Please use this identifier to cite or link to this item: https://ahro.austin.org.au/austinjspui/handle/1/27194
Title: How cutting-edge trial design can assess outcomes.
Austin Authors: Serpa Neto, Ary ;Goligher, Ewan C;Hodgson, Carol L
Affiliation: Department of Physiotherapy, The Alfred Hospital, Melbourne, Victoria, Australia
Department of Critical Care Medicine, Hospital Israelita Albert Einstein, Sao Paulo, Brazil
Interdepartmental Division of Critical Care Medicine, University of Toronto
Department of Medicine, Division of Respirology, University of Health Network Toronto General Hospital Research Institute, Toronto, Canada
Australian and New Zealand Intensive Care Research Centre (ANZIC-RC), School of Public Health and Preventive Medicine, Monash University
Department of Critical Care, Melbourne Medical School, University of Melbourne
Data Analytics Research and Evaluation (DARE) Centre
Issue Date: 30-Jul-2021
Date: 2021-07-30
Publication information: Current Opinion in Critical Care 2021; online first: 30 July
Abstract: Randomized clinical trials (RCTs) have come to be accepted as the gold standard for assessing the efficacy and effectiveness of therapeutics and interventions in medicine. In this paper, we aim to describe some evolving concepts associated with the design and conduct of RCTs and outline new approaches aiming to increase efficiency and reduce costs. A well-powered and performed RCT is usually a study involving several different centers from different geographical areas that enrolls a large number of patients in diverse clinical settings. Altogether, these features increase the generalizability of the study and make the rapid implementation of the findings more likely. However, this does not come without cost. Among several possible alternatives to conventional RCTs, the most important ones are related to the unit of randomization (individual vs. cluster), study design (conventional vs. adaptive), randomization scheme (fixed vs. response-adaptive), data collection (conventional case report forms vs. registry-embedded) and statistical approach (frequentist vs. Bayesian). While conventional RCTs remain the gold standard for generating evidence, new trial designs may be considered to reduce sample size and costs while improving trial efficiency and power. However, they raise new challenges for testing feasibility, conduct, ethical oversight and statistical analysis.
URI: https://ahro.austin.org.au/austinjspui/handle/1/27194
DOI: 10.1097/MCC.0000000000000854
Journal: Current Opinion in Critical Care
PubMed URL: 34334624
Type: Journal Article
Appears in Collections:Journal articles

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