Contemporary strategies to improve clinical trial design for critical care research: insights from the First Critical Care Clinical Trialists Workshop
Contemporary strategies to improve clinical trial design for critical care research: insights from the First Critical Care Clinical Trialists Workshop
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DOI：
10.1007/s00134-020-05934-6
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发表时间：
2020-02-18
影响因子：
38.9
通讯作者：
Mebazaa, Alexandre
中科院分区：
文献类型：
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作者：
Harhay, Michael O.;Casey, Jonathan D.;Mebazaa, Alexandre
在危重患者人群中进行研究是具有挑战性的，大多数危重患者的随机试验尚未达到预先规定的统计阈值，以得出正在研究的干预措施是有益的结论。方法2019年，由患者代表、来自美国和欧盟的监管机构、联邦拨款经理、行业代表、临床试验专家、流行病学家和临床医生组成的多元化团体召开了第一次重症监护临床试验专家（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，我们提出了临床试验设计，实施和分析的现有方法的优点和缺点，以及一系列建议，以潜在地改善未来的试验在重症监护。结论：3CT研讨会的参与者确定了改善重症监护试验的机会，使用优化样本量计算的策略，考虑患者和疾病的异质性，提高试验实施的效率，最大限度地利用试验数据，并完善和标准化收集以患者为中心和患者知情的结局指标，而不是死亡率。
Background 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. Methods 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. Conclusion 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.