Whether guided by economics or an instinct to shield children from potential harm, the way we bring new pediatric drugs to market does not always serve the best interests of children with life-threatening illness for whom few options exist. As a pediatric oncologist for 40 years, I have witnessed a revolution in cancer treatment, launched by early advances in treating children that later also benefited adults. Today, however, children with intractable disease are too often left behind and denied timely access to breakthroughs in cancer treatment that could save their lives. This must change.
Current practice has children awaiting completion of Phase I safety trials in adults, if not considerably longer, before pediatric trials open. But children are not small adults. They tolerate and respond to medical interventions differently from adults. Moreover, tomorrow’s pediatric drug may prove useful for treating a far more common adult cancer whose connection to the drug seems far-fetched today.