US Perspective on Off‐label Use in Pediatrics

Abstract

JPGN Volume 56, N T he evaluation of off-label use published by Ruiz-Antoran et al (1) in this issue of the Journal of Pediatric Gastroenterology and Nutrition provides an opportunity to discuss evolving US legislative measures to improve available clinical trial–derived pediatric data and provide coherent labeling for pediatric providers in dosing of drugs for children. Dr Ruiz-Antoran et al present findings of frequent off-label use of drugs in a Spanish outpatient pediatric gastroenterology clinic, which appear generally consistent with the use of off-label drugs in the United States (1–4). There are multiple reasons why providers may prescribe drugs off-label. Providers may not be aware that they are prescribing off-label, or may intentionally choose off-label treatments that are supported by widely accepted recommendations or treatment guidelines (1). Providers may also prescribe drugs off-label because other on-label treatments have been ineffective, or because there is an established effect from that class of medications (5). Also, off-label prescribing may be necessary to provide a product in a pediatric-appropriate strength and formulation (6). The potential negative effect of off-label prescribing in pediatric patients has been evaluated, but the direct relation between off-label prescribing and adverse events has not been definitively established, as noted by Ruiz-Antoran et al (1,7). The potential for adverse reactions associated with off-label drug use exists and steps, such as regulation, clinical practice guidelines, and local initiatives, to decrease adverse reactions associated with off-label use of products are described (1). Given the breadth of potential reasons for off-label prescribing, no one approach appears likely to address all the reasons providers may choose off-label prescribing (2,5,8). In the United States, legislation designed to encourage the appropriate use of drugs in pediatric patients may decrease the frequency of adverse