The current drug safety landscape inadequately serves the millions of
children prescribed medications every year. Randomized clinical trials
are limited in detecting pediatric adverse drug effects due to low
participant enrollment, complicated study design, and short trial
duration. While the biology of human development and the pathology of
childhood diseases are well studied research topics, current
pharmacoepidemiology approaches do not use this knowledge when
investigating drug safety in children. Here, we describe how adverse
drug effects manifest as children develop from birth through the teenage
years. We discuss the benefits of an empirical approach for evaluating
clinical relevance and biological causality of identified adverse drug
effects in children. We argue that a data-driven strategy that leverages
observational data and biomedical knowledge is an ethical and effective
methodology to improve pediatric drug safety.