Data considerations in ischemic stroke trials

Data drive the analyses of any ischemic stroke trial, culminating in the main results and potential next steps. The distinct purpose of a given trial, advancing a novel treatment or examining routine clinical practice, determines the nature of essential data elements. Information gathering for an effective trial depends on ample data, adequate infrastructure, and properly planned statistical analyses. This review highlights the fact that successful future trials will require appropriate expertise that extends far beyond these basic considerations in order to move from identification of basic risk factors that are associated with outcomes to knowledge of pathophysiology and causation of outcomes. Efficient and productive data collection by local and central sites must be complemented by expert core lab adjudications. Source data archiving, including complete DICOM imaging datasets or biological specimens, are needed to maximize the potential for study interpretation and financial investment. Standard terminology, such as common data elements and definitions, enhance study comparisons. Screening logs attest to generalizability of a study. Real-time data transmission and core lab evaluation will be critical to guide adaptive trial design. Despite the overwhelming focus on the intervention in a particular treatment trial, individual pathophysiology must be considered. Understanding individual subject characteristics is a tenet of the coming era of precision stroke care, where the course of a given patient and eventual outcome is paramount. This will require a new approach to data collection in clinical trials.