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Contemp Clin Trials. 2022 Oct;121:106890. doi: 10.1016/j.cct.2022.106890. Epub 2022 Aug 24.
The Food and Drug Administration (FDA) has stressed the need to ensure that clinical trial study populations accurately reflect the patients likely to use the product, if approved. However, the FDA has not provided specific guidance on how clinically relevant demographic characteristics might be defined. Therefore, the present study was designed to develop a framework that could be used to rapidly identify population demographics for any medical condition. Then, these real-world data were used as the basis to calculate acceptable demographic parameters (with 95% confidence intervals) for clinical trial populations. Data on Alzheimer's Disease were used as an example of the proposed approach.
美国食品和药物管理局（FDA）强调，需要确保临床试验研究人群如果获得批准，能够准确反映可能使用该产品的患者。然而，FDA 尚未就如何定义与临床相关的人口统计学特征提供具体指导。因此，本研究旨在制定一个框架，可用于快速确定任何医疗条件的人群人口统计学特征。然后，将这些真实世界的数据用作计算临床试验人群可接受的人口统计学参数（95%置信区间）的基础。以阿尔茨海默病的数据为例说明了所提出的方法。
