Objectives: To describe the frequency of off-label drug use in 2014 as defined by the Food and Drug Administration (FDA)-approved age ranges in patients ≤18 years of age, to determine the rate of off-label drug use in 2014 by drug classification, and to compare current off-label medication usage rates with historical rates.
Methods: This is a retrospective cohort study of an administrative database containing inpatient resource use data from January 1, 2014, to December 31, 2014. Patients ≤18 years of age receiving 1 of 76 selected commonly prescribed medications are included. Off-label drug use is defined as use in a patient younger than the lower limit of the FDA-approved age range for any indication or dosage form of that drug.
Results: At least 1 drug was prescribed off label in 779 270 of 2 773 770 (28.1%) patient visits during the study period. Younger age, longer hospital stays, and mortality were associated with higher rates of off-label medication prescription. Off-label usage of certain medications differed between care settings. Rates of off-label medication use were higher in observational (45.5%), inpatient (53.9%), and ambulatory (54.2%) settings.
Conclusions: Although off-label drug use at major US pediatric hospitals is declining, 1 out of every 4 medications is not in accordance with FDA label indications for patient age. There exists substantial variation in off-label drug use among drug categories and encounter types. Although many commonly prescribed medications are FDA-approved for use in subpopulations of pediatric patients, studies of their safety, efficacy, pharmacokinetics, and optimal dosing are ongoing.
Copyright © 2019 by the American Academy of Pediatrics.