Defining demographic cohorts in clinical trial populations using large electronic health records databases

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.