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：

Export PDF Favorites Scan Get Citation

Abstract Full text Figures/Tables Video References Cited by

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.	孫昱. 有效應對流行病的新型臨床試驗設計. 藥物評價研究, 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.
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, 楊麗娜, 江川, 等. 適應性治療策略的研究進展. 醫學理論與實踐, 2022, 35(22): 3812-3813, 3821.
12.	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.
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.	國家藥品監督管理局藥品審評中心. 藥物臨床試驗適應性設計指導原則(試行)(2021年第6號).
21.	國家心血管病中心中國醫學科學院阜外醫院藥物臨床試驗設計與實施中疑難問題處理的專家共識組, 中華醫學會心血管病學分會心血管病臨床研究學組. 藥物臨床試驗設計與實施中疑難問題處理的專家共識. 中國循證醫學雜志, 2022, 22(3): 249-260.
22.	胡海霞, 王陵, 李嬋娟, 等. 籃子試驗在抗腫瘤靶向藥物研發中的應用現狀. 中國衛生統計, 2018, 35(3): 477-480.
23.	Redig AJ, J?nne 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.	尹環, 王維真, 張忠占. 考慮療效和安全性及其相關性的最優兩階段設計. 北京工業大學學報, 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, 于亞南, 荊志偉, 等. 適應性治療臨床試驗設計與辨證論治療效評價的思考. 中國中醫基礎醫學雜志, 2012, 18(3): 316-318.
35.	姜輝, 呂肖芳, 張麗, 等. 代謝組學在中醫藥現代化研究中的應用. 中華中醫藥雜志, 2013, 28(3): 588-590.
36.	元唯安, 唐健元, 高蕊, 等. 中藥新藥臨床試驗質量控制關鍵問題的專家共識. 中國中藥雜志, 2021, 46(7): 1701-1705.
37.	洪凈. 中醫辨證的思維模式和方法研究. 中醫雜志, 2003, 44(1): 8-10.
38.	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.
39.	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.
40.	錢真真, 周莎, 于亞南, 等. 適應性隨機化及其在臨床試驗中的應用操作要點. 中國循證醫學雜志, 2022, 22(8): 971-977.

1. 孫昱. 有效應對流行病的新型臨床試驗設計. 藥物評價研究, 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.
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, 楊麗娜, 江川, 等. 適應性治療策略的研究進展. 醫學理論與實踐, 2022, 35(22): 3812-3813, 3821.
12. 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.
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. 國家藥品監督管理局藥品審評中心. 藥物臨床試驗適應性設計指導原則(試行)(2021年第6號).
21. 國家心血管病中心中國醫學科學院阜外醫院藥物臨床試驗設計與實施中疑難問題處理的專家共識組, 中華醫學會心血管病學分會心血管病臨床研究學組. 藥物臨床試驗設計與實施中疑難問題處理的專家共識. 中國循證醫學雜志, 2022, 22(3): 249-260.
22. 胡海霞, 王陵, 李嬋娟, 等. 籃子試驗在抗腫瘤靶向藥物研發中的應用現狀. 中國衛生統計, 2018, 35(3): 477-480.
23. Redig AJ, J?nne 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. 尹環, 王維真, 張忠占. 考慮療效和安全性及其相關性的最優兩階段設計. 北京工業大學學報, 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, 于亞南, 荊志偉, 等. 適應性治療臨床試驗設計與辨證論治療效評價的思考. 中國中醫基礎醫學雜志, 2012, 18(3): 316-318.
35. 姜輝, 呂肖芳, 張麗, 等. 代謝組學在中醫藥現代化研究中的應用. 中華中醫藥雜志, 2013, 28(3): 588-590.
36. 元唯安, 唐健元, 高蕊, 等. 中藥新藥臨床試驗質量控制關鍵問題的專家共識. 中國中藥雜志, 2021, 46(7): 1701-1705.
37. 洪凈. 中醫辨證的思維模式和方法研究. 中醫雜志, 2003, 44(1): 8-10.
38. 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.
39. 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.
40. 錢真真, 周莎, 于亞南, 等. 適應性隨機化及其在臨床試驗中的應用操作要點. 中國循證醫學雜志, 2022, 22(8): 971-977.

Previous Article
Application of research methods of master protocol in precision medicine
Next Article
Features and progress of metadata standards of clinical research

Chinese Journal of Evidence-Based Medicine

Application exploration and thinking of master protocol with adaptive design in traditional Chinese medicine clinical research

Abstract Full text Figures/Tables Video References Cited by

Previous Article

Next Article

Format

Content