Global Health Outcomes, Eli Lilly and Company, Indianapolis, IN, USA.
Value Health. 2009 Nov-Dec;12(8):1044-52. doi: 10.1111/j.1524-4733.2009.00600.x. Epub 2009 Sep 29.
Health insurers, physicians, and patients worldwide need information on the comparative effectiveness and safety of prescription drugs in routine care. Nonrandomized studies of treatment effects using secondary databases may supplement the evidence based from randomized clinical trials and prospective observational studies. Recognizing the challenges to conducting valid retrospective epidemiologic and health services research studies, a Task Force was formed to develop a guidance document on state of the art approaches to frame research questions and report findings for these studies.
The Task Force was commissioned and a Chair was selected by the International Society for Pharmacoeconomics and Outcomes Research Board of Directors in October 2007. This Report, the first of three reported in this issue of the journal, addressed issues of framing the research question and reporting and interpreting findings.
The Task Force Report proposes four primary characteristics-relevance, specificity, novelty, and feasibility while defining the research question. Recommendations included: the practice of a priori specification of the research question; transparency of prespecified analytical plans, provision of justifications for any subsequent changes in analytical plan, and reporting the results of prespecified plans as well as results from significant modifications, structured abstracts to report findings with scientific neutrality; and reasoned interpretations of findings to help inform policy decisions.
Comparative effectiveness research in the form of nonrandomized studies using secondary databases can be designed with rigorous elements and conducted with sophisticated statistical methods to improve causal inference of treatment effects. Standardized reporting and careful interpretation of results can aid policy and decision-making.
全球的健康保险公司、医生和患者都需要了解处方药物在常规治疗中的比较效果和安全性信息。利用二级数据库进行的非随机治疗效果研究可以补充基于随机临床试验和前瞻性观察研究的证据。鉴于开展有效的回顾性流行病学和卫生服务研究存在挑战，成立了一个工作组来制定一份指导文件，介绍这些研究中提出研究问题和报告研究结果的最新方法。
2007 年 10 月，国际药物经济学和结果研究学会理事会委托并选举了一个主席来领导工作组。本报告是该期刊本期报道的三篇报告中的第一篇，主要探讨了提出研究问题和报告和解释研究结果的问题。
工作组的报告提出了四项主要特征，即相关性、特异性、新颖性和可行性，同时定义了研究问题。建议包括：预先规定研究问题的实践；预先指定分析计划的透明度，为任何后续分析计划的变更提供理由，并报告预先指定计划的结果以及重大修改后的结果，使用结构化摘要以科学中立的方式报告研究结果；以及对研究结果进行合理的解释，以帮助做出政策决策。
利用二级数据库进行的非随机研究形式的比较效果研究可以通过严格的设计和复杂的统计方法来提高治疗效果的因果推断。标准化的报告和仔细的结果解释可以帮助制定政策和决策。
