Research on the application strategy of transforming real-world clinical research data into evidence

LI Yu-jia; CHEN Chui-xiong; HUANG Li-hong; WANG Min-hui; LI Chen; QIU Jing-jun; ZHAO Yang; XIE Hai-tang; WANG Ling

doi:10.12025/j.issn.1008-6358.2021.20212080

Chin J Clin Med > 2021 > 28(5): 744-750. > DOI: 10.12025/j.issn.1008-6358.2021.20212080

LI Yu-jia, CHEN Chui-xiong, HUANG Li-hong, et al. Research on the application strategy of transforming real-world clinical research data into evidence[J]. Chin J Clin Med, 2021, 28(5): 744-750. DOI: 10.12025/j.issn.1008-6358.2021.20212080

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Research on the application strategy of transforming real-world clinical research data into evidence

LI Yu-jia^{1, 2,},
CHEN Chui-xiong²,
HUANG Li-hong^{3, 7},
WANG Min-hui^{4, 7},
LI Chen^{2, 7},
QIU Jing-jun^{5, 7},
ZHAO Yang^{6, 7},
XIE Hai-tang^{4, 7},
WANG Ling^{2, 7, ,}

1.
School of Public Health, Shaanxi University of Chinese Medicine, Xi'an 710032, Shaanxi, China
2.
Department of Health Statistics, Air Military Medical University, Xi'an 710032, Shaanxi, China
3.
Department of Biostatistics, Zhongshan Hospital, Fudan University, Shanghai 200032, China
4.
The First Affiliated Hospital of Wannan Medical College, Wuhu 241000, Anhui, China
5.
Beijing Technology Ltd, FOSUN Pharmacy, Beijing 100005, China
6.
Department of Biostatistics, School of Public Health, Nanjing Medical University, Nanjing 211166, Jiangsu, China
7.
CSCO Biostatistics Expert Committee RWE Working Group

Funds:

National Natural Science Foundation of China 81773553

National Natural Science Foundation of China 81903407

National Natural Science Foundation of China 81803328

National Natural Science Foundation of China 81973141

More Information

Received Date: September 06, 2021
Accepted Date: September 23, 2021

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Abstract

Abstract

By consulting the published policies and regulations and reviewing the literature about the related expert consensus and guidelines on real-world research, we summarized the current status of real-world clinical researches which have been conducted at home and abroad, generalized the views on the transformation of real-world clinical research evidence in different fields combined with typical cases, and then provided supportive strategies for the application of real-world evidence generated by real-world data.
- real-world study,
- real-world data,
- real-world evidence,
- application strategy

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References (33)

References

[1]	SHERMAN R E, ANDERSON S A, DAL PAN G J, et al. Real-world evidence-what is it and what can it tell us?[J]. N Engl J Med, 2016, 375(23): 2293-2297. DOI: 10.1056/NEJMsb1609216
[2]	KAPLAN N M, SPROUL L E, MULCAHY W S. Large prospective study of ramipril in patients with hypertension. CARE Investigators[J]. Clin Ther, 1993, 15(5): 810-818.
[3]	Food and Drug Administration. Use of real-world evidence to support regulatory decision-making for medical devices: draft guidance for industry and Food and Drug Administration staff[EB/OL]. https://www.fda.gov/media/99447/download.
[4]	Network for Excellence in Health Innovation. Real world evidence: a new era for health care innovation[EB/OL]. https://www.nehi-us.org/publications/66-real-world-evidence-a-new-era-for-health-care-innovation/view.
[5]	US. Government. 21st century cures act[EB/OL]. https://www.congress.gov/bill/114th-congress/housebill/6?q=%7B%22search%22%3A%2221st+Century+Cures+Act%22%7D&s=8&r=5.
[6]	CAVE A, BRUN N C, SWEENEY F, et al. Bigdata-how to realize the promise[J]. Clin Pharmacol Ther, 2020, 107: 753-761. DOI: 10.1002/cpt.1736
[7]	BRECKENRIDGE A M, BRECKENRIDGE R A, PECK C C. Report on the current status of the use of real-world data (RWD) and real-world evidence (RWE) in drug development and regulation[J]. Br J Clin Pharmacol, 2019, 85(9): 1874-1877. DOI: 10.1111/bcp.14026
[8]	CENTER FOR DRUG EVALUATION, NMPA. Real-world data guidelines for generating real-world evidence (Trial version)[EB/OL]. https://www.cde.org.cn/main/news/viewInfoCommon/2a1c437ed54e7b838a7e86f4ac21c539.
[9]	WANG M H, ZHAO Y, DENG Y Z, et al. Comparison of policies and guidelines regarding using of real world data/real word evidence[J]. Chin J Clin Pharmacol Ther, 2020, 25(8): 878-889.
[10]	ROBERT HONIGBERG M, NEIL BELSON J. Contemporary applications of real-world evidence in regulatory decision making: a case series review[EB/OL]. https://medicalaffairs.org/real-world-evidence-regulatory-decision-making/.
[11]	边蕾, 赵频, 杨悦. FDA前哨行动运行情况研究与启示[J]. 中国药物警戒, 2013, 10(1): 25-29. DOI: 10.3969/j.issn.1672-8629.2013.01.008 BIAN L, ZHAO P, YANG Y. The Research of operating conditions of FDA sentinel initiative and enlightenment[J]. Chinese Journal of Pharmacovigilance, 2013, 10(1): 25-29. DOI: 10.3969/j.issn.1672-8629.2013.01.008
[12]	RATH A, SALAMON V, PEIXOTO S, et al. A systematic literature review of evidence-based clinical practice for rare diseases: what are the perceived and real barriers forimproving the evidence and how can they be overcome?[J]. Trials, 2017, 18(1): 556. DOI: 10.1186/s13063-017-2287-7
[13]	孙宇昕, 魏芬芳, 杨悦. 真实世界证据用于药械监管与卫生决策的机遇与挑战[J]. 中国药物警戒, 2017, 14(6): 353-358. DOI: 10.3969/j.issn.1672-8629.2017.06.009 SUN Y X, WEI F F, YANG Y. Opportunities and challenges for real-world evidence applied in drug regulation and health decision making[J]. Chinese Journal of Pharmacovigilance, 2017, 14(6): 353-358. DOI: 10.3969/j.issn.1672-8629.2017.06.009
[14]	LI M, CHEN S, LAI Y, et al. Integrating real-world evidence in the regulatory decision-making process: a systematic analysis of experiences in the US, EU, and China using a logic model[J]. Front Med (Lausanne), 2021, 8: 669509.
[15]	吴宏辉, 宋海波, 张力, 等. 欧盟药品上市后有效性研究管理制度简介[J]. 药物评价研究, 2021, 44(6): 1134-1140. https://www.cnki.com.cn/Article/CJFDTOTAL-YWPJ202106002.htm WU H H, SONG H B, ZHANG L, et al. Introduction of post-authorisation efficacy study management system in European Union[J]. Drug Evaluation Research, 2021, 44(6): 1134-1140. https://www.cnki.com.cn/Article/CJFDTOTAL-YWPJ202106002.htm
[16]	莫成林, 连桂玉. 真实世界研究支持孤儿药审评审批的质量风险考量[J]. 质量与市场, 2020, (16): 62-64. https://www.cnki.com.cn/Article/CJFDTOTAL-ZLSC202016022.htm MO C L, LIAN G Y. Quality risk consideration of orphan drug review and approval supported by real-world research[J]. Quality and market, 2020, (16): 62-64. https://www.cnki.com.cn/Article/CJFDTOTAL-ZLSC202016022.htm
[17]	NMPA. Technical guidelines for clinical evaluation of medical devices using real-world data. (Trial version)[EB/OL]. https://www.nmpa.gov.cn/xxgk/ggtg/qtggtg/20201126090030150.html.
[18]	刘露, 钟佑锦, 张宇晶, 等. 真实世界证据与医疗器械上市前临床评价: 思考与建议[J]. 中国循证医学杂志, 2019, 19(8): 883-886. https://www.cnki.com.cn/Article/CJFDTOTAL-ZZXZ201908001.htm LIU L, ZHONG Y J, ZHANG Y J, et al. The consideration and suggestion of real-world evidence and pre-marketing evaluation of medical devices[J]. Chinese Journal of Evidence-Based Medicine, 2019, 19(8): 883-886. https://www.cnki.com.cn/Article/CJFDTOTAL-ZZXZ201908001.htm
[19]	任燕, 梁远波, 刘密密, 等. 构建博鳌乐城创新药械真实世界数据研究体系: 基于我国首个使用境内真实世界数据获批上市的医疗器械研究的探讨[J]. 中国循证医学杂志, 2020, 20(10): 1117-1123. https://www.cnki.com.cn/Article/CJFDTOTAL-ZZXZ202010001.htm REN Y, LIANG Y B, LIU M M, et al. Exploration and practice of real-world data studies on innovative medical products in Boao Lecheng: analysis based on Chinese first case of approved medical device using domestic real-world data[J]. Chinese Journal of Evidence-Based Medicine, 2020, 20(10): 1117-1123. https://www.cnki.com.cn/Article/CJFDTOTAL-ZZXZ202010001.htm
[20]	王雪, 杨悦. 真实世界证据应用于儿科医药产品研发中的挑战及对策研究[J]. 中国药房, 2021, 32(10): 1165-1172. DOI: 10.6039/j.issn.1001-0408.2021.10.03 WANG X, YANG Y. Research on challenges and policies of real-world evidence applied in pediatric medical product R & D[J]. China Pharmacy, 2021, 32(10): 1165-1172. DOI: 10.6039/j.issn.1001-0408.2021.10.03
[21]	BEVANS K B, RILEY A W, MOON J, et al. Conceptual and methodological advances in child-reported outcomes measurement[J]. Expert Rev Pharmacoecon Outcomes Res, 2010, 10(4): 385-396. DOI: 10.1586/erp.10.52
[22]	CARPENTER D, GONZALEZ D, RETSCH-BOGART G, et al. Methodological and ethical issues in pediatric medication safety research[J]. Pediatrics, 2017, 140(3): e20170195. DOI: 10.1542/peds.2017-0195
[23]	史源, 陈妍如, 陈龙. 真实世界研究在新生儿医学中的应用[J]. 中国当代儿科杂志, 2018, 20(3): 169-173. https://www.cnki.com.cn/Article/CJFDTOTAL-DDKZ201803001.htm SHI Y, CHEN Y R, CHEN L. Application of real world study in neonatal medicine[J]. Chin J Contemp Pediatr, 2018, 20(3): 169-173. https://www.cnki.com.cn/Article/CJFDTOTAL-DDKZ201803001.htm
[24]	李晓春, 戴国华, 刘小翠. 基于HIS真实世界研究方法及其在中医临床疗效评价中的应用[J]. 山东中医药大学学报, 2016, 40(5): 415-418. https://www.cnki.com.cn/Article/CJFDTOTAL-SDYX201605006.htm LI X C, DAI G H, LIU X C. Real world study methods and its application in clinical efficacy evaluation of traditional Chinese medicine based on HIS[J]. Journal of Shandong University of Traditional Chinese Medicine, 2016, 40(5): 415-418. https://www.cnki.com.cn/Article/CJFDTOTAL-SDYX201605006.htm
[25]	刘保延. 真实世界的中医临床科研范式[J]. 中医杂志, 2013, 54(6): 451-455. https://www.cnki.com.cn/Article/CJFDTOTAL-ZZYZ201306003.htm LIU B Y. Real-world traditional Chinese medicine clinical research paradigm[J]. Journal of Traditional Chinese Medicine, 2013, 54(6): 451-455. https://www.cnki.com.cn/Article/CJFDTOTAL-ZZYZ201306003.htm
[26]	戴亮, 季光. 基于FDA《真实世界证据计划框架》论析真实世界研究对中药新药研发的意义[J]. 中药新药与临床药理, 2019, 30(11): 1403-1408. https://www.cnki.com.cn/Article/CJFDTOTAL-ZYXY201911022.htm DAI L, JI G. Status and value of real world study on research and development of new chinese medicine-interpreting framework for FDA's real-world evidence program[J]. Traditional Chinese Drug Research & Clinical Pharmacology, 2019, 30(11): 1403-1408. https://www.cnki.com.cn/Article/CJFDTOTAL-ZYXY201911022.htm
[27]	邢永发, 王保和, 黄宇虹. 真实世界研究在中医药临床评价方面应用进展及数据质控[J]. 环球中医药, 2018, 11(4): 625-630. DOI: 10.3969/j.issn.1674-1749.2018.04.040 XING Y F, WANG B H, HUANG Y H. Research progress and data quality control of application of real-world study on clinical evaluation of traditional Chinese medicine[J]. Global Traditional Chinese Medicine, 2018, 11(4): 625-630. DOI: 10.3969/j.issn.1674-1749.2018.04.040
[28]	周强, 朱彦, 朱春洋, 等. 基于真实世界的名中医经验数据挖掘思路初探[J]. 中国中西医结合消化杂志, 2017, 25(10): 793-795. https://www.cnki.com.cn/Article/CJFDTOTAL-ZXPW201710021.htm ZHOU Q, ZHU Y, ZHU C Y, et al. The method of mining clinical experience based on real-world data[J]. Chin J Integr Trad West Med Dig, 2017, 25(10): 793-795. https://www.cnki.com.cn/Article/CJFDTOTAL-ZXPW201710021.htm
[29]	XING D M, LI C X, LIU X C, et al. Problems and strategies in the field of traditional Chinese medicine real-world study[J]. CJTCMP, 2021, 36(4): 1798-1801.
[30]	JONES W S, MULDER H, WRUCK L M, et al. Comparative effectiveness of aspirin dosing in cardiovascular disease[J]. N Engl J Med, 2021, 384(21): 1981-1990. DOI: 10.1056/NEJMoa2102137
[31]	JARA A. Effectiveness of an inactivated SARS-CoV-2 vaccine in Chile[J]. N Engl J Med, 2021, 385(10): 875-884. DOI: 10.1056/NEJMoa2107715
[32]	石舒原, 赵厚宇, 周庆欣, 等. 真实世界证据与随机对照试验: RCT DUPLICATE项目方法学介绍[J]. 药物流行病学杂志, 2019, 29(3): 198-205. https://www.cnki.com.cn/Article/CJFDTOTAL-YWLX202003012.htm SHI S Y, ZHAO H Y, ZHOU Q X, et al. Real-world evidence and randomized controlled trials: the methodology of RCT DUPLICATE[J]. Chin J Pharmacoepidemiol, 2019, 29(3): 198-205. https://www.cnki.com.cn/Article/CJFDTOTAL-YWLX202003012.htm
[33]	孙鑫, 张俊华, 王雯, 等. 制定中国中成药真实世界研究技术指导原则, 促进中成药临床研究向决策证据转化[J]. 中国循证医学杂志, 2020, 20(9): 993-999. https://www.cnki.com.cn/Article/CJFDTOTAL-ZZXZ202009002.htm SUN X, ZHANG J H, WANG W, et al. Formulate technical guidance for real-world studies of traditional Chinese medicine in China to promote the transformation of clinical research of Chinese patent medicine into decision evidence[J]. Chinese Journal of Evidence-Based Medicine, 2020, 20(9): 993-999. https://www.cnki.com.cn/Article/CJFDTOTAL-ZZXZ202009002.htm

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Research on the application strategy of transforming real-world clinical research data into evidence

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