Making Progress for How Medicines Are Used in Children

We are writing in response to the article “Off-label Drug Use in Hospitalized Children” by Shah et al,¹ which describes the magnitude of off-label pediatric prescribing in the United States. We would like to applaud the efforts of the authors in tackling such an important topic, as off-label prescribing potentially puts our children at risk of lack of efficacy due to improper dosing as well as unexpected adverse events. We would, however, like to correct statements made by the authors regarding the role of the National Institutes of Health, specifically the National Institute of Child Health and Human Development (NICHD), in the implementation of the Best Pharmaceuticals for Children Act (BPCA). The authors incorrectly state that they “identified specific drugs that were commonly used off-label that did not appear on the FDA [Food and Drug Administration] list of medications requiring further study in children.” This list of drugs for which pediatric studies are needed is developed and published annually by the National Institutes of Health, in consultation with pediatric experts as well as colleagues from the FDA.