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.

1.	孙广仁. 中医基础理论. 北京: 科学出版社, 1994: 10-11.
2.	王瑞平, 肇晖, 李斌. 随机对照临床试验设计要点和规范. 上海医药, 2022, 43(7): 72-77.
3.	王梅, 王建华, 张抗, 等. 中医药疗效评价队列研究的方法学质量评价. 中医杂志, 2016, 57(16): 1379-1383.
4.	于亚南, 杜培艳, 刘骏, 等. 精准医学创新性临床试验设计"主方案"研究的概念、设计与案例. 中国新药杂志, 2020, 29(23): 2712-2717.
5.	郭晓慧, 黄仟, 谢雁鸣, 等. 伞式设计与篮式设计在中医药循证评价中的应用. 中国中药杂志, 2021, 46(8): 2010-2015.
6.	钱真真, 李庆娜, 于亚南, 等. 适应性主方案设计在中医临床研究中的应用探索与思考. 中国循证医学杂志, 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, 苏丽文, 袁鹰, 等. 抗肿瘤药物主方案试验设计进展及统计考量. 中国卫生统计, 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.	国家药品监督管理局药品审评中心. 关于《药物临床试验样本量估计指导原则(征求意见稿)》公开征求意见的通知.
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.	国家药品监督管理局药品审评中心. 关于发布《药物临床试验适应性设计指导原则(试行)》的通告(2021年第6号).
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.	何浩强, 惠小珊, 周思远, 等. 基于CiteSpace软件的证候类中药新药研究可视化分析. 世界中西医结合杂志, 2022, 17(8): 1497-1502,1557.
24.	国家药品监督管理局. 关于发布证候类中药新药临床研究技术指导原则的通告(2018年第109号).
25.	赵晨, 张晓雨, 胡嘉元, 等. 中医同证候系疾病临床研究母方案设计方法. 中医杂志, 2018, 59(2): 111-115.
26.	马莲, 王可彬, 吴圣贤. 基于“理论创新”视角探讨证候类中药新药的研发. 中国中药杂志, 2020, 45(20): 5048-5056.
27.	李灿东, 甘慧娟, 鲁玉辉, 等. 基于证素辨证原理的健康状态辨识研究. 中华中医药杂志, 2011, 26(4): 754-757.
28.	赖新梅, 杨雪梅, 陈梅妹, 等. 基于证素辨识原理的证型名称及内涵规范实践与思考. 中华中医药杂志, 2017, 32(6): 2470-2472.
29.	国家药品监督管理局药品审评中心. 关于发布《单臂临床试验用于支持抗肿瘤药上市申请的适用性技术指导原则》的通告(2023年第13号).
30.	袁冰. 建立适合中医学的科学的临床评价体系. 医学与哲学, 2020, 41(10): 56-63.

1. 孙广仁. 中医基础理论. 北京: 科学出版社, 1994: 10-11.
2. 王瑞平, 肇晖, 李斌. 随机对照临床试验设计要点和规范. 上海医药, 2022, 43(7): 72-77.
3. 王梅, 王建华, 张抗, 等. 中医药疗效评价队列研究的方法学质量评价. 中医杂志, 2016, 57(16): 1379-1383.
4. 于亚南, 杜培艳, 刘骏, 等. 精准医学创新性临床试验设计"主方案"研究的概念、设计与案例. 中国新药杂志, 2020, 29(23): 2712-2717.
5. 郭晓慧, 黄仟, 谢雁鸣, 等. 伞式设计与篮式设计在中医药循证评价中的应用. 中国中药杂志, 2021, 46(8): 2010-2015.
6. 钱真真, 李庆娜, 于亚南, 等. 适应性主方案设计在中医临床研究中的应用探索与思考. 中国循证医学杂志, 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, 苏丽文, 袁鹰, 等. 抗肿瘤药物主方案试验设计进展及统计考量. 中国卫生统计, 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. 国家药品监督管理局药品审评中心. 关于《药物临床试验样本量估计指导原则(征求意见稿)》公开征求意见的通知.
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. 国家药品监督管理局药品审评中心. 关于发布《药物临床试验适应性设计指导原则(试行)》的通告(2021年第6号).
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. 何浩强, 惠小珊, 周思远, 等. 基于CiteSpace软件的证候类中药新药研究可视化分析. 世界中西医结合杂志, 2022, 17(8): 1497-1502,1557.
24. 国家药品监督管理局. 关于发布证候类中药新药临床研究技术指导原则的通告(2018年第109号).
25. 赵晨, 张晓雨, 胡嘉元, 等. 中医同证候系疾病临床研究母方案设计方法. 中医杂志, 2018, 59(2): 111-115.
26. 马莲, 王可彬, 吴圣贤. 基于“理论创新”视角探讨证候类中药新药的研发. 中国中药杂志, 2020, 45(20): 5048-5056.
27. 李灿东, 甘慧娟, 鲁玉辉, 等. 基于证素辨证原理的健康状态辨识研究. 中华中医药杂志, 2011, 26(4): 754-757.
28. 赖新梅, 杨雪梅, 陈梅妹, 等. 基于证素辨识原理的证型名称及内涵规范实践与思考. 中华中医药杂志, 2017, 32(6): 2470-2472.
29. 国家药品监督管理局药品审评中心. 关于发布《单臂临床试验用于支持抗肿瘤药上市申请的适用性技术指导原则》的通告(2023年第13号).
30. 袁冰. 建立适合中医学的科学的临床评价体系. 医学与哲学, 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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