Palliative and Advanced Illness Research (PAIR) Center, Perelman School of Medicine, University of Pennsylvania, 304 Blockley Hall, 423 Guardian Drive, Philadelphia, PA, 19104-6021, USA.
Department of Biostatistics, Epidemiology and Informatics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Intensive Care Med. 2020 May;46(5):930-942. doi: 10.1007/s00134-020-05934-6. Epub 2020 Feb 18.
Conducting research in critically-ill patient populations is challenging, and most randomized trials of critically-ill patients have not achieved pre-specified statistical thresholds to conclude that the intervention being investigated was beneficial.
In 2019, a diverse group of patient representatives, regulators from the USA and European Union, federal grant managers, industry representatives, clinical trialists, epidemiologists, and clinicians convened the First Critical Care Clinical Trialists (3CT) Workshop to discuss challenges and opportunities in conducting and assessing critical care trials. Herein, we present the advantages and disadvantages of available methodologies for clinical trial design, conduct, and analysis, and a series of recommendations to potentially improve future trials in critical care.
The 3CT Workshop participants identified opportunities to improve critical care trials using strategies to optimize sample size calculations, account for patient and disease heterogeneity, increase the efficiency of trial conduct, maximize the use of trial data, and to refine and standardize the collection of patient-centered and patient-informed outcome measures beyond mortality.
在重症患者人群中开展研究具有挑战性，大多数重症患者的随机试验都未达到预先设定的统计学阈值，无法得出所研究的干预措施有益的结论。
在 2019 年，一组多元化的患者代表、来自美国和欧盟的监管机构代表、联邦拨款管理者、行业代表、临床试验人员、流行病学家和临床医生举行了首次重症监护临床研究人员（3CT）研讨会，讨论开展和评估重症监护试验的挑战和机遇。在此，我们介绍了现有的临床试验设计、实施和分析方法的优缺点，并提出了一系列建议，以期改善未来的重症监护试验。
3CT 研讨会的参与者确定了利用策略来优化样本量计算、考虑患者和疾病异质性、提高试验实施效率、最大限度地利用试验数据以及完善和标准化除死亡率以外的以患者为中心和患者知情的结局指标的收集，从而改善重症监护试验的机会。
