This systematic review identified 29 studies published in 2016 using real-world evidence from observational studies to examine pediatric medication safety or effectiveness.

Studies varied in population, age groups, diseases or conditions, medications, study designs, data source, and methodologic rigor, and most relied on research-driven data collection rather than leveraging electronic health records or administrative claims databases.

Real-world evidence has not been fully applied to questions of pediatric medication safety and effectiveness.