Explore the Applications and Challenges of Adaptive Design in Platform trial
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摘要: 适应性设计以动态调整试验设计、减少资源浪费、提高试验效率等优势突破了新药研发的竞争态势,逐渐满足临床需求。近年来,结合适应性设计的平台试验作为一种创新研究模式,为新药研发增添了动力。本文简要概述适应性设计的研究进展、内容及特点、设计类型、统计分析方法和案例解读,介绍自适应平台试验的概念、类型及应用,以期为深入探索临床试验和新药研发提供科学参考。Abstract: Adaptive design, with advantages such as dynamically adjusting trial plans, reducing resource waste, and improving trial efficiency, has broken through the competitive situation of new drug development and gradually met the needs of clinical research. In recent years, the use of adaptive design in platform trials as an innovative research model has added impetus to new drug development. This article briefly outlines the research progress , content and characteristics, common design types, statistical analysis, and case interpretation of adaptive design, introduces the concept, types, and applications of adaptive platform trials, in the hope to provide scientific references for further exploration of clinical trials and new drug development.
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Key words:
- adaptive design /
- master protocol design /
- platform trial
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[1] 杜艾桦. 药物临床试验适应性设计的科学性与伦理性问题[J]. 药物流行病学杂志, 2017, 26(12): 837- 840. [2] 王雨宁,徐畅,邓可等.适应性设计在临床试验中的应用[J]. 中国循证医学杂志, 2020, 20(4): 487-491. [3] 杨凯璇,周齐,姜英玉等.适应性设计随机对照临床试验[J].中国卒中杂志, 2022, 7(10): 1152-1158. [4] Food and Drug Administration. Deputy Commissioner for Medical and Scientific Affairs. Washington, DC, 2006. [5] European Medicines Agency. Reflection paper on methodological issues in confirmatory clinical trials planned with an adaptive design. 2007. [6] Collignon O, Koenig F, Koch A. et al. Adaptive designs in clinical trials: from scientific advice to marketing authorization to the European medicine agency[J]. Trials, 2018. 19(1):642. [7] Food and Drug Administration. Guidance for industry : adaptive design clinical trials for drugs and biologics. 2010. [8] United States Government. Rules committee print 114-67, text of House amendment to the Senate, amendment to H.R.34, Tsunami Warning, Education, and Research Act of 2015, 2016: 162-3. 114th Congress. [9] US Food and Drug Administration. Adaptive designs for clinical trials of drugs and biologics guidance for industry. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/adaptive-designclinical-trials-drugs-and-biologics-guidance-industry. [10] 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)[EB/0L1.2021-01- 29)(2023-11-26). https://www.cde.org.cn/zdyz/domesticinfopage?zdyzIdCODE=4409e51a403a911757af6caf3ecef129. [11] 衡明莉,王北琪,王骏. 对美国FDA适应性设计指导原则的介绍[J]. 中国临床药理学杂志, 2019, 35(12): 1316-1320. [12] Wasson JMS, Demario M, Biggs K, et al. Practical guidance for planning resources required to support publicly funded adaptive clinical trials[J]. BMC Med, 2022, 20(1): 254. [13] Burnett T, Mozgunov P, Pallmann P, et al. Adding flexibility to clinical trial designs: an example-based guide to the practical use of adaptive designs[J]. BMC Med, 2020, 18: 352. [14] Pallmann P, Bedding AW, Choodari-Oskooei B, et al. Adaptive designs in clinical trials: Why use them, and how to run and report them[J]. BMC Med, 2018. [15] Jones RL, Ravi V, Brohl AS, et al. Efficacy and safety of TRC105 plus pazopanib vs pazopanib alone for treatment of patients with advanced angiosarcoma: A randomized clinical trial[J]. JAMA Oncol, 2022, 8(5): 740-747. [16] Perkins GD, Quinn T, Deakin CD, et al. Pre-hospital Assessment of the Role of Adrenaline: Measuring the Effectiveness of Drug administration In Cardiac arrest (PARAMEDIC-2): Trial protocol[J]. Resuscitation. 2016, 108: 75-81. [17] Perkins GD, Ji C, Deakin CD, et al. A randomized trial of epinephrine in out-of-hospital cardiac arrest[J]. N Engl J Med, 2018, 379(8): 711-721. [18] Kapur J, Elm J, Chamberlain JM, et al. Randomized trial of three anticonvulsant medications for status epilepticus[J]. N Engl J Med, 2019, 381(22): 2103-2113. [19] Suchting R, Green CE, de Dios C, et al. Citalopram for treatment of cocaine use disorder: A Bayesian dropthe-loser randomized clinical trial[J]. Drug Alcohol Depen, 2021, 228: 109054. [20] Filozof C, Chow SC, Dimick-Santos L, et al. Clinical endpoints and adaptive clinical trials in precirrhotic nonalcoholic steatohepatitis: Facilitating development approaches for an emerging epidemic[J]. Hepatol Commun, 2017, 1(7): 577-585. [21] Dawson SN, Chiu YD, Klein AA, et al. Effect of high-flow nasal therapy on patient-centred outcomes in patients at high risk of postoperative pulmonary complications after cardiac surgery: A statistical analysis plan for NOTACS, a multicentre adaptive randomized controlled trial[J]. Trials, 2022, 23(1): 699. [22] Sevransky JE, Rothman RE, Hager DN, et al Effect of Vitamin C, Thiamine, and Hydrocortisone on Ventilatorand Vasopressor-Free Days in Patients with Sepsis: The VICTAS Randomized Clinical Trial[J]. JAMA, 2021, 325(8): 742-750. Giovagnoli A. The Bayesian Design of Adaptive Clinical Trials. Int J Environ Res Public Health. 2021; 18(2): 530. [23] Giovagnoli A. The Bayesian Design of Adaptive Clinical Trials. Int J Environ Res Public Health. 2021; 18(2): 530. [24] Lai TL, Lavori PW, Shih MC. Adaptive trial designs. Annu Rev Pharmacol Toxicol. 2012; 52: 101-110. [25] Grayling MJ, Wheeler GM. A review of available software for adaptive clinical trial design. Clin Trials. 2020; 17(3): 323-331. [26] Swanson CJ, Zhang Y, Dhadda S, et al. A randomized, double-blind, phase 2b proof-of-concept clinical trial in early Alzheimer's disease with lecanemab,an anti-Aβ protofibril antibody. Alzheimers Res Ther. 2022; 14(1): 70. [27] Hager DN, Hooper MH, Bernard GR, et al. The vitamin C, thiamine, and steroids in sepsis (VICTAS) protocol: A prospective, multi-center, double-blind, adaptive sample size, randomized, placebo-controlled, clinical trial[J]. Trials, 2019, 20(1): 1-16. [28] Sudhop T, Brun NC, Riedel C, Rosso A, Broich K, Senderovitz T. Master protocols in clinical trials: a universal Swiss Army knife[J]. Lancet Oncol, 2019, 20(6): e336-e342. [29] Hirakawa A, Asano J, Sato H, et al. Master protocol trials in oncology: Review and new trial designs[J]. Contemp Clin Trials Commun, 2018, 12: 1-8. [30] Nature Reviews Drug Discovery. Adaptive platform trials coalition. Adaptive platform trials: Definition, design, conduct and reporting considerations[J]. Nat Rev Drug Discov, 2019, 18(10): 79-807. [31] 金秋百,张颖,姜枫等. 反应适应性随机的实施要点及其在中医药临床研究中的应用前景[J]. 中医杂志, 2021, 62(17): 1478-1483. [32] Berry SM, Connor JT, Lewis RJ. The platform trial: an efficient strategy for evaluating multiple treatments[J]. JAMA, 2015, 313(16): 1619-1620. [33] Hey SP, Kimmelman J. Are outcome-adaptive allocation trials ethical[J]. Clin Trials, 2015, 12(2): 102-106. [34] Parmar MKB, Sydes MR, Cafferty FH, et al. Testing many treatments within a single protocol over 10 years at MRC Clinical Trials Unit at UCL: Multi-arm, multi-stage platform, umbrella and basket protocols[J]. Clin Trials, 2017, 14(5): 451-61. [35] Riddell CA, Zhao Y, Petkau J. An adaptive clinical trials procedure for a sensitive subgroup examined in themultiple sclerosis context[J]. Stat Methods Med Res, 2016, 25(4): 1330-45. [36] Wang G, Kennedy RE, Cutter GR, et al. Effect of sample size re-estimation in adaptive clinical trials for Alzheimer's disease and mild cognitive impairment[J]. Alzheimers Dement(N Y), 2015, 1(1): 63-71. [37] Meurer WJ, Barsan WG. Spinal cord injury neuroprotection and the promise of flexible adaptive clinical trials[J]. World Neurosurg, 2014, 82(3-4): e541-6. [38] Rugo HS, Olopade OI, DeMichele A, et al. Adaptive randomization of veliparib-carboplatin treatment in breast cancer[J]. N Engl J Med, 2016, 375(1): 23-34. [39] James ND, Sydes MR, Clarke NW, et al. Systemic therapy for advancing or metastatic prostate cancer (STAMPEDE): A multi-arm, multistage randomized controlled trial[J]. BJU Int, 2009, 103(4): 464-9. [40] Petersen E, Koopmans M, Go U, et al. Comparing SARS-CoV-2 with SARS-CoV and influenza pandemics[J]. Lancet Infect Dis, 2020, 20(9): e238-e244. [41] Khubchandani J, Jordan TR, Yang YT. Ebola, zika, corona…what is next for our world[J]. Int J Environ Res Public Health, 2020, 17(9): 3171. [42] Ellenberg SS: Clinical trials in the time of a pandemic[J]. Clin Trials, 2020, 17(5): 467-471. [43] Mulangu S, Dodd LE, Davey RT, et al. A randomized, controlled trial of Ebola virus disease therapeutics[J]. N Engl J Med, 2019, 381(24): 2293-303. [44] RECOVERY Collaborative Group. Horby P, Lim WS, Emberson JR, et al. Dexamethasone in Hospitalized Patients with Covid-19[J]. N Engl J Med. 2021, 384(8): 693-704. [45] Beigel JH, Tomashek KM, Dodd LE, et al. Remdesivir for the Treatment of Covid-19-Final Report[J]. N Engl J Med, 2020, 383(19): 1813-1826. [46] Noor NM, Pett SL, Esmail H, et al. Adaptive platform trials using multi-arm, multi-stage protocols: getting fast answers in pandemic settings. F1000Res, 2020; (9): 1109. [47] Morgan CC, Huyck Jenkins M, et al. Adaptive Design: Results of 2012 Survey on Perception and Use[J]. Ther Innov Regul Sci, 2014, 48(4): 473-81. [48] Bothwell LE, Avorn J, Khan NF, et al. Adaptive design clinical trials: A review of the literature and ClinicalTrials.gov[J]. BMJ Open, 2018, 8(2): e018320. [49] 赵超.临床适应性设计与药物评价的考虑[J].中国临床药理学与治疗学, 2008(1): 1-5. [50] 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[J]. 中国循证医学杂志, 2022, 22(8): 971-977. [51] 范扬.贝叶斯响应适应性随机化及其交互式Web应用实现[D]. 东南大学, 2020. [52] Wason JMS, Demario M, Biggs K, et al. Practical guidance for planning resources required to support publicly funded adaptive clinical trials[J]. BMC Med, 2022, 20(1): 254.
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