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自适应平台试验在临床研究中的应用

马艳 程千吉 陆瑶 宁金铃 葛龙

马艳, 程千吉, 陆瑶, 宁金铃, 葛龙. 自适应平台试验在临床研究中的应用[J]. 协和医学杂志. doi: 10.12290/xhyxzz.2023-0424
引用本文: 马艳, 程千吉, 陆瑶, 宁金铃, 葛龙. 自适应平台试验在临床研究中的应用[J]. 协和医学杂志. doi: 10.12290/xhyxzz.2023-0424
MY Yan, CHENG Qianji, LU Yao, NING Jinling, GE Long. Explore the Applications and Challenges of Adaptive Design in Platform trial[J]. Medical Journal of Peking Union Medical College Hospital. doi: 10.12290/xhyxzz.2023-0424
Citation: MY Yan, CHENG Qianji, LU Yao, NING Jinling, GE Long. Explore the Applications and Challenges of Adaptive Design in Platform trial[J]. Medical Journal of Peking Union Medical College Hospital. doi: 10.12290/xhyxzz.2023-0424

自适应平台试验在临床研究中的应用

doi: 10.12290/xhyxzz.2023-0424
基金项目: 

中国中医科学院创新基金(CI2021A05502)

详细信息
  • 中图分类号: R969.4;R965.3;R181.2

Explore the Applications and Challenges of Adaptive Design in Platform trial

Funds: 

China Academy of Chinese Medical Sciences Innovation Fund (CI2021A05502)

  • 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。
  • [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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出版历程
  • 收稿日期:  2023-09-07
  • 录用日期:  2023-12-13
  • 网络出版日期:  2024-01-30

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