Off-Label Medication use in Children, More Common than We Think: A Systematic Review of the Literature

J Okla State Med Assoc. 2018 Oct;111(8):776-783.


Content: Clinician prescribing of off-label medications is common due to a lack of pediatric-specific data regarding the dosing, efficacy and safety of medications regularly prescribed to children.

Objective: This systematic review summarizes the published incidence of off-label medication use in children from the past 10 years. We also performed a retrospective chart review to determine the incidence of off-label prescriptions for children seen in the OU Physicians clinics.

Data sources: We conducted a literature search of PubMed and OVID Medline from 2007 to 2017. Search terms included off-label use of medications and all child. For the local review, the outpatient electronic medical record (EMR) was queried.

Study selection: Studies were eligible for inclusion if the study included children < 18 years of age, defined off-label use in the paper, and included the incidence of off-label drug use.

Data extraction: Each review author extracted the study data from their assigned studies. For the retrospective chart review, the EMR was queried for patients <21 years of age who had a clinic visit and received a new prescription during 2017.

Results: We identified 31 studies, with off-label prescription rates from 3.2 % to 95%. The local retrospective chart review included 1,323 prescriptions; 504 were off-label (38.1%) and 819 were approved. The frequency of off-label prescriptions does not differ significantly between the meta-analysis from the systematic review and the local retrospective chart review (30.9% vs 38.1%).

Conclusions: The use of off-label medications in children remains a common practice for pediatric providers.