Division of Urology, Rhode Island Hospital and the Miriam Hospital, Providence, Rhode Island; Warren Alpert Medical School, Providence, Rhode Island.
Division of Urology, Rhode Island Hospital and the Miriam Hospital, Providence, Rhode Island; Warren Alpert Medical School, Providence, Rhode Island.
Fertil Steril. 2018 Jun;109(6):946-951. doi: 10.1016/j.fertnstert.2018.04.005.
Randomized clinical trials are considered the preferred approach for comparing the effects of treatments, yet data from high-quality clinical trials are often unavailable and many clinical decisions are made on the basis of evidence from observational studies. Using clinical examples about the management of infertility, we discuss how we can use observational data from large and information-rich health-care databases combined with modern epidemiological and statistical methods to learn about the effects of interventions when clinical trial evidence is unavailable or not applicable to the clinically relevant target population. When trial evidence is unavailable, we can conduct observational analyses emulating the hypothetical pragmatic target trials that would address the clinical questions of interest. When trial evidence is available but not applicable to the clinically relevant target population, we can transport inferences from trial participants to the target population using the trial data and a sample of observational data from the target population. Clinical trial emulations and transportability analyses can be coupled with methods for examining heterogeneity of treatment effects, providing a path toward personalized medicine.
随机临床试验被认为是比较治疗效果的首选方法，但高质量的临床试验数据往往不可用，许多临床决策是基于观察性研究的证据做出的。我们将使用关于不孕症管理的临床示例，讨论如何使用来自大型、信息丰富的医疗保健数据库的观察性数据，并结合现代流行病学和统计方法，在缺乏临床试验证据或该证据不适用于临床相关目标人群的情况下，了解干预措施的效果。当没有试验证据时，我们可以进行观察性分析，模拟可能会解决感兴趣的临床问题的假想实用目标试验。当有试验证据但不适用于临床相关目标人群时，我们可以使用试验数据和目标人群的观察性数据样本，从试验参与者向目标人群推断。临床试验模拟和可转移性分析可以与检查治疗效果异质性的方法相结合，为个性化医疗提供途径。
