Off-label use and evidence of efficacy and safety for drugs in a tertiary-care neonatal intensive care unit

Article Outline

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In this issue of The Journal, Kumar et al report reassuring, as well as concerning findings from a Chicago neonatal intensive care unit (NICU). Of parenteral medications administered to NICU patients over a 3-year period, 45% were used off-label (i.e., not approved by the U.S. Food and Drug Administration for neonates). However, for more than 90% of parenteral medications used off-label, there was “Level 1” evidence of efficacy and safety in neonates (i.e., at least one randomized clinical trial regardless of size or the heterogeneity of patients).

The authors and other neonatologists advocate for more robust, as well as longer outcome studies of medications used in this very vulnerable population.