Off-Label Prescribing in Children Remains High: A Call for Prioritized Research

Abstract

* Abbreviations:\n BPCA — : Best Pharmaceuticals for Children Act\n PREA — : Pediatric Research Equity Act\n\nThe US Food and Drug Administration requires that medications be tested for safety and efficacy at a specific dosage, and for a specific time period, before approval for clinical use in a particular population.1 Use of medications outside these parameters is considered “off-label” drug use.\n\nHistorically, studies evaluating medications in the pediatric population have been challenging. Children present unique considerations in clinical trials owing to scientific, ethical, clinical, and logistic concerns, which have previously limited and even discouraged the testing of medications in this population.1 Consequently, the majority of medications used in the care of children have historically been used off label without an adequate … \n\nAddress correspondence to Katelyn Yackey, MD, Department of Emergency Medicine, University of Kentucky, 800 Rose St, Room M53, Lexington, KY 40536. E-mail: katelyn.yackey{at}uky.edu