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：

Export PDF Favorites Scan Get Citation

Abstract Full text Figures/Tables Video References Cited by

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

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.	國家藥品監督管理局藥品審評中心. 真實世界證據支持藥物研發與審評的指導原則(試行). Available at: https://www.nmpa.gov.cn/yaopin/ypggtg/ypqtgg/20200107151901190.html.
4.	國家藥品監督管理局藥品審評中心. 真實世界研究支持兒童藥物研發與審評的技術指導原則(試行). 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.	國家藥品監督管理局. 真實世界數據用于醫療器械臨床評價技術指導原則(試行). Available at: https://www.nmpa.gov.cn/xxgk/ggtg/qtggtg/20201126090030150.html.
9.	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.
10.	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.
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, 賈玉龍, 任燕, 等. 特許醫療政策下的藥械真實世界數據研究常見設計及其關鍵考慮. 中華流行病學雜志, 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, 譚婧, 任燕, 等. 重新認識真實世界數據研究: 更新與展望. 中國循證醫學雜志, 2020, 20(11): 1241-1246.
16.	國家藥品監督管理局藥品審評中心. 用于產生真實世界證據的真實世界數據指導原則(試行). 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.
20.	黃麗紅, 魏永越, 陳峰. 如何控制觀察性療效比較研究中的混雜因素: (一)已測量混雜因素的統計學分析方法. 中華流行病學雜志, 2019, 40(10): 1304-1309.
21.	黃麗紅, 魏永越, 陳峰. 如何控制觀察性療效比較研究中的混雜因素: (二)未知或未測量混雜因素的統計學分析方法. 中華流行病學雜志, 2019, 40(11): 1450-1455.
22.	黃麗紅, 趙楊, 魏永越, 等. 如何控制觀察性療效比較研究中的混雜因素: (三)混雜因素控制的敏感性分析方法. 中華流行病學雜志, 2019, 40(12): 1645-1649.
23.	國家藥品監督管理局, 國家衛生健康委員會. 藥物臨床試驗質量管理規范. Available at: https://www.nmpa.gov.cn/xxgk/ggtg/qtggtg/20200426162401243.html.
24.	張海洪, 姚晨, 李海燕. 真實世界研究的倫理挑戰與倫理審查. 中國食品藥品監管, 2020, (11): 56-63.
25.	姚賀之, 孫明月, 高蕊, 等. 基于CIOMS準則的真實世界研究倫理問題探討. 中國醫學倫理學, 2019, 32(5): 559-563.
26.	中華人民共和國中央人民政府. 中華人民共和國醫師法. 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. 國家藥品監督管理局藥品審評中心. 真實世界證據支持藥物研發與審評的指導原則(試行). Available at: https://www.nmpa.gov.cn/yaopin/ypggtg/ypqtgg/20200107151901190.html.
4. 國家藥品監督管理局藥品審評中心. 真實世界研究支持兒童藥物研發與審評的技術指導原則(試行). 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. 國家藥品監督管理局. 真實世界數據用于醫療器械臨床評價技術指導原則(試行). Available at: https://www.nmpa.gov.cn/xxgk/ggtg/qtggtg/20201126090030150.html.
9. 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.
10. 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.
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, 賈玉龍, 任燕, 等. 特許醫療政策下的藥械真實世界數據研究常見設計及其關鍵考慮. 中華流行病學雜志, 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, 譚婧, 任燕, 等. 重新認識真實世界數據研究: 更新與展望. 中國循證醫學雜志, 2020, 20(11): 1241-1246.
16. 國家藥品監督管理局藥品審評中心. 用于產生真實世界證據的真實世界數據指導原則(試行). 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.
20. 黃麗紅, 魏永越, 陳峰. 如何控制觀察性療效比較研究中的混雜因素: (一)已測量混雜因素的統計學分析方法. 中華流行病學雜志, 2019, 40(10): 1304-1309.
21. 黃麗紅, 魏永越, 陳峰. 如何控制觀察性療效比較研究中的混雜因素: (二)未知或未測量混雜因素的統計學分析方法. 中華流行病學雜志, 2019, 40(11): 1450-1455.
22. 黃麗紅, 趙楊, 魏永越, 等. 如何控制觀察性療效比較研究中的混雜因素: (三)混雜因素控制的敏感性分析方法. 中華流行病學雜志, 2019, 40(12): 1645-1649.
23. 國家藥品監督管理局, 國家衛生健康委員會. 藥物臨床試驗質量管理規范. Available at: https://www.nmpa.gov.cn/xxgk/ggtg/qtggtg/20200426162401243.html.
24. 張海洪, 姚晨, 李海燕. 真實世界研究的倫理挑戰與倫理審查. 中國食品藥品監管, 2020, (11): 56-63.
25. 姚賀之, 孫明月, 高蕊, 等. 基于CIOMS準則的真實世界研究倫理問題探討. 中國醫學倫理學, 2019, 32(5): 559-563.
26. 中華人民共和國中央人民政府. 中華人民共和國醫師法. 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

Abstract Full text Figures/Tables Video References Cited by

Previous Article

Next Article

Format

Content