School of Health and Population Sciences, University of Birmingham, Edgbaston, Birmingham B15 2TT, UK.
J Clin Epidemiol. 2010 Oct;63(10):1053-8. doi: 10.1016/j.jclinepi.2009.07.013. Epub 2009 Oct 31.
Health care providers, policy makers, and importantly patients themselves are increasingly interested in the outcomes of clinical trials yet often expect different questions to be addressed than those commonly asked in conventional phase 3 trials.
Review of methodological articles.
Conventional randomized controlled trials (RCTs) emphasize internal validity through standardization and control but by design reduce external validity, that is, generalizability of results and conclusions. Ongoing uncertainty about effectiveness or safety of medical interventions in the real world is the major driver for developing improved phase 3b and phase 4 study designs. Factors that should improve the relevance of these real-world trials (RWTs) include choice of endpoints; investigator specialty, appropriate patient selection criteria; emphasis on patient-physician interaction; admittance of relevant interventions in all study groups; and more flexible, simple, and possibly event-driven study visits and procedures, while maintaining randomization as a critical element to address confounders.
Although we do not believe that RWTs will supplant conventional RCTs, properly designed RWTs will enrich our understanding of the effectiveness of new health care interventions and better inform patients and health care providers alike.
医疗保健提供者、政策制定者，尤其是患者自身，对临床试验的结果越来越感兴趣，但他们通常期望解决的问题与传统的 3 期试验中常见的问题不同。
方法学文章的回顾。
传统的随机对照试验（RCT）通过标准化和控制强调内部有效性，但设计上降低了外部有效性，即结果和结论的普遍性。在现实世界中，对于医疗干预措施的有效性或安全性的持续不确定性是开发改进的 3b 期和 4 期研究设计的主要驱动力。提高这些真实世界试验（RWT）相关性的因素包括终点的选择；研究人员的专业知识、适当的患者选择标准；强调患者-医生的互动；允许所有研究组接受相关干预措施；以及更灵活、简单、可能是基于事件的研究访问和程序，同时保持随机化作为解决混杂因素的关键要素。
尽管我们不认为 RWT 将取代传统的 RCT，但设计得当的 RWT 将丰富我们对新医疗干预措施有效性的理解，并更好地为患者和医疗保健提供者提供信息。
