Each year, hundreds of millions of prescription medications are dispensed to pediatric patients.1 A significant proportion of prescriptions are used in an off-label manner, outside the specifications approved by the US Food and Drug Administration (FDA), rendering off-label prescribing a “public health issue for infants, children and adolescents,” as described by the Committee on Drugs for the American Academy of Pediatrics.2 The committee also explicitly states that off-label use “does not imply an improper, illegal, contraindicated or investigational use.” Yet, when used in research, clinical practice, or even the lay media, the term off-label commonly carries a negative connotation. This interpretation probably reflects the sense of uncertainty in understanding the risk–benefit balance of a medication without FDA review and approval. However, prescribing according to the package insert does not necessarily translate to the safe and effective use of a medication.3 The clinical trial data required for FDA...

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