, 等. 基于真實世界數據評價治療結局研究的統計分析技術規范. 中國循證醫學雜志, 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. 國家藥品監督管理局. 國家藥監局關于發布真實世界證據支持藥物研發與審評的指導原則(試行)的通告(2020 年第 1 號). Available from: https://www.nmpa.gov.cn/yaopin/ypggtg/ypqtgg/20200107151901190.html.
7. 國家藥品監督管理局醫療器械技術審評中心. 關于公開征求《真實世界數據用于醫療器械臨床評價技術指導原則(征求意見稿)》意見的通知. 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. 孫鑫, 譚婧, 唐立, 等. 重新認識真實世界研究. 中國循證醫學雜志, 2017, 17(2): 126-130.
10. 王雯, 劉艷梅, 譚婧, 等. 回顧性數據庫研究的概念、策劃與研究數據庫構建. 中國循證醫學雜志, 2018, 18(2): 230-237.
11. 譚婧, 程亮亮, 王雯, 等. 患者登記研究的策劃與患者登記數據庫構建: 基于觀察性設計的真實世界研究. 中國循證醫學雜志, 2017, 17(12): 1365-1372.
12. 唐立, 康德英, 喻佳潔, 等. 實效性隨機對照試驗: 真實世界研究的重要設計. 中國循證醫學雜志, 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. 國家衛生計生委辦公廳, 國家中醫藥管理局辦公室. 關于印發電子病歷應用管理規范(試行)的通知. Available from: http://www.nhc.gov.cn/mohwsbwstjxxzx/s8553/201702/fb49f9487d884645b7247218b764bba3.shtml.
17. 王雯, 高培, 吳晶, 等. 構建基于既有健康醫療數據的研究型數據庫技術規范. 中國循證醫學雜志, 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. 譚婧, 彭曉霞, 舒嘯塵, 等. 患者登記數據庫構建技術規范. 中國循證醫學雜志, 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. 彭曉霞, 舒嘯塵, 譚婧, 等. 基于真實世界數據評價治療結局的觀察性研究設計技術規范. 中國循證醫學雜志, 2019, 19(7): 779-786.
24. 溫澤淮, 李玲, 劉艷梅, 等. 實效性隨機對照試驗的技術規范. 中國循證醫學雜志, 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, 王楊, 羅劍鋒, 等. 基于真實世界數據評價治療結局研究的統計分析技術規范. 中國循證醫學雜志, 2019, 19(7): 787-793.

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