Off-label drug use in neonates and infants in Spain: A five-year observational study

neonates and infants up to 1 year in Spain. Also, to analyse the off-label prescription of

medicines under current practice in this age group according to different evidence

sources.

Study design: A five-year (2015–2019) exploratory observational study about off-label

prescription in neonates and infants (0 to 1 year) at primary health care in Spain. All drug

prescriptions in this age group were analysed and classified according to their labelling in

off-label or on-label. The drugs prescribed off-label were subsequently reviewed in

national formularies and other databases to assess its evidence of use beyond what is

recommended in the Summary of Product Characteristics (SmPC).

Results: On average 34.50% of total prescriptions were prescribed off-label

according to the SmPC. 17.93% of total prescriptions in neonates and infants up to

1 year old were not based on clinical evidence from SmPC, Pediamécum, BNF or

DailyMed. In more than 88% of cases, off-label use was related to the posology

section of the SmPC, followed by the therapeutic indications and contraindications

sections, in 35.20% and 24.10% of cases, respectively. Almost 13% of off-label drugs

were over-the-counter. Salbutamol followed by topical tobramycin and colecalciferol

were the drugs most prescribed off-label.

Conclusions: Off-label use of drugs remains as an important public health concern, especially

for neonates and infants up to 1 year, who receive the greatest proportion of offlabel

prescriptions. The evidence-based off-label prescription is a widespread practice

that has shown a stable trend during the 5-year study period providing also a certain

extent of flexibility to paediatricians in some therapeutic decisions.