Key considerations for using real-world evidence to support label expansions_Chinese Journal of Evidence-Based Medicine

Authors：

JIA Yulong ^1,2,3 , YAO Minghong ^1,2,3 , XU Jiayue ^1,2,3 , WANG Yuning ^1,2,3 , LIN Kai ^2,4 , ZOU Kang ^1,2,3 ,  REN Yan ^1,2,3 ,  SUN Xin ^1,2,3

1. Chinese Evidence-based Medicine Center, West China Hospital, Sichuan University, Chengdu 610041, P. R. China;
2. NMPA Key Laboratory for Real World Data Research and Evaluation in Hainan, Chengdu 610041, P. R. China;
3. Sichuan Center of Technology Innovation for Real World Data, Chengdu 610041, P. R. China;
4. Center for Pharmacovigilance of Hainan Province, Haikou 570216, P. R. China;

Corresponding author：

REN Yan, Email: ren_yan87@163.com; SUN Xin, Email: sunxin@wchscu.cn

Keywords：

Real-world evidence; Off-label use; Label expansion; Key consideration

DOI：

10.7507/1672-2531.202206130

Video：

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With the real-world study (RWS) becoming a hotspot for clinical research, health data collected from routine clinical practice have gained increasing attention worldwide, particularly the data related to the off-label use of drugs, which have been at the forefront of clinical research in recent years. The guidance from the National Medical Products Administration has proposed that real-world evidence (RWE) can be an important consideration in supporting label expansions where randomized controlled trials are unfeasible. Nevertheless, how to use the RWE to support the approval of new or expanded indications remains unclear. This study aims to explore the structured process for the use of RWE in supporting label expansions of approved drugs, and to discuss the key considerations in such process by reviewing the documents from relevant regulatory agencies and publications from public databases, which can inform future directions for studies in this area.

Citation： JIA Yulong, YAO Minghong, XU Jiayue, WANG Yuning, LIN Kai, ZOU Kang, REN Yan, SUN Xin. Key considerations for using real-world evidence to support label expansions. Chinese Journal of Evidence-Based Medicine, 2022, 22(10): 1219-1223. doi: 10.7507/1672-2531.202206130 Copy

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7.	Dagenais S, Russo L, Madsen A, et al. Use of real-world evidence to drive drug development strategy and inform clinical trial design. Clin Pharmacol Ther, 2022, 111(1): 77-89.
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19.	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.
20.	黄丽红, 魏永越, 陈峰. 如何控制观察性疗效比较研究中的混杂因素: (一)已测量混杂因素的统计学分析方法. 中华流行病学杂志, 2019, 40(10): 1304-1309.
21.	黄丽红, 魏永越, 陈峰. 如何控制观察性疗效比较研究中的混杂因素: (二)未知或未测量混杂因素的统计学分析方法. 中华流行病学杂志, 2019, 40(11): 1450-1455.
22.	黄丽红, 赵杨, 魏永越, 等. 如何控制观察性疗效比较研究中的混杂因素: (三)混杂因素控制的敏感性分析方法. 中华流行病学杂志, 2019, 40(12): 1645-1649.
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24.	张海洪, 姚晨, 李海燕. 真实世界研究的伦理挑战与伦理审查. 中国食品药品监管, 2020, (11): 56-63.
25.	姚贺之, 孙明月, 高蕊, 等. 基于CIOMS准则的真实世界研究伦理问题探讨. 中国医学伦理学, 2019, 32(5): 559-563.
26.	中华人民共和国中央人民政府. 中华人民共和国医师法. Available at: http://www.gov.cn/xinwen/2021-08/20/content_5632496.htm.
27.	Wedam S, Fashoyin-Aje L, Bloomquist E, et al. FDA approval summary: palbociclib for male patients with metastatic breast cancer. Clin Cancer Res, 2020, 26(6): 1208-1212.
28.	Abernethy AP, Gippetti J, Parulkar R, et al. Use of electronic health record data for quality reporting. J Oncol Pract, 2017, 13(8): 530-534.
29.	Griffith SD, Tucker M, Bowser B, et al. Generating real-world tumor burden endpoints from electronic health record data: comparison of RECIST, radiology-anchored, and clinician-anchored approaches for abstracting real-world progression in non-small cell lung cancer. Adv Ther, 2019, 36(8): 2122-2136.

1. Government US. H. R. 34-114th Congress (2015-2016): 21st Century Cures Act, H. R. 34, 114th Cong. Available at: https://www.congress.gov/114/plaws/publ255/PLAW-114publ255.pdf.
2. Goble JA. The potential effect of the 21st century cures act on drug development. J Manag Care Spec Pharm, 2018, 24(7): 677-681.
3. 国家药品监督管理局药品审评中心. 真实世界证据支持药物研发与审评的指导原则(试行). Available at: https://www.nmpa.gov.cn/yaopin/ypggtg/ypqtgg/20200107151901190.html.
4. 国家药品监督管理局药品审评中心. 真实世界研究支持儿童药物研发与审评的技术指导原则(试行). Available at: https://www.nmpa.gov.cn/xxgk/ggtg/qtggtg/20200901104448101.html.
5. Franklin JM, Liaw KL, Iyasu S, et al. Real-world evidence to support regulatory decision making: new or expanded medical product indications. Pharmacoepidemiol Drug Saf, 2021, 30(6): 685-693.
6. Russell ME, Friedman MI, Mascioli SR, et al. Off-label use: an industry perspective on expanding use beyond approved indications. J Interv Cardiol, 2006, 19(5): 432-438.
7. Dagenais S, Russo L, Madsen A, et al. Use of real-world evidence to drive drug development strategy and inform clinical trial design. Clin Pharmacol Ther, 2022, 111(1): 77-89.
8. 国家药品监督管理局. 真实世界数据用于医疗器械临床评价技术指导原则(试行). Available at: https://www.nmpa.gov.cn/xxgk/ggtg/qtggtg/20201126090030150.html.
9. 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.
10. 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.
11. U. S. Food & Drug Administration. Use of real-world evidence to support regulatory decision-making for medical devices. Available at: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/use-real-world-evidence-support-regulatory-decision-making-medical-devices.
12. U. S. Food & Drug Administration. Framework for FDA’s real-world evidence program. Available at: https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence.
13. 姚明宏, 贾玉龙, 任燕, 等. 特许医疗政策下的药械真实世界数据研究常见设计及其关键考虑. 中华流行病学杂志, 2021, 42(7): 1306-1311.
14. Burcu M, Dreyer NA, Franklin JM, et al. Real-world evidence to support regulatory decision-making for medicines: considerations for external control arms. Pharmacoepidemiol Drug Saf, 2020, 29(10): 1228-1235.
15. 王雯, 谭婧, 任燕, 等. 重新认识真实世界数据研究: 更新与展望. 中国循证医学杂志, 2020, 20(11): 1241-1246.
16. 国家药品监督管理局药品审评中心. 用于产生真实世界证据的真实世界数据指导原则(试行). Available at: http://www.cde.org.cn/news.do?method=largeInfo&id=eaed86b800e8d9d9.
17. U. S. Food & Drug Administration. Real-world data: assessing electronic health records and medical claims data to support regulatory decision-making for drug and biological products. Available at: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/real-world-data-assessing-electronic-health-records-and-medical-claims-data-support-regulatory.
18. Sarri G, Patorno E, Yuan H, et al. Framework for the synthesis of non-randomised studies and randomised controlled trials: a guidance on conducting a systematic review and meta-analysis for healthcare decision making. BMJ Evid Based Med, 2022, 27(2): 109-119.
19. 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.
20. 黄丽红, 魏永越, 陈峰. 如何控制观察性疗效比较研究中的混杂因素: (一)已测量混杂因素的统计学分析方法. 中华流行病学杂志, 2019, 40(10): 1304-1309.
21. 黄丽红, 魏永越, 陈峰. 如何控制观察性疗效比较研究中的混杂因素: (二)未知或未测量混杂因素的统计学分析方法. 中华流行病学杂志, 2019, 40(11): 1450-1455.
22. 黄丽红, 赵杨, 魏永越, 等. 如何控制观察性疗效比较研究中的混杂因素: (三)混杂因素控制的敏感性分析方法. 中华流行病学杂志, 2019, 40(12): 1645-1649.
23. 国家药品监督管理局, 国家卫生健康委员会. 药物临床试验质量管理规范. Available at: https://www.nmpa.gov.cn/xxgk/ggtg/qtggtg/20200426162401243.html.
24. 张海洪, 姚晨, 李海燕. 真实世界研究的伦理挑战与伦理审查. 中国食品药品监管, 2020, (11): 56-63.
25. 姚贺之, 孙明月, 高蕊, 等. 基于CIOMS准则的真实世界研究伦理问题探讨. 中国医学伦理学, 2019, 32(5): 559-563.
26. 中华人民共和国中央人民政府. 中华人民共和国医师法. Available at: http://www.gov.cn/xinwen/2021-08/20/content_5632496.htm.
27. Wedam S, Fashoyin-Aje L, Bloomquist E, et al. FDA approval summary: palbociclib for male patients with metastatic breast cancer. Clin Cancer Res, 2020, 26(6): 1208-1212.
28. Abernethy AP, Gippetti J, Parulkar R, et al. Use of electronic health record data for quality reporting. J Oncol Pract, 2017, 13(8): 530-534.
29. Griffith SD, Tucker M, Bowser B, et al. Generating real-world tumor burden endpoints from electronic health record data: comparison of RECIST, radiology-anchored, and clinician-anchored approaches for abstracting real-world progression in non-small cell lung cancer. Adv Ther, 2019, 36(8): 2122-2136.

Chinese Journal of Evidence-Based Medicine

Key considerations for using real-world evidence to support label expansions

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