大型简单随机对照试验——从药物到医疗设备:来自近期经验的教训。

IF 2 4区 医学 Q3 MEDICINE, RESEARCH & EXPERIMENTAL
Trials Pub Date : 2025-01-21 DOI:10.1186/s13063-025-08724-x
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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引用次数: 0

摘要

随机对照试验(RCTs)是现代循证医学的基石。它们被认为是确定新药有效性和安全性的明确证据所必需的,并且在可能的情况下,它们也应该是调查新的高风险医疗器械的首选方法。设计良好的研究为临床实践指南和决策提供了强有力的信息,但行政上的障碍使得开展信息性随机对照试验越来越困难。对于高风险医疗设备的随机对照试验来说,由于植入设备所需的介入程序的额外费用,在整个试验过程中随机分配患者的意愿面临挑战,以及即使使用假程序也难以确保适当的盲法,这些障碍更加复杂。一种可能有所帮助的策略是促进更广泛地使用更简单和更精简的随机对照试验,这些随机对照试验使用在医疗保健提供过程中常规收集的数据。最近的大型简单随机对照试验成功地将药物和高风险医疗设备的性能与替代疗法进行了比较;他们在短时间内招募了许多患者,限制了成本,提高了效率,同时也取得了重大影响。在CORE-MD项目中进行的一项任务中,我们报告了我们在COVID-19大流行期间设计和实施大型药物试验以及计划和协调大型基于注册表的心血管装置随机对照试验的综合经验。我们总结了大型简单随机对照试验的基本原则和效用,可能适用于所有干预措施,但特别是为了促进其更广泛地应用于评估新的医疗器械。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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.

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来源期刊
Trials
Trials 医学-医学:研究与实验
CiteScore
3.80
自引率
4.00%
发文量
966
审稿时长
6 months
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.
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