The Utilization of Externally Sourced Data and Data Collected and Analyzed from Previously Completed Studies Using Modelling and Simulation and Other Methodologies to Support Pediatric-Focused Regulatory Commitments.

There can be many challenges to conducting and completing clinical research trials, which may impact the successful and timely achievement of regulatory commitments. General difficulties can include a rare disease under study which limits the available study population, a relatively low number of interested study sites that have potential study participants, numerous required study procedures, invasive study procedures and, with regard to pediatric clinical trials, parents may be unwilling to provide consent for their children to participate. The utilization of externally sourced data as well as data collected and analyzed from internal previously completed clinical trials can be a useful and strategic method of supplementing clinical trial data that is needed to meet regulatory requirements. However, this strategy may not be widely known, understood or leveraged. The authors conducted a review of clinical trials and other types of regulatory commitments that successfully included external data and data that was collected and analyzed from previously completed studies, to meet regulatory requirements. It was concluded that the use of externally sourced data and data from other internally sponsored completed studies can offer a critical source of information, thereby adding robustness to regulatory submissions, particularly when there are gaps in clinical trials due to recruitment barriers and other challenges.