Off-Label and Unlicensed Drug Use in Neonatology: Reality in a Portuguese University Hospital

Joana Silva, Filipa Flor-de-Lima, Henrique Soares, Hercília Guimarães


Introduction: This study aims to characterize the drugs used in a Portuguese Neonatal Intensive Unit Care, assess the rate of off-label or unlicensed drugs use according to the information available in the Summary of Product Characteristics and compare results between preterm and full-term neonates.
Material and Methods: A 6-month period retrospective cross-sectional study was conducted in our Neonatal Intensive Unit Care in 2013. Prescribed drugs data were recorded and compared with the paediatric information contained in drugs Summary of Product Characteristics.
Results: We analyzed 1011 prescriptions of 84 active substances, made in 218 admissions. In 42.9% of the cases, medicines were used according to Summary of Product Characteristics information; 27.9% of drugs were approved for neonatal period but used in an off-label manner; off-label drugs for neonates were used in 10.1%, whereas those with undetermined approval state and contraindicated were used
6.0% and 8.7% of the cases, respectively. Unlicensed prescriptions accounted for 4.4% of total. Preterm received a higher rate of drugs used according to Summary of Product Characteristics (p < 0.0001), whereas full-term received more off-label drugs for dose/frequency (p < 0.0001) and contra-indicated for neonates (p < 0.012).
Discussion: Preterm neonates received a higher median number of drugs, since they stayed longer in the unit. The main reason for off-label prescribing was the use of doses/frequencies of administration different from those stated in the Summary of Product Characteristics, suggesting that updating these documents is necessary. Manipulation of medicines is one of the causes for unlicensed drugs use, emphasizing the lack of appropriate formulations for neonatal age.
Conclusion: Progresses have been made to reduce the risks of off-label/unlicensed prescriptions, but competent authorities must continue their efforts to develop safer and more effective drugs for neonatal period.


Drug Labeling; Drug Therapy; Infant, Newborn; Intensive Care Units, Neonatal; Off-Label Use; Portugal.

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