1 Cochin Hospital Group, APHP and University Paris Descartes, Paris, France;
Ann Am Thorac Soc. 2014 Feb;11 Suppl 2:S99-104. doi: 10.1513/AnnalsATS.201309-300RM.
Real-world research can use observational or clinical trial designs, in both cases putting emphasis on high external validity, to complement the classical efficacy randomized controlled trials (RCTs) with high internal validity. Real-world research is made necessary by the variety of factors that can play an important a role in modulating effectiveness in real life but are often tightly controlled in RCTs, such as comorbidities and concomitant treatments, adherence, inhalation technique, access to care, strength of doctor-caregiver communication, and socio-economic and other organizational factors. Real-world studies belong to two main categories: pragmatic trials and observational studies, which can be prospective or retrospective. Focusing on comparative database observational studies, the process aimed at ensuring high-quality research can be divided into three parts: preparation of research, analyses and reporting, and discussion of results. Key points include a priori planning of data collection and analyses, identification of appropriate database(s), proper outcomes definition, study registration with commitment to publish, bias minimization through matching and adjustment processes accounting for potential confounders, and sensitivity analyses testing the robustness of results. When these conditions are met, observational database studies can reach a sufficient level of evidence to help create guidelines (i.e., clinical and regulatory decision-making).
真实世界研究可以采用观察性或临床试验设计，这两种方法都强调外部有效性，以补充具有高内部有效性的经典疗效随机对照试验（RCT）。由于多种因素可能在调节现实生活中的疗效方面发挥重要作用，但在 RCT 中通常受到严格控制，例如合并症和伴随治疗、依从性、吸入技术、获得医疗保健的机会、医生-护理人员沟通的力度以及社会经济和其他组织因素等，因此需要进行真实世界研究。真实世界研究属于两个主要类别：实用试验和观察性研究，可分为前瞻性或回顾性。关注比较数据库观察性研究，旨在确保高质量研究的过程可分为三部分：研究准备、分析和报告，以及结果讨论。关键点包括预先规划数据收集和分析、确定适当的数据库、适当的结局定义、通过匹配和调整过程考虑潜在混杂因素最小化偏倚，并进行敏感性分析以测试结果的稳健性。当满足这些条件时，观察性数据库研究可以达到足够的证据水平，以帮助制定指南（即临床和监管决策）。
