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

1.	Yuan B. Towards a clinical efficacy evaluation system adapted for personalized medicine. Pharmgenomics Pers Med, 2021, 14: 487-496.
2.	吳家睿. 精確醫學5年歷程的回顧與思考(上). 醫學與哲學, 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.	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.
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.
17.	胡嘉元, 張曉雨, 趙晨, 等. 母方案設計用于冠心病中醫藥防治方案循證優化的思路和實施方法. 中國循證醫學雜志, 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, 孫楊, 張曉雨, 等. 以證統病—中醫臨床思維的回歸與創新. 世界中醫藥, 2019, 14(10): 2552-2556, 2562.
20.	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.
21.	李建生. 基于病證結合模式重視以證統病的診療形式. 中醫雜志, 2011, 52(7): 558-561.
22.	黃曉珊. 抗敏鎮咳顆粒治療風咳證異病同治的臨床研究. 南京: 南京中醫藥大學, 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.	楊娜, 趙京紅. 基于中醫理論指導下異病同治(痰瘀互阻證)的臨床研究. 中國實用醫藥, 2014, (26): 245-245, 246.
26.	蔣雪峰, 徐雯, 周臘梅. 益腎蠲痹丸治療腰痛的臨床效果觀察. 黑龍江醫藥, 2019, 32(6): 1369-1370.
27.	黎鳳儀, 葉婷慧. 止渴降糖方對糖尿病腎病和哮喘的異病同治的臨床療效. 大眾科技, 2021, 23(9): 47-50.
28.	關之玥, 劉巖, 張晶晶, 等. 母方案平臺試驗設計方法及其在相關領域中的應用. 中國循證醫學雜志, 2021, 21(8): 986-992.
29.	陳凱先. 精準醫學和中醫藥創新發展. 世界科學技術-中醫藥現代化, 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. 吳家睿. 精確醫學5年歷程的回顧與思考(上). 醫學與哲學, 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. 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.
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.
17. 胡嘉元, 張曉雨, 趙晨, 等. 母方案設計用于冠心病中醫藥防治方案循證優化的思路和實施方法. 中國循證醫學雜志, 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, 孫楊, 張曉雨, 等. 以證統病—中醫臨床思維的回歸與創新. 世界中醫藥, 2019, 14(10): 2552-2556, 2562.
20. 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.
21. 李建生. 基于病證結合模式重視以證統病的診療形式. 中醫雜志, 2011, 52(7): 558-561.
22. 黃曉珊. 抗敏鎮咳顆粒治療風咳證異病同治的臨床研究. 南京: 南京中醫藥大學, 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. 楊娜, 趙京紅. 基于中醫理論指導下異病同治(痰瘀互阻證)的臨床研究. 中國實用醫藥, 2014, (26): 245-245, 246.
26. 蔣雪峰, 徐雯, 周臘梅. 益腎蠲痹丸治療腰痛的臨床效果觀察. 黑龍江醫藥, 2019, 32(6): 1369-1370.
27. 黎鳳儀, 葉婷慧. 止渴降糖方對糖尿病腎病和哮喘的異病同治的臨床療效. 大眾科技, 2021, 23(9): 47-50.
28. 關之玥, 劉巖, 張晶晶, 等. 母方案平臺試驗設計方法及其在相關領域中的應用. 中國循證醫學雜志, 2021, 21(8): 986-992.
29. 陳凱先. 精準醫學和中醫藥創新發展. 世界科學技術-中醫藥現代化, 2017, 19(1): 7-18.

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