Sample size estimation methods for master protocol design and insights into their application in clinical research on syndrome-based traditional Chinese medicine_Chinese Journal of Evidence-Based Medicine

Authors：

ZHOU Luochen ¹ , ZHANG Wangjian ¹ , CHEN Xinlin ² , LIU Lanying ³ ,  LAI Yingsi ¹ , GU Jing ¹

1. School of Public Health, Sun Yat-sen University, Guangzhou 510080, P. R. China;
2. Basic Medical College, Guangzhou University of Chinese Medicine, Guangzhou 510006, P. R. China;
3. Department of Nephrology, The Third Affiliated Hospital of Beijing University of Chinese Medicine, Beijing 100029, P. R. China;

Corresponding?author：

LAI Yingsi, Email: laiys3@mail.sysu.edu.cn

Keywords：

Sample size estimation; Master protocol design; Clinical research on syndrome-based traditional Chinese medicine

DOI：

10.7507/1672-2531.202509006

Video：

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As a novel research model that can address multiple research questions within an overall trial structure, master protocol design shares similarities with the clinical research on syndrome-based traditional Chinese medicine in terms of study design. The sample size estimation in master protocol design is characterized by analyzing the subtrials separately and re-estimation at interim analyses. Specific methods include the combination of Simon’s two-stage design and Bayesian hierarchical design that facilitates information borrowing. By drawing on these methods to estimate dynamically and adjust the sample size for each subtrial in a targeted manner, it is expected to provide a feasible approach for the methodological development of sample size estimation in the field of clinical research on syndrome-based traditional Chinese medicine.

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2.	王瑞平, 肇暉, 李斌. 隨機對照臨床試驗設計要點和規范. 上海醫藥, 2022, 43(7): 72-77.
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5.	郭曉慧, 黃仟, 謝雁鳴, 等. 傘式設計與籃式設計在中醫藥循證評價中的應用. 中國中藥雜志, 2021, 46(8): 2010-2015.
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26.	馬蓮, 王可彬, 吳圣賢. 基于“理論創新”視角探討證候類中藥新藥的研發. 中國中藥雜志, 2020, 45(20): 5048-5056.
27.	李燦東, 甘慧娟, 魯玉輝, 等. 基于證素辨證原理的健康狀態辨識研究. 中華中醫藥雜志, 2011, 26(4): 754-757.
28.	賴新梅, 楊雪梅, 陳梅妹, 等. 基于證素辨識原理的證型名稱及內涵規范實踐與思考. 中華中醫藥雜志, 2017, 32(6): 2470-2472.
29.	國家藥品監督管理局藥品審評中心. 關于發布《單臂臨床試驗用于支持抗腫瘤藥上市申請的適用性技術指導原則》的通告(2023年第13號).
30.	袁冰. 建立適合中醫學的科學的臨床評價體系. 醫學與哲學, 2020, 41(10): 56-63.

1. 孫廣仁. 中醫基礎理論. 北京: 科學出版社, 1994: 10-11.
2. 王瑞平, 肇暉, 李斌. 隨機對照臨床試驗設計要點和規范. 上海醫藥, 2022, 43(7): 72-77.
3. 王梅, 王建華, 張抗, 等. 中醫藥療效評價隊列研究的方法學質量評價. 中醫雜志, 2016, 57(16): 1379-1383.
4. 于亞南, 杜培艷, 劉駿, 等. 精準醫學創新性臨床試驗設計"主方案"研究的概念、設計與案例. 中國新藥雜志, 2020, 29(23): 2712-2717.
5. 郭曉慧, 黃仟, 謝雁鳴, 等. 傘式設計與籃式設計在中醫藥循證評價中的應用. 中國中藥雜志, 2021, 46(8): 2010-2015.
6. 錢真真, 李慶娜, 于亞南, 等. 適應性主方案設計在中醫臨床研究中的應用探索與思考. 中國循證醫學雜志, 2023, 23(4): 471-477.
7. Tamanna RJ, Alam MI, Hossain A, et al. On sample size calculation in testing treatment efficacy in clinical trials. Biom Lett, 2021, 58(2): 133-147.
8. 高珺, 蘇麗文, 袁鷹, 等. 抗腫瘤藥物主方案試驗設計進展及統計考量. 中國衛生統計, 2021, 38(6): 950-955.
9. Lu CC, Li XN, Broglio K, et al. Practical considerations and recommendations for master protocol framework: basket, umbrella and platform trials. Ther Innov Regul Sci, 2021, 55(6): 1145-1154.
10. 國家藥品監督管理局藥品審評中心. 關于《藥物臨床試驗樣本量估計指導原則(征求意見稿)》公開征求意見的通知.
11. Yee LM, McShane LM, Freidlin B, et al. Biostatistical and logistical considerations in the development of basket and umbrella clinical trials. Cancer J, 2019, 25(4): 254-263.
12. Lee JJ, Chu CT. Bayesian clinical trials in action. Stat Med, 2012, 31(25): 2955-2972.
13. 國家藥品監督管理局藥品審評中心. 關于發布《藥物臨床試驗適應性設計指導原則(試行)》的通告(2021年第6號).
14. Simon R. Optimal two-stage designs for phase II clinical trials. Control Clin Trials, 1989, 10(1): 1-10.
15. Simon R, Geyer S, Subramanian J, et al. The Bayesian basket design for genomic variant-driven phase II trials. Semin Oncol, 2016, 43(1): 13-18.
16. Berry SM, Broglio KR, Groshen S, et al. Bayesian hierarchical modeling of patient subpopulations: efficient designs of phase II oncology clinical trials. Clin Trials, 2013, 10(5): 720-734.
17. Chu Y, Yuan Y. A Bayesian basket trial design using a calibrated Bayesian hierarchical model. Clin Trials, 2018, 15(2): 149-158.
18. Zheng H, Grayling MJ, Mozgunov P, et al. Bayesian sample size determination in basket trials borrowing information between subsets. Biostatistics, 2023, 24(4): 1000-1016.
19. Ouma LO, Wason JMS, Zheng H, et al. Design and analysis of umbrella trials: where do we stand. Front Med (Lausanne), 2022, 9: 1037439.
20. Kang D, Coffey C, Smith B, et al. Hierarchical Bayesian clustering design of multiple biomarker subgroups (HCOMBS). Stat Med, 2021, 40(12): 2893-2921.
21. Simon R. Critical review of umbrella, basket, and platform designs for oncology clinical trials. Clin Pharmacol Ther, 2017, 102(6): 934-941.
22. Hobbs BP, Chen N, Lee JJ. Controlled multi-arm platform design using predictive probability. Stat Methods Med Res, 2018, 27(1): 65-78.
23. 何浩強, 惠小珊, 周思遠, 等. 基于CiteSpace軟件的證候類中藥新藥研究可視化分析. 世界中西醫結合雜志, 2022, 17(8): 1497-1502,1557.
24. 國家藥品監督管理局. 關于發布證候類中藥新藥臨床研究技術指導原則的通告(2018年第109號).
25. 趙晨, 張曉雨, 胡嘉元, 等. 中醫同證候系疾病臨床研究母方案設計方法. 中醫雜志, 2018, 59(2): 111-115.
26. 馬蓮, 王可彬, 吳圣賢. 基于“理論創新”視角探討證候類中藥新藥的研發. 中國中藥雜志, 2020, 45(20): 5048-5056.
27. 李燦東, 甘慧娟, 魯玉輝, 等. 基于證素辨證原理的健康狀態辨識研究. 中華中醫藥雜志, 2011, 26(4): 754-757.
28. 賴新梅, 楊雪梅, 陳梅妹, 等. 基于證素辨識原理的證型名稱及內涵規范實踐與思考. 中華中醫藥雜志, 2017, 32(6): 2470-2472.
29. 國家藥品監督管理局藥品審評中心. 關于發布《單臂臨床試驗用于支持抗腫瘤藥上市申請的適用性技術指導原則》的通告(2023年第13號).
30. 袁冰. 建立適合中醫學的科學的臨床評價體系. 醫學與哲學, 2020, 41(10): 56-63.

Chinese Journal of Evidence-Based Medicine

Latest ArticlesSample size estimation methods for master protocol design and insights into their application in clinical research on syndrome-based traditional Chinese medicine

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