Sergio Buccheri, Stefan James, Marion Mafham, Martin Landray, Tom Melvin, Jonas Oldgren, Richard Bulbulia, Louise Bowman, Lotje Anna Hoogervorst, Perla J Marang-van de Mheen, Peter Juni, Peter McCulloch, Alan G Fraser
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The obstacles are compounded for RCTs of high-risk medical devices by extra costs related to the interventional procedure that is needed to implant the device, challenges with willingness to randomize patients throughout a trial, and difficulties in ensuring proper blinding even with sham procedures. One strategy that may help is to promote the wider use of simpler and more streamlined RCTs using data that are collected routinely during healthcare delivery. Recent large simple RCTs have successfully compared the performance of drugs and of high-risk medical devices, against alternative treatments; they enrolled many patients in a short time, limited costs, and improved efficiency, while also achieving major impact. From a task conducted within the CORE-MD project, we report from our combined experience of designing and conducting large pharmaceutical trials during the COVID-19 pandemic, and of planning and coordinating large registry-based RCTs of cardiovascular devices. 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Large simple randomized controlled trials-from drugs to medical devices: lessons from recent experience.
Randomized controlled trials (RCTs) are the cornerstone of modern evidence-based medicine. They are considered essential to establish definitive evidence of efficacy and safety for new drugs, and whenever possible they should also be the preferred method for investigating new high-risk medical devices. Well-designed studies robustly inform clinical practice guidelines and decision-making, but administrative obstacles have made it increasingly difficult to conduct informative RCTs. The obstacles are compounded for RCTs of high-risk medical devices by extra costs related to the interventional procedure that is needed to implant the device, challenges with willingness to randomize patients throughout a trial, and difficulties in ensuring proper blinding even with sham procedures. One strategy that may help is to promote the wider use of simpler and more streamlined RCTs using data that are collected routinely during healthcare delivery. Recent large simple RCTs have successfully compared the performance of drugs and of high-risk medical devices, against alternative treatments; they enrolled many patients in a short time, limited costs, and improved efficiency, while also achieving major impact. From a task conducted within the CORE-MD project, we report from our combined experience of designing and conducting large pharmaceutical trials during the COVID-19 pandemic, and of planning and coordinating large registry-based RCTs of cardiovascular devices. We summarize the essential principles and utility of large simple RCTs, likely applicable to all interventions but especially in order to promote their wider adoption to evaluate new medical devices.
期刊介绍:
Trials is an open access, peer-reviewed, online journal that will encompass all aspects of the performance and findings of randomized controlled trials. Trials will experiment with, and then refine, innovative approaches to improving communication about trials. We are keen to move beyond publishing traditional trial results articles (although these will be included). We believe this represents an exciting opportunity to advance the science and reporting of trials. Prior to 2006, Trials was published as Current Controlled Trials in Cardiovascular Medicine (CCTCVM). All published CCTCVM articles are available via the Trials website and citations to CCTCVM article URLs will continue to be supported.