Pregnant women and their neonates are perhaps the last true therapeutic orphans. The majority of pregnant women take at least one medication to treat a maternal condition but very few pharmacokinetic, pharmacodynamic or clinical trials are currently conducted during pregnancy. As a consequence, evidence to guide effective drug treatment of pregnant women is largely lacking. Off-label use of drugs in neonates is a worldwide issue and of paramount concern. Neonatologists are primarily dependent on clinical experience and data extrapolation from adults, children, and infants to guide optimal drug choice, and to select the most safe and effective dosing regimen. Unfortunately, this has resulted in considerable differences in prescribing practices both between and within countries, for many frequently used drugs in neonates. There is a moral obligation to adopt pregnant women and their neonates into the mainstream of health care research and practice.