Pediatric policy and regulation
Over the past two decades, policymakers in the US and Europe have passed legislation and regulations specifically aimed at promoting the development of pediatric drugs and devices and to increase the information available on their use. Comprehensive study of the implementation and impact of these policies enables us to define their specific strengths and limitations and to inform our proposals for policy reforms.
Utilization and health outcomes
The Initiative leverages its expertise in pediatric pharmacoepidemiology to analyze medication utilization patterns and treatment outcomes in large datasets of pediatric patients. These types of studies are an essential tool for defining the effectiveness of drugs and devices in real-world settings. It also supports the study of inequities and variations in medication use, non-evidence-based or harmful medication practices, and adverse events associated with new treatments.
Clinical trial methods and ethics
Pediatric trials face numerous ethical, social, and practical challenges, including concerns around exposing children to risk, low prevalence of many childhood diseases, and inadequate reporting of pediatric trial results. This has led to underrepresentation of pediatric patients in clinical trials and widespread off-label use of many medicines studied only in adults. Patient-centric approaches are needed to design and prioritize pediatric drug trials that are ethical and safe, and reflect areas of greatest clinical need.