TY - JOUR
T1 - Contemporary strategies to improve clinical trial design for critical care research
T2 - insights from the First Critical Care Clinical Trialists Workshop
AU - Harhay, Michael O.
AU - Casey, Jonathan D.
AU - Clement, Marina
AU - Collins, Sean P.
AU - Gayat, Étienne
AU - Gong, Michelle Ng
AU - Jaber, Samir
AU - Laterre, Pierre François
AU - Marshall, John C.
AU - Matthay, Michael A.
AU - Monroe, Rhonda E.
AU - Rice, Todd W.
AU - Rubin, Eileen
AU - Self, Wesley H.
AU - Mebazaa, Alexandre
N1 - Funding Information:
MOH was supported by the United States National Institutes of Health (NIH)/National Heart, Lung, and Blood Institute (NHLBI) grant K99 HL141678 and R00 HL141678. JDC was supported in part by the NIH/NHLBI (T32HL087738-12, K12HL133117). MAM was supported in part by NHLBI U01 HL 123004, HL140026, HL134828. MNG was supported by NHLBI UH2 HL125119, U01 HL122998, AHRQ R18 HS026188. TWR was supported in part by NHLBI U01 HL123009, NCRR U54 RR 032646, NCATS U24 TR 001608. WHS was supported in part by NHLBI U01HL123009 and NIGMS K23GM110469. JCM was supported in part by the Canadian Institutes of Health Research MYG158584.
Publisher Copyright:
© 2020, Springer-Verlag GmbH Germany, part of Springer Nature.
PY - 2020/5/1
Y1 - 2020/5/1
N2 - 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.
AB - 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.
KW - Acute respiratory distress syndrome
KW - Clinical trials
KW - Critical care
KW - Sepsis
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U2 - 10.1007/s00134-020-05934-6
DO - 10.1007/s00134-020-05934-6
M3 - Article
C2 - 32072303
AN - SCOPUS:85079745437
VL - 46
SP - 930
EP - 942
JO - Intensive Care Medicine
JF - Intensive Care Medicine
SN - 0342-4642
IS - 5
ER -