Design of the master protocol platform trial and its application in related fields_Chinese Journal of Evidence-Based Medicine

Authors：

GUAN Zhiyue , LIU Yan , ZHANG Jingjing , CHEN Ying , DAI Hengheng , ZHANG Xuecheng , HU Mingzhi ,  SHANG Hongcai

Dongzhimen Hospital Affiliated to Beijing University of Chinese Medicine, Beijing 100700, P.R.China;

Corresponding?author：

SHANG Hongcai, Email: shanghongcai@126.com

Keywords：

Master protocol; Platform trial; Randomized controlled trial; COVID-19

DOI：

10.7507/1672-2531.202104005

Video：

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The master protocol platform trial is a trial in which multiple treatments or different combinations of treatments are consistently evaluated in the context of a single disease, possibly within several sub-studies for different disease subtypes, allowing multiple treatments to enter or exit the trial platform based on a certain decision algorithm. Compared with the traditional clinical trial design, the master protocol platform trial as a scientific innovative model of clinical trial can accelerate drug evaluation to deal with the contradiction between the awaited evaluation of numerous drugs and lack of effective therapy for diseases. By using the large-scale randomized controlled clinical trial for COVID-19 inpatients (RECOVERY) led by the University of Oxford in the UK in 2020 as an illustration, this paper introduced the concept, design principle, and basic trial process of the master protocol platform trial to provide methodological references for clinical research of drugs.

Citation： GUAN Zhiyue, LIU Yan, ZHANG Jingjing, CHEN Ying, DAI Hengheng, ZHANG Xuecheng, HU Mingzhi, SHANG Hongcai. Design of the master protocol platform trial and its application in related fields. Chinese Journal of Evidence-Based Medicine, 2021, 21(8): 986-992. doi: 10.7507/1672-2531.202104005 Copy

1.	Redman MW, Allegra CJ. The master protocol concept. Semin Oncol, 2015, 42(5): 724-730.
2.	Tang R, Shen J, Yuan Y. ComPAS: a Bayesian drug combination platform trial design with adaptive shrinkage. Stat Med, 2019, 38(7): 1120-1134.
3.	Woodcock J, LaVange LM. Master protocols to study multiple therapies, multiple diseases, or both. N Engl J Med, 2017, 377(1): 62-70.
4.	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.
5.	U. S. 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/media/120721/download.
6.	Hirakawa A, Asano J, Sato H, et al. Master protocol trials in oncology: review and new trial designs. Contemp Clin Trials Commun, 2018, 12: 1-8.
7.	Ledford H. 'Master protocol' aims to revamp cancer trials. Nature, 2013, 498(7453): 146-147.
8.	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.
9.	Angus DC, Berry S, Lewis RJ, et al. The REMAP-CAP (Randomized Embedded Multifactorial Adaptive Platform for Community-acquired Pneumonia) study. Rationale and design. Ann Am Thorac Soc, 2020, 17(7): 879-891.
10.	U. S. Food and Drug Administration. Adaptive designs for clinical trials of drugs and biologics. Available at: https://www.fda.gov/media/78495/download.
11.	于亞南, 杜培艷, 劉駿, 等. 精準醫學創新性臨床試驗設計. 中國新藥雜志, 2020, 29(23): 2712-2717.
12.	Adaptive Platform Trials Coalition. Adaptive platform trials: definition, design, conduct and reporting considerations. Nat Rev Drug Discov, 2019, 18(10): 797-807.
13.	RECOVERY Central Coordinating Office. RECOVERY protocol V13.0. Available at: https://www.recoverytrial.net/files/recovery-protocol-v13-0-2021-01-26.pdf.
14.	衡明莉, 王北琪, 王駿. 對美國FDA適應性設計指導原則的介紹. 中國臨床藥理學雜志, 2019, 35(12): 1316-1320.
15.	Cerqueira FP, Jesus AMC, Cotrim MD. Adaptive design: a review of the technical, statistical, and regulatory aspects of implementation in a clinical trial. Ther Innov Regul Sci, 2020, 54(1): 246-258.
16.	Proschan MA, Hunsberger SA. Designed extension of studies based on conditional power. Biometrics, 1995, 51(4): 1315-1324.
17.	Asendorf T, Henderson R, Schmidli H, et al. Sample size re-estimation for clinical trials with longitudinal negative binomial counts including time trends. Stat Med, 2019, 38(9): 1503-1528.
18.	Denne JS. Sample size recalculation using conditional power. Stat Med, 2001, 20(17-18): 2645-2660.
19.	Park JJH, Siden E, Zoratti MJ, et al. Systematic review of basket trials, umbrella trials, and platform trials: a landscape analysis of master protocols. Trials, 2019, 20(1): 572.
20.	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.
21.	Berry SM, Connor JT, Lewis RJ. The platform trial: an efficient strategy for evaluating multiple treatments. JAMA, 2015, 313(16): 1619-1620.
22.	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.
23.	Steuer CE, Papadimitrakopoulou V, Herbst RS, et al. Innovative clinical trials: the LUNG-MAP Study. Clin Pharmacol Ther, 2015, 97(5): 488-491.
24.	Angus DC, Derde L, Al-Beidh F, et al. Effect of hydrocortisone on mortality and organ support in patients with severe COVID-19: the REMAP-CAP COVID-19 corticosteroid domain randomized clinical trial. JAMA, 2020, 324(13): 1317-1329.
25.	RECOVERY Central Coordinating Office. RECOVERY protocol V1.0. Available at: https://www.recoverytrial.net/files/protocol-archive/recovery-protocol-v1-0-2020-03-18-final.pdf.

1. Redman MW, Allegra CJ. The master protocol concept. Semin Oncol, 2015, 42(5): 724-730.
2. Tang R, Shen J, Yuan Y. ComPAS: a Bayesian drug combination platform trial design with adaptive shrinkage. Stat Med, 2019, 38(7): 1120-1134.
3. Woodcock J, LaVange LM. Master protocols to study multiple therapies, multiple diseases, or both. N Engl J Med, 2017, 377(1): 62-70.
4. 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.
5. U. S. 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/media/120721/download.
6. Hirakawa A, Asano J, Sato H, et al. Master protocol trials in oncology: review and new trial designs. Contemp Clin Trials Commun, 2018, 12: 1-8.
7. Ledford H. 'Master protocol' aims to revamp cancer trials. Nature, 2013, 498(7453): 146-147.
8. 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.
9. Angus DC, Berry S, Lewis RJ, et al. The REMAP-CAP (Randomized Embedded Multifactorial Adaptive Platform for Community-acquired Pneumonia) study. Rationale and design. Ann Am Thorac Soc, 2020, 17(7): 879-891.
10. U. S. Food and Drug Administration. Adaptive designs for clinical trials of drugs and biologics. Available at: https://www.fda.gov/media/78495/download.
11. 于亞南, 杜培艷, 劉駿, 等. 精準醫學創新性臨床試驗設計. 中國新藥雜志, 2020, 29(23): 2712-2717.
12. Adaptive Platform Trials Coalition. Adaptive platform trials: definition, design, conduct and reporting considerations. Nat Rev Drug Discov, 2019, 18(10): 797-807.
13. RECOVERY Central Coordinating Office. RECOVERY protocol V13.0. Available at: https://www.recoverytrial.net/files/recovery-protocol-v13-0-2021-01-26.pdf.
14. 衡明莉, 王北琪, 王駿. 對美國FDA適應性設計指導原則的介紹. 中國臨床藥理學雜志, 2019, 35(12): 1316-1320.
15. Cerqueira FP, Jesus AMC, Cotrim MD. Adaptive design: a review of the technical, statistical, and regulatory aspects of implementation in a clinical trial. Ther Innov Regul Sci, 2020, 54(1): 246-258.
16. Proschan MA, Hunsberger SA. Designed extension of studies based on conditional power. Biometrics, 1995, 51(4): 1315-1324.
17. Asendorf T, Henderson R, Schmidli H, et al. Sample size re-estimation for clinical trials with longitudinal negative binomial counts including time trends. Stat Med, 2019, 38(9): 1503-1528.
18. Denne JS. Sample size recalculation using conditional power. Stat Med, 2001, 20(17-18): 2645-2660.
19. Park JJH, Siden E, Zoratti MJ, et al. Systematic review of basket trials, umbrella trials, and platform trials: a landscape analysis of master protocols. Trials, 2019, 20(1): 572.
20. 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.
21. Berry SM, Connor JT, Lewis RJ. The platform trial: an efficient strategy for evaluating multiple treatments. JAMA, 2015, 313(16): 1619-1620.
22. 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.
23. Steuer CE, Papadimitrakopoulou V, Herbst RS, et al. Innovative clinical trials: the LUNG-MAP Study. Clin Pharmacol Ther, 2015, 97(5): 488-491.
24. Angus DC, Derde L, Al-Beidh F, et al. Effect of hydrocortisone on mortality and organ support in patients with severe COVID-19: the REMAP-CAP COVID-19 corticosteroid domain randomized clinical trial. JAMA, 2020, 324(13): 1317-1329.
25. RECOVERY Central Coordinating Office. RECOVERY protocol V1.0. Available at: https://www.recoverytrial.net/files/protocol-archive/recovery-protocol-v1-0-2020-03-18-final.pdf.

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