Unlicensed and Off-Label Medication Use in Pediatric and Neonatal Intensive Care Units: No Change Over a Decade

Many of the medications prescribed to children are off-label and/or unlicensed because pharmacologic evaluations have not been performed in this age group.

All drugs prescribed to patients admitted to the neonatal intensive care units (NICU) (n = 134) and pediatric intensive care units (PICU) (n = 56) during a 2-month observation period were recorded and classified according to patient age, drug license status, indicated use, and typical dosing, frequency and way of administration. Results were compared with prior data collected in 2002, from the same units.

In the NICU, among the 1064 prescriptions for 49 medications, 312 (29.2%) were licensed and 63 (5.9%) unlicensed, and 693 (64.8%) were off-label use. For the neonates, 23.9% and 96.3% received at least one unlicensed medication and one off-label medication, respectively. While the difference in off-label use between the two time periods was not statistically significant, unlicensed medications were less frequently prescribed in 2016 (5.9 versus 16.6%, p = 0.001). Regarding the PICU, among the 388 prescriptions for 75 medications, 205 (52%) were licensed and 13 (3.4%) unlicensed, and 170 (43.8%) were off-label. In contrast, in 2002, none of the medications prescribed were unlicensed (p = 0.001). The number of off-label medications (41%) and number of PICU patients receiving at least one unlicensed/off-label medication in these two time periods (88.7% versus 90.5% for 2016 and 2002, respectively) were similar.

The current study confirms the high prevalence of unlicensed and off-label drug use in a PICU and NICU setting. Compared with a similar study conducted in the same PICU in 2002, despite regulatory efforts conducted in this area, the prevalence of unlicensed medications was surprisingly higher.