Kinnier Christine V, Chung Jeanette W, Bilimoria Karl Y
Department of Surgery, Massachusetts General Hospital, Boston, MA, USA.
Cancer Treat Res. 2015;164:1-14. doi: 10.1007/978-3-319-12553-4_1.
While randomized controlled trials (RCTs) are the gold standard for research, many research questions cannot be ethically and practically answered using an RCT. Comparative effectiveness research (CER) techniques are often better suited than RCTs to address the effects of an intervention under routine care conditions, an outcome otherwise known as effectiveness. CER research techniques covered in this section include: effectiveness-oriented experimental studies such as pragmatic trials and cluster randomized trials, treatment response heterogeneity, observational and database studies including adjustment techniques such as sensitivity analysis and propensity score analysis, systematic reviews and meta-analysis, decision analysis, and cost effectiveness analysis. Each section describes the technique and covers the strengths and weaknesses of the approach.
虽然随机对照试验(RCT)是研究的金标准,但许多研究问题无法通过RCT在伦理和实际操作上得到解答。比较效果研究(CER)技术通常比RCT更适合解决常规护理条件下干预措施的效果,这一结果也被称为有效性。本节涵盖的CER研究技术包括:以有效性为导向的实验研究,如实用试验和整群随机试验;治疗反应异质性;观察性和数据库研究,包括敏感性分析和倾向得分分析等调整技术;系统评价和荟萃分析;决策分析;以及成本效益分析。每个部分都描述了该技术,并阐述了该方法的优缺点。