Application of Real-World Evidence in the Approval of Antitumor Drugs

摘要/Abstract

摘要： Objective To study the information of real-world evidence (RWE) that can make up the deficiency of RCT results, the unfeasibility of some trials and the possible ethical problems, and to promote the approval of RWE for anti-tumor drugs. Methods Literature analysis, policy analysis and comparative analysis were used. Results and Conclusion The studies and application of RWE in the United States, the European Union, and China were reviewed. The approval of anti-tumor drugs supported by RWE was generally used for external control groups in single arm trials, clinical efficacy and safety of adjuvant support drugs, or background analysis. The types of approvals included innovative anti-tumor drugs, supplemented indications of the approved drugs, expanding patient population, and combination medication. This study can provide reference for the approval of anti-tumor drugs using RWE in China. It is recommended to combine traditional RCT with RWE to promote the approval of anti-tumor drugs.

关键词: real-world evidence, antitumor drugs, approval

Abstract: Objective To study the information of real-world evidence (RWE) that can make up the deficiency of RCT results, the unfeasibility of some trials and the possible ethical problems, and to promote the approval of RWE for anti-tumor drugs. Methods Literature analysis, policy analysis and comparative analysis were used. Results and Conclusion The studies and application of RWE in the United States, the European Union, and China were reviewed. The approval of anti-tumor drugs supported by RWE was generally used for external control groups in single arm trials, clinical efficacy and safety of adjuvant support drugs, or background analysis. The types of approvals included innovative anti-tumor drugs, supplemented indications of the approved drugs, expanding patient population, and combination medication. This study can provide reference for the approval of anti-tumor drugs using RWE in China. It is recommended to combine traditional RCT with RWE to promote the approval of anti-tumor drugs.

Key words: real-world evidence, antitumor drugs, approval

Shang Ya’nan, Huang Zhe. Application of Real-World Evidence in the Approval of Antitumor Drugs[J]. 亚洲社会药学, 2024, 19(4): 311-318.

Shang Ya’nan, Huang Zhe. Application of Real-World Evidence in the Approval of Antitumor Drugs[J]. Asian Journal of Social Pharmacy, 2024, 19(4): 311-318.

[1]	Li Yuan, Wang Yijie, Wang Shuling. Research Progress and Suggestions on China’s Drug Registration Management Based on CiteSpace Knowledge Maps [J]. 亚洲社会药学, 2024, 19(3): 216-224.
[2]	Xiao Feiyi, Zhang Fang, Li Xue, Dong Li. Current Situation and Prospect of the Application of Real-World Evidence in Health Care[J]. 亚洲社会药学, 2023, 18(4): 374-386.
[3]	Li He, Huang Zhe. Research and Enlightenment of Accelerated Approval Pathway in China [J]. 亚洲社会药学, 2023, 18(1): 1-7.
[4]	Lv Xiaowen, Huang Zhe. Present Situation and Enlightenment of Post-Approval Change Management of Drugs in China, USA and EU [J]. 亚洲社会药学, 2023, 18(1): 17-23.
[5]	Yang Lingling, Xu Fengxiang. EU Real-World Evidence Supports New Drug Research and Development Decisions and Its Implications for China[J]. 亚洲社会药学, 2021, 16(3): 197-202.
[6]	Lan Yipeng, Huang Zhe. Real-World Evidence Supporting New Drug Review and Approval in European Union and Its Enlightenment to China[J]. 亚洲社会药学, 2021, 16(3): 203-208.
[7]	Yu Zhongyi, Xu Fengxiang. The Enlightenment of EU Real-World Evidence Supporting the Inclusion of Rare Disease Drugs in Medical Insurance Decisions to China[J]. 亚洲社会药学, 2021, 16(3): 209-213.
[8]	Wang JiaoJiao, Huang Zhe. EU Real-World Evidence Supports the Expansion Indications for Drugs and Its Enlightenment to China[J]. 亚洲社会药学, 2021, 16(3): 214-218.
[9]	Wang Chao, Lian Guiyu. Research on Real-World Evidence and Application in EU and Its Enlightment to China[J]. 亚洲社会药学, 2021, 16(3): 219-224.
[10]	Yu Xiuna, Li Wanying, Dong Li. The Enlightenment of FDA’s “Real-World Evidence Program Framework” to China[J]. 亚洲社会药学, 2021, 16(3): 225-229.
[11]	Huang Xianqin, Chen Chuhan, Dong Li. Application of Real-World Evidence in Regulatory Decision-Making for Medical Devices[J]. 亚洲社会药学, 2021, 16(3): 230-237.
[12]	Li Longxiao, Lian Guiyu. How Real-World Evidence Supports Healthcare Decisions in EU and Its Enlightment to China[J]. 亚洲社会药学, 2021, 16(3): 238-241.
[13]	Fu Min, Huang Zhe, Xu Fengxiang. EU Real-World Evidence Supporting Drug Regulatory Decision and Its Enlightenment to China[J]. 亚洲社会药学, 2021, 16(2): 103-107.
[14]	ZHAO Jun-rui;NI Jing-yun;SI TOU Ka-weng;HU Yuan-jia*. Drug Registration and Approval System in China: CurrentStatus, Controversies and Future Perspectives[J]. , 2015, 10(4): 187-196.

亚洲社会药学