Design of master protocol basket trial in precision medicine and its application in traditional Chinese medicine_Chinese Journal of Evidence-Based Medicine

Authors：

CHEN Ying ^1,2 , CHANG Jingling ³ , DAI Hengheng ^1,2 , ZHANG Kaige ¹ , ZHANG Jingjing ^1,2 , ZHANG Xuecheng ^1,2 , WAN Siqi ^1,2 , GUAN Zhiyue ^1,2 , HU Mingzhi ^1,2 , LIU Yan ¹ ,  SHANG Hongcai ¹

1. Key Laboratory of Chinese Internal Medicine of Ministry of Education, Dongzhimen Hospital, Beijing University of Chinese Medicine, Beijing 100700, P. R. China;
2. Beijing University of Chinese Medicine, Beijing 100029, P. R. China;
3. Department of Encephalopathy, Dongzhimen Hospital, Beijing University of Chinese Medicine, Beijing 100700, P. R. China;

Corresponding author：

SHANG Hongcai, Email: shanghongcai@126.com

Keywords：

Precision medicine; Master protocol; Basket trial; Same treatment for different diseases; Syndrome dominating disease; Clinical research of traditional Chinese medicine; Efficacy evaluation method

DOI：

10.7507/1672-2531.202201063

Video：

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Precision medicine is a personalized medical system based on patients' individual biological information, clinical symptoms and signs, forming a new clinical research model and medical practice path. The basic idea of traditional Chinese medicine and the concept of precision medicine share many similarities. The basket trial developed for precision medicine is also suitable for clinical trials and evaluation of the efficacy of traditional Chinese medicine syndrome differentiation and treatment systems. Basket trials are used to evaluate the efficacy of a drug in the treatment of multiple diseases or disease subtypes. It has the advantages of sharing a master protocol, unifying management of subsidiary studies, simplifying the test implementation process, unifying statistical analysis, saving resources, reducing budgets and accelerating the drug evaluation progress. This is similar to the concept of using the "same treatment for different diseases" found in traditional Chinese medicine. This paper introduced the concept and method of basket trials and explored their application and advantages in clinical research into traditional Chinese medicine. This study is expected to provide references for the methodological innovation of clinical research into traditional Chinese medicine.

Citation： CHEN Ying, CHANG Jingling, DAI Hengheng, ZHANG Kaige, ZHANG Jingjing, ZHANG Xuecheng, WAN Siqi, GUAN Zhiyue, HU Mingzhi, LIU Yan, SHANG Hongcai. Design of master protocol basket trial in precision medicine and its application in traditional Chinese medicine. Chinese Journal of Evidence-Based Medicine, 2022, 22(4): 483-489. doi: 10.7507/1672-2531.202201063 Copy

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16.	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.
17.	胡嘉元, 张晓雨, 赵晨, 等. 母方案设计用于冠心病中医药防治方案循证优化的思路和实施方法. 中国循证医学杂志, 2019, 19(1): 102-106.
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21.	李建生. 基于病证结合模式重视以证统病的诊疗形式. 中医杂志, 2011, 52(7): 558-561.
22.	黄晓珊. 抗敏镇咳颗粒治疗风咳证异病同治的临床研究. 南京: 南京中医药大学, 2020.
23.	Liu W, Zhou L, Feng L, et al. BuqiTongluo granule for ischemic stroke, stable angina pectoris, diabetic peripheral neuropathy with Qi deficiency and blood stasis syndrome: rationale and novel basket design. Front Pharmacol, 2021, 12: 764669.
24.	Sherman RE, Anderson SA, Dal Pan GJ, et al. Real-world evidence-what is it and what can it tell us. N Engl J Med, 2016, 375(23): 2293-2297.
25.	杨娜, 赵京红. 基于中医理论指导下异病同治(痰瘀互阻证)的临床研究. 中国实用医药, 2014, (26): 245-245, 246.
26.	蒋雪峰, 徐雯, 周腊梅. 益肾蠲痹丸治疗腰痛的临床效果观察. 黑龙江医药, 2019, 32(6): 1369-1370.
27.	黎凤仪, 叶婷慧. 止渴降糖方对糖尿病肾病和哮喘的异病同治的临床疗效. 大众科技, 2021, 23(9): 47-50.
28.	关之玥, 刘岩, 张晶晶, 等. 母方案平台试验设计方法及其在相关领域中的应用. 中国循证医学杂志, 2021, 21(8): 986-992.
29.	陈凯先. 精准医学和中医药创新发展. 世界科学技术-中医药现代化, 2017, 19(1): 7-18.

1. Yuan B. Towards a clinical efficacy evaluation system adapted for personalized medicine. Pharmgenomics Pers Med, 2021, 14: 487-496.
2. 吴家睿. 精确医学5年历程的回顾与思考(上). 医学与哲学, 2021, 42(20): 1-4,10.
3. Food and Drug Administration. Master protocols: efficient clinical trial design strategies to expedite development of oncology drugs and biologics. Available at: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/master-protocols-efficient-clinical-trial-design-strategies-expedite-development-oncology-drugs-and.
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. 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.
6. Woodcock J, LaVange LM. Master protocols to study multiple therapies, multiple diseases, or both. N Engl J Med, 2017, 377(1): 62-70.
7. Ornes S. Core concept: basket trial approach capitalizes on the molecular mechanisms of tumors. Proc Natl Acad Sci USA, 2016, 113(26): 7007-7008.
8. Park JJH, Hsu G, Siden EG, et al. An overview of precision oncology basket and umbrella trials for clinicians. CA Cancer J Clin, 2020, 70(2): 125-137.
9. Hyman DM, Puzanov I, Subbiah V, et al. Vemurafenib in multiple nonmelanoma cancers with BRAF V600 mutations. N Engl J Med, 2015, 373(8): 726-736.
10. Volovat SR, Ciuleanu TE, Koralewski P, et al. A multicenter, single-arm, basket design, phase II study of NC-6004 plus gemcitabine in patients with advanced unresectable lung, biliary tract, or bladder cancer. Oncotarget, 2020, 11(33): 3105-3117.
11. Hyman DM, Piha-Paul SA, Won H, et al. HER kinase inhibition in patients with HER2- and HER3-mutant cancers. Nature, 2018, 554(7691): 189-194.
12. 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.
13. 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.
14. Chu Y, Yuan Y. A Bayesian basket trial design using a calibrated Bayesian hierarchical model. Clin Trials, 2018, 15(2): 149-158.
15. Hobbs BP, Landin R. Bayesian basket trial design with exchangeability monitoring. Stat Med, 2018, 37(25): 3557-3572.
16. 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.
17. 胡嘉元, 张晓雨, 赵晨, 等. 母方案设计用于冠心病中医药防治方案循证优化的思路和实施方法. 中国循证医学杂志, 2019, 19(1): 102-106.
18. Hyman DM, Solit DB, Arcila ME, et al. Precision medicine at Memorial Sloan Kettering Cancer Center: clinical next-generation sequencing enabling next-generation targeted therapy trials. Drug Discov Today, 2015, 20(12): 1422-1428.
19. 李涵, 孙杨, 张晓雨, 等. 以证统病—中医临床思维的回归与创新. 世界中医药, 2019, 14(10): 2552-2556, 2562.
20. 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.
21. 李建生. 基于病证结合模式重视以证统病的诊疗形式. 中医杂志, 2011, 52(7): 558-561.
22. 黄晓珊. 抗敏镇咳颗粒治疗风咳证异病同治的临床研究. 南京: 南京中医药大学, 2020.
23. Liu W, Zhou L, Feng L, et al. BuqiTongluo granule for ischemic stroke, stable angina pectoris, diabetic peripheral neuropathy with Qi deficiency and blood stasis syndrome: rationale and novel basket design. Front Pharmacol, 2021, 12: 764669.
24. Sherman RE, Anderson SA, Dal Pan GJ, et al. Real-world evidence-what is it and what can it tell us. N Engl J Med, 2016, 375(23): 2293-2297.
25. 杨娜, 赵京红. 基于中医理论指导下异病同治(痰瘀互阻证)的临床研究. 中国实用医药, 2014, (26): 245-245, 246.
26. 蒋雪峰, 徐雯, 周腊梅. 益肾蠲痹丸治疗腰痛的临床效果观察. 黑龙江医药, 2019, 32(6): 1369-1370.
27. 黎凤仪, 叶婷慧. 止渴降糖方对糖尿病肾病和哮喘的异病同治的临床疗效. 大众科技, 2021, 23(9): 47-50.
28. 关之玥, 刘岩, 张晶晶, 等. 母方案平台试验设计方法及其在相关领域中的应用. 中国循证医学杂志, 2021, 21(8): 986-992.
29. 陈凯先. 精准医学和中医药创新发展. 世界科学技术-中医药现代化, 2017, 19(1): 7-18.

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