Application exploration and thinking of master protocol with adaptive design in traditional Chinese medicine clinical research_Chinese Journal of Evidence-Based Medicine

Authors：

QIAN Zhenzhen ^1,2 , LI Qingna ³ , YU Yanan ² , WU Hongli ² ,  JING Zhiwei ² ,  GAO Rui ³

1. Postdoctoral Research Station, China Academy of Chinese Medical Sciences, Beijing 100700, P. R. China;
2. Institute of Basic Clinical Medicine of Chinese Medicine, China Academy of Chinese Medical Sciences, Beijing 100700, P. R. China;
3. Institute of Clinical Pharmacology, China Academy of Chinese Medical Sciences, Beijing 100091, P. R. China;

Corresponding author：

JING Zhiwei, Email: drjzw@163.com; GAO Rui, Email: ruigao@126.com

Keywords：

Adaptive design; Master protocol; Master protocol with adaptive design; Traditional Chinese medicine clinical research

DOI：

10.7507/1672-2531.202210084

Video：

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Master protocol with adaptive design is a new complex innovative trial design that combines an adaptive treatment strategy and master protocol. It is more flexible and adjustable. In the complex clinical trial environment, the dynamics emphasized in this design are consistent with the idea of traditional Chinese medicine (TCM) syndrome differentiation and treatment. In this study, we summarized its concept, characteristics and advantages, and we also discussed its application in TCM clinical research. We hope this paper can provide more thinking and suggestions for TCM clinical trials.

Citation： QIAN Zhenzhen, LI Qingna, YU Yanan, WU Hongli, JING Zhiwei, GAO Rui. Application exploration and thinking of master protocol with adaptive design in traditional Chinese medicine clinical research. Chinese Journal of Evidence-Based Medicine, 2023, 23(4): 471-477. doi: 10.7507/1672-2531.202210084 Copy

1.	孙昱. 有效应对流行病的新型临床试验设计. 药物评价研究, 2020, 43(6): 977-986.
2.	Woodcock J, LaVange LM. Master protocols to study multiple therapies, multiple diseases, or both. N Engl J Med, 2017, 377(1): 62-70.
3.	U. S. FDA. Master protocols: efficient clinical trial design strategies to expedite development of oncology drugs and biologics.
4.	U. S. FDA. Adaptive design clinical trials for drugs and biologics.
5.	Heinrich MC, Joensuu H, Demetri GD, et al. Phase II, open-label study evaluating the activity of imatinib in treating life-threatening malignancies known to be associated with imatinib-sensitive tyrosine kinases. Clin Cancer Res, 2008, 14(9): 2717-2725.
6.	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.
7.	Bethesda. NCI-MATCH Trial (Molecular Analysis forTherapy Choice).
8.	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.
9.	Brahmachari B, Bhatt A. Adaptive design - an innovative tool in drug development. Indian J Med Res, 2011, 133(3): 243-245.
10.	FDA. Guidance for industry adaptive design clinica drugs and biologics (draft guidance), 2018.
11.	房凌旭, 杨丽娜, 江川, 等. 适应性治疗策略的研究进展. 医学理论与实践, 2022, 35(22): 3812-3813, 3821.
12.	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.
13.	Cunanan KM, Iasonos A, Shen R, et al. An efficient basket trial design. Stat Med, 2017, 36(10): 1568-1579.
14.	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.
15.	Chen C, Li X, Yuan S, et al. Statistical design and considerations of a phase 3 basket trial for simultaneous investigation of multiple tumor types in one study. Stat Biopharm Res, 2016, 8(3): 248-257.
16.	Yuan SS, Chen A, He L, et al. On group sequential enrichment design for basket trial. Stat Biopharm Res, 2016, 8(3): 293-306.
17.	Barker AD, Sigman CC, Kelloff GJ, et al. I-SPY 2: an adaptive breast cancer trial design in the setting of neoadjuvant chemotherapy. Clin Pharmacol Ther, 2009, 86(1): 97-100.
18.	Esserman LJ, Woodcock J. Accelerating identification and regulatory approval of investigational cancer drugs. JAMA, 2011, 306(23): 2608-2609.
19.	Berry SM, Connor JT, Lewis RJ. The platform trial: an efficient strategy for evaluating multiple treatments. JAMA, 2015, 313(16): 1619-1620.
20.	国家药品监督管理局药品审评中心. 药物临床试验适应性设计指导原则(试行)(2021年第6号).
21.	国家心血管病中心中国医学科学院阜外医院药物临床试验设计与实施中疑难问题处理的专家共识组, 中华医学会心血管病学分会心血管病临床研究学组. 药物临床试验设计与实施中疑难问题处理的专家共识. 中国循证医学杂志, 2022, 22(3): 249-260.
22.	胡海霞, 王陵, 李婵娟, 等. 篮子试验在抗肿瘤靶向药物研发中的应用现状. 中国卫生统计, 2018, 35(3): 477-480.
23.	Redig AJ, Jänne PA. Basket trials and the evolution of clinical trial design in an era of genomic medicine. J Clin Oncol, 2015, 33(9): 975-977.
24.	尹环, 王维真, 张忠占. 考虑疗效和安全性及其相关性的最优两阶段设计. 北京工业大学学报, 2019, 45(12): 1269-1276.
25.	Simon R. Optimal two-stage designs for phase II clinical trials. Control Clin Trials, 1989, 10(1): 1-10.
26.	Krajewska M, Rauch G. A new basket trial design based on clustering of homogeneous subpopulations. J Biopharm Stat, 2021, 31(4): 425-447.
27.	Herbst RS, Gandara DR, Hirsch FR, et al. Lung Master Protocol (Lung-MAP)-a biomarker-driven protocol for accelerating development of therapies for squamous cell lung cancer: SWOG S1400. Clin Cancer Res, 2015, 21(7): 1514-1524.
28.	Kim ES, Herbst RS, Wistuba II, et al. The BATTLE trial: personalizing therapy for lung cancer. Cancer Discov, 2011, 1(1): 44-53.
29.	Ledford H. 'Master protocol' aims to revamp cancer trials. Nature, 2013, 498(7453): 146-147.
30.	Meyer EL, Mesenbrink P, Dunger-Baldauf C, et al. The evolution of master protocol clinical trial designs: a systematic literature review. Clin Ther, 2020, 42(7): 1330-1360.
31.	Park JW, Liu MC, Yee D, et al. Adaptive randomization of neratinib in early breast cancer. N Engl J Med, 2016, 375(1): 11-22.
32.	Rugo HS, Olopade OI, DeMichele A, et al. Adaptive randomization of veliparib-carboplatin treatment in breast cancer. N Engl J Med, 2016, 375(1): 23-34.
33.	Adaptive Platform Trials Coalition. Adaptive platform trials: definition, design, conduct and reporting considerations. Nat Rev Drug Discov, 2019, 18(10): 797-807.
34.	王永炎, 于亚南, 荆志伟, 等. 适应性治疗临床试验设计与辨证论治疗效评价的思考. 中国中医基础医学杂志, 2012, 18(3): 316-318.
35.	姜辉, 吕肖芳, 张丽, 等. 代谢组学在中医药现代化研究中的应用. 中华中医药杂志, 2013, 28(3): 588-590.
36.	元唯安, 唐健元, 高蕊, 等. 中药新药临床试验质量控制关键问题的专家共识. 中国中药杂志, 2021, 46(7): 1701-1705.
37.	洪净. 中医辨证的思维模式和方法研究. 中医杂志, 2003, 44(1): 8-10.
38.	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.
39.	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.
40.	钱真真, 周莎, 于亚南, 等. 适应性随机化及其在临床试验中的应用操作要点. 中国循证医学杂志, 2022, 22(8): 971-977.

1. 孙昱. 有效应对流行病的新型临床试验设计. 药物评价研究, 2020, 43(6): 977-986.
2. Woodcock J, LaVange LM. Master protocols to study multiple therapies, multiple diseases, or both. N Engl J Med, 2017, 377(1): 62-70.
3. U. S. FDA. Master protocols: efficient clinical trial design strategies to expedite development of oncology drugs and biologics.
4. U. S. FDA. Adaptive design clinical trials for drugs and biologics.
5. Heinrich MC, Joensuu H, Demetri GD, et al. Phase II, open-label study evaluating the activity of imatinib in treating life-threatening malignancies known to be associated with imatinib-sensitive tyrosine kinases. Clin Cancer Res, 2008, 14(9): 2717-2725.
6. 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.
7. Bethesda. NCI-MATCH Trial (Molecular Analysis forTherapy Choice).
8. 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.
9. Brahmachari B, Bhatt A. Adaptive design - an innovative tool in drug development. Indian J Med Res, 2011, 133(3): 243-245.
10. FDA. Guidance for industry adaptive design clinica drugs and biologics (draft guidance), 2018.
11. 房凌旭, 杨丽娜, 江川, 等. 适应性治疗策略的研究进展. 医学理论与实践, 2022, 35(22): 3812-3813, 3821.
12. 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.
13. Cunanan KM, Iasonos A, Shen R, et al. An efficient basket trial design. Stat Med, 2017, 36(10): 1568-1579.
14. 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.
15. Chen C, Li X, Yuan S, et al. Statistical design and considerations of a phase 3 basket trial for simultaneous investigation of multiple tumor types in one study. Stat Biopharm Res, 2016, 8(3): 248-257.
16. Yuan SS, Chen A, He L, et al. On group sequential enrichment design for basket trial. Stat Biopharm Res, 2016, 8(3): 293-306.
17. Barker AD, Sigman CC, Kelloff GJ, et al. I-SPY 2: an adaptive breast cancer trial design in the setting of neoadjuvant chemotherapy. Clin Pharmacol Ther, 2009, 86(1): 97-100.
18. Esserman LJ, Woodcock J. Accelerating identification and regulatory approval of investigational cancer drugs. JAMA, 2011, 306(23): 2608-2609.
19. Berry SM, Connor JT, Lewis RJ. The platform trial: an efficient strategy for evaluating multiple treatments. JAMA, 2015, 313(16): 1619-1620.
20. 国家药品监督管理局药品审评中心. 药物临床试验适应性设计指导原则(试行)(2021年第6号).
21. 国家心血管病中心中国医学科学院阜外医院药物临床试验设计与实施中疑难问题处理的专家共识组, 中华医学会心血管病学分会心血管病临床研究学组. 药物临床试验设计与实施中疑难问题处理的专家共识. 中国循证医学杂志, 2022, 22(3): 249-260.
22. 胡海霞, 王陵, 李婵娟, 等. 篮子试验在抗肿瘤靶向药物研发中的应用现状. 中国卫生统计, 2018, 35(3): 477-480.
23. Redig AJ, Jänne PA. Basket trials and the evolution of clinical trial design in an era of genomic medicine. J Clin Oncol, 2015, 33(9): 975-977.
24. 尹环, 王维真, 张忠占. 考虑疗效和安全性及其相关性的最优两阶段设计. 北京工业大学学报, 2019, 45(12): 1269-1276.
25. Simon R. Optimal two-stage designs for phase II clinical trials. Control Clin Trials, 1989, 10(1): 1-10.
26. Krajewska M, Rauch G. A new basket trial design based on clustering of homogeneous subpopulations. J Biopharm Stat, 2021, 31(4): 425-447.
27. Herbst RS, Gandara DR, Hirsch FR, et al. Lung Master Protocol (Lung-MAP)-a biomarker-driven protocol for accelerating development of therapies for squamous cell lung cancer: SWOG S1400. Clin Cancer Res, 2015, 21(7): 1514-1524.
28. Kim ES, Herbst RS, Wistuba II, et al. The BATTLE trial: personalizing therapy for lung cancer. Cancer Discov, 2011, 1(1): 44-53.
29. Ledford H. 'Master protocol' aims to revamp cancer trials. Nature, 2013, 498(7453): 146-147.
30. Meyer EL, Mesenbrink P, Dunger-Baldauf C, et al. The evolution of master protocol clinical trial designs: a systematic literature review. Clin Ther, 2020, 42(7): 1330-1360.
31. Park JW, Liu MC, Yee D, et al. Adaptive randomization of neratinib in early breast cancer. N Engl J Med, 2016, 375(1): 11-22.
32. Rugo HS, Olopade OI, DeMichele A, et al. Adaptive randomization of veliparib-carboplatin treatment in breast cancer. N Engl J Med, 2016, 375(1): 23-34.
33. Adaptive Platform Trials Coalition. Adaptive platform trials: definition, design, conduct and reporting considerations. Nat Rev Drug Discov, 2019, 18(10): 797-807.
34. 王永炎, 于亚南, 荆志伟, 等. 适应性治疗临床试验设计与辨证论治疗效评价的思考. 中国中医基础医学杂志, 2012, 18(3): 316-318.
35. 姜辉, 吕肖芳, 张丽, 等. 代谢组学在中医药现代化研究中的应用. 中华中医药杂志, 2013, 28(3): 588-590.
36. 元唯安, 唐健元, 高蕊, 等. 中药新药临床试验质量控制关键问题的专家共识. 中国中药杂志, 2021, 46(7): 1701-1705.
37. 洪净. 中医辨证的思维模式和方法研究. 中医杂志, 2003, 44(1): 8-10.
38. 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.
39. 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.
40. 钱真真, 周莎, 于亚南, 等. 适应性随机化及其在临床试验中的应用操作要点. 中国循证医学杂志, 2022, 22(8): 971-977.

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