Beyond the Label: Steering the Focus Toward Safe and Effective Prescribing

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...