The use of US Food and Drug Administration (FDA)-approved drugs for the treatment of an unapproved indication or in an unapproved age group, or at doses or route of administration not indicated on the label is known as off-label use. Off-label use may be beneficial in circumstances when the standard-of-care treatment has failed, and/or no other FDA-approved medications are available for a particular condition. In pediatric patients, off-label use may increase the risk of adverse events as pharmacokinetic and pharmacodynamic data are limited in children. Approximately 73% of off-label drugs currently prescribed for various conditions do not have sufficient scientific evidence for safety and efficacy. For example, β-blockers are a class of drugs with FDA-approval for very few indications in pediatrics but are commonly used for various off-label indications. Interestingly, the proportion of off-label use of β-blockers in adults is at about 52% (66.2 million) of the total number of β-blockers prescribed. The frequency of off-label use of β-blockers in children is also high with limited data on the indications as well as safety and efficacy. We present trends in off-label use of β-blockers in children to discuss drug safety and efficacy and include recommendations for pediatric providers.
Keywords: off-label use; pediatrics; pharmacodynamics; pharmacokinetics; pharmacotherapeutics; β-blockers.
© 2019 Japan Pediatric Society.