Using real-world evidence for drug and medical device evaluation and regulatory decisions_Chinese Journal of Evidence-Based Medicine

Authors：

 SUN Xin ¹ ^* , TAN Jing ¹ ^* , WANG Wen ¹ ^* , LI Ling ¹ ^* , LI Jing ¹ , LI Youping ¹ , WANG Lanming ² , HU Zengyao ³ , WU Guizhi ⁴ , WANG Jun ⁵

1. Chinese Evidence-based Medicine Centre, West China Hospital, Sichuan University, Chengdu, 610041, P.R.China;
2. Department of Medical Device Registration, National Medical Products Administration, Beijing, 100000, P.R.China;
3. Department of Drug Regulation, National Medical Products Administration, Beijing, 100000, P.R.China;
4. Center for Drug Reevaluation, National Medical Products Administration, Beijing, 100000, P.R.China;
5. Center for Drug Evaluation, National Medical Products Administration, Beijing, 100000, P.R.China;

Corresponding author：

SUN Xin, Email: sunx79@hotmail.com

Keywords：

Real-world evidence; Drugs and medical devices evaluation; Regulatory decision

DOI：

10.7507/1672-2531.201904144

Video：

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In recent years, real-world evidence data (RWD) and real-world evidence (RWE) have gained substantial attentions from healthcare practitioners and health authorities worldwide. In particular, the needs from regulatory bodies have promoted the production and use of real-world evidence. In the context of drug and device evaluation and regulation decisions, the pattern for using real world evidence may differ. This article aimed to discuss the potential uses of RWE for pre-approval clinical evaluation, post-approval monitoring and evaluation, and associated regulatory decisions, which may ultimately improve the production and use of RWE for regulatory decisions.

Citation： SUN Xin, TAN Jing, WANG Wen, LI Ling, LI Jing, LI Youping, WANG Lanming, HU Zengyao, WU Guizhi, WANG Jun. Using real-world evidence for drug and medical device evaluation and regulatory decisions. Chinese Journal of Evidence-Based Medicine, 2019, 19(5): 521-526. doi: 10.7507/1672-2531.201904144 Copy

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23.	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): 1-8.
24.	U.S. Food and Drug Administration. Exenatide (marketed as BYETTA): Acute Pancreatitis. Available at: http://www.fda.gov/Drugs/DrugSafety/DrugSafetyNewsletter/ucm119034.htm#exenatide.
25.	Dore DD, Bloomgren GL, Wenten M, et al. A cohort study of acute pancreatitis in relation to exenatide use. Diabetes Obes Metab, 2011, 13(6): 559-66.
26.	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.
27.	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): 1365-1372.
28.	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, 2017(17): 999-1004.
29.	李洪, 魏来, 郭晓蕙, 等. 真实世界研究伦理审查初探. 中国循证医学杂志, 2018, 18(11): 1198-1202.
30.	王雯, 谭婧, 于川, 等. 基于中国医院电子病历数据的药品安全性评价模式探索. 中国药物警戒, 2019, 16(3): 134-138.

1. U.S. Food & Drug Administration. FDA's Sentinel Initiative 2016. Available at: https://www.fda.gov/Safety/FDAsSentinelInitiative/default.htm.
2. U.S. Food & Drug Administration. Use of real-world evidence to support regulatory decision- making for medical devices. Available at: https://www.sogou.com/link?url=hedJjaC291MKYTE9c75enrRl1lre67CaI2smYvubWPDFTrmuzC6_HxuOpabFoY9DDAtF2Uxnf-_K7H6UfOzzMu_peCDdarnS0mCuc_NSvjlMb-O2TiRDw9Lmp63ypS7ArUC4tv2Zulat5pUc3zy0dg.
3. Oyinlola, J O, Campbell J, Kousoulis AA. Is real world evidence influencing practice? A systematic review of CPRD research in NICE guidances. BMC Health Serv Res, 2016, 16: 299.
4. Suzuki H, Shimada K, Fujiwara K. Antihypertensive effectiveness of combination therapy with losartan/hydrochlorothiazide for 'real world' management of isolated systolic hypertension. Ther Adv Cardiovasc Dis, 2015, 9(1): 10-18.
5. Barclay K, Carruthers R, Traboulsee A, et al. Best practices for long-term monitoring and follow-up of alemtuzumab-treated ms patients in real-world clinical settings. Front Neurol, 2019, 10: 253.
6. Garrison LP, Jr. 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): 326-35.
7. Hiroi S, Sugano K, Tanaka S, et al. Impact of health insurance coverage for Helicobacter pylori gastritis on the trends in eradication therapy in Japan: retrospective observational study and simulation study based on real-world data. BMJ Open, 2017, 7(7): e015855.
8. 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.
9. Stang PE, Ryan PB, Racoosin JA, et al. Advancing the science for active surveillance: rationale and design for the observational medical outcomes partnership. Ann Intern Med, 2010, 153(9): 600-606.
10. U.S. Food & Drug Administration. Framework for FDA's real world evidence program. Available at: https://www.fda.gov/downloads/ScienceResearch/SpecialTopics/RealWorldEvidence/UCM627769.pdf.
11. U.S. Food & Drug Administration. Best practice for conducting and reporting pharmacoepidemiologic safety studies using electronic healthcare data. Available at: https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/U CM243537.pdf.
12. Sun X, Li Y, Guyatt G. Available at: https://blogs.bmj.com/bmj/2018/11/06/production-of-trustworthy-real-world-data-and-evidence-in-china/.
13. 贝伐珠单抗又有新适应症获批, 真实世界数据为其提供新思路. Available at: https://www.sohu.com/a/273433437_564028.
14. Sun X, Tan J, Tang L, et al. Real world evidence: experience and lessons from China. BMJ, 2018, 360: j5262.
15. 国家药品监督管理局. 关于政协十三届全国委员会第一次会议第2489号(医疗体育类247号)提案答复的函. Available at: http://www.nmpa.gov.cn/WS04/CL2141/331878.html.
16. Han, S, Jeong HS, Kim H, et al. The treatment pattern and adherence to direct oral anticoagulants in patients with atrial fibrillation aged over 65. PLoS One, 2019, 14(4): e0214666.
17. U.S. Food & Drug Administration. Guidance for Industry and FDA Staff: Guidance for the Use of Bayesian Statistics in Medical Device Clinical Trials. Available at: https://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocu ments/ucm071121.pdf.
18. Find Drugs & Conditions. Ibrance approval expanded to include men with breast cancer. Available at: https://www.drugs.com/news/ibrance-approval-expanded-include-men-breast-cancer-81595.html.
19. Resnic FS, Majithia A, Marinac-Dabic D, et al. Registry-based prospective, active surveillance of medical-device safety. N Engl J Med, 2017, 376(6): 526-535.
20. 中华人民共和国卫生部. 药品不良反应报告和监测管理办法(卫生部令第81号). Available at: http://www.sda.gov.cn/WS01/CL0053/62621.html.
21. 国家食品药品监督管理总局安监司. 关于征求《关于推动生产企业开展药品重点监测工作的通知( 征求意见稿) 》意见的函. Available at: http://www.cfda.gov.cn/WS01/CL0778/79321.html.
22. 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.
23. 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): 1-8.
24. U.S. Food and Drug Administration. Exenatide (marketed as BYETTA): Acute Pancreatitis. Available at: http://www.fda.gov/Drugs/DrugSafety/DrugSafetyNewsletter/ucm119034.htm#exenatide.
25. Dore DD, Bloomgren GL, Wenten M, et al. A cohort study of acute pancreatitis in relation to exenatide use. Diabetes Obes Metab, 2011, 13(6): 559-66.
26. 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.
27. 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): 1365-1372.
28. 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, 2017(17): 999-1004.
29. 李洪, 魏来, 郭晓蕙, 等. 真实世界研究伦理审查初探. 中国循证医学杂志, 2018, 18(11): 1198-1202.
30. 王雯, 谭婧, 于川, 等. 基于中国医院电子病历数据的药品安全性评价模式探索. 中国药物警戒, 2019, 16(3): 134-138.

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