National Institutes of Health Office of Extramural Research, One Center Drive, Building 1, Room 144, Bethesda, Maryland 20892, USA.
Division of Cardiovascular Sciences of the National Heart, Lung, and Blood Institute, 6701 Rockledge Drive, 8th Floor, Bethesda, Maryland 20892, USA.
Nat Rev Cardiol. 2017 Aug;14(8):493-501. doi: 10.1038/nrcardio.2017.60. Epub 2017 Apr 27.
Randomized clinical trials and large-scale, cohort studies continue to have a critical role in generating evidence in cardiovascular medicine; however, the increasing concern is that ballooning costs threaten the clinical trial enterprise. In this Perspectives article, we discuss the changing landscape of clinical research, and clinical trials in particular, focusing on reasons for the increasing costs and inefficiencies. These reasons include excessively complex design, overly restrictive inclusion and exclusion criteria, burdensome regulations, excessive source-data verification, and concerns about the effect of clinical research conduct on workflow. Thought leaders have called on the clinical research community to consider alternative, transformative business models, including those models that focus on simplicity and leveraging of digital resources. We present some examples of innovative approaches by which some investigators have successfully conducted large-scale, clinical trials at relatively low cost. These examples include randomized registry trials, cluster-randomized trials, adaptive trials, and trials that are fully embedded within digital clinical care or administrative platforms.
随机临床试验和大规模队列研究继续在心血管医学领域产生重要证据；然而，人们越来越担心的是，不断膨胀的成本威胁着临床试验事业。在这篇观点文章中，我们讨论了临床研究，特别是临床试验不断变化的格局，重点探讨了成本不断增加和效率低下的原因。这些原因包括设计过于复杂、纳入和排除标准过于严格、监管负担过重、过度的数据来源验证，以及对临床研究实施对工作流程影响的担忧。思想领袖呼吁临床研究界考虑替代的、变革性的商业模式，包括那些专注于简化和利用数字资源的商业模式。我们介绍了一些创新方法的示例，一些研究人员通过这些方法成功地以相对较低的成本进行了大规模临床试验。这些示例包括随机登记试验、整群随机试验、适应性试验，以及完全嵌入数字临床护理或行政平台的试验。
