Department of Radiation Oncology, Abramson Cancer Center, University of Pennsylvania, Philadelphia, Pennsylvania; Leonard Davis Institute for Health Economics, University of Pennsylvania, Philadelphia, Pennsylvania.
Department of Radiation Oncology, Abramson Cancer Center, University of Pennsylvania, Philadelphia, Pennsylvania.
Pract Radiat Oncol. 2011 Apr-Jun;1(2):72-80. doi: 10.1016/j.prro.2011.02.001. Epub 2011 Apr 8.
The essence of comparative effectiveness research (CER) is to understand what health interventions work, for which patients, and under what conditions. The objective of this article is to introduce the relative strengths and weaknesses of several forms of evidence to illustrate the potential for CER evidence generation within radiation oncology.
We introduce the underlying concepts of effectiveness and efficacy. We describe the design of traditional explanatory randomized trials (RCTs). We introduce the rationale, strengths, and weaknesses of several alternative study designs for comparative effectiveness, including pragmatic clinical trials, adaptive trials, and observational (nonrandomized) studies.
Explanatory RCTs are designed to assess the efficacy of an intervention while achieving a high degree of internal validity. Pragmatic clinical trials (PCTs) are prospective studies performed in typical, real-world clinical practice settings. The emphasis of PCTs is to maintain a degree of internal validity while also maximizing external validity. Adaptive trials can be modified at interim stages using existing or evolving evidence in the course of a trial, which may allow trials to maintain clinical relevance by studying current treatments. Observational data are becoming increasingly important, given substantial funding for clinical registries and greater availability of electronic medical records and claims databases, but need to address well-known limitations such as selection bias.
With the rapid proliferation of new and evolving radiotherapy technologies, it is incumbent upon our field to invest in building the evidence base for radiotherapy CER and to actively participate in current initiatives for generating comparative evidence.
比较疗效研究（CER）的本质是了解哪些干预措施有效，对哪些患者有效，以及在什么条件下有效。本文的目的是介绍几种证据形式的相对优势和劣势，以说明在放射肿瘤学中生成 CER 证据的潜力。
我们介绍了有效性和疗效的基本概念。我们描述了传统解释性随机对照试验（RCT）的设计。我们介绍了几种替代比较有效性研究设计的原理、优势和劣势，包括实用临床试验、适应性试验和观察性（非随机）研究。
解释性 RCT 旨在评估干预措施的疗效，同时实现高度的内部有效性。实用临床试验（PCTs）是在典型的、真实世界的临床实践环境中进行的前瞻性研究。PCT 的重点是在保持一定程度的内部有效性的同时，最大限度地提高外部有效性。适应性试验可以在试验过程中使用现有或不断发展的证据在中期阶段进行修改，这可能允许试验通过研究当前的治疗方法来保持临床相关性。观察性数据变得越来越重要，因为临床登记处有大量资金投入，电子病历和索赔数据库的可用性也越来越高，但需要解决选择偏差等众所周知的局限性。
随着新的和不断发展的放射治疗技术的迅速普及，我们有责任投资于放射治疗 CER 的证据基础建设，并积极参与当前生成比较证据的倡议。
