A literature review on off-label drug use in children


A literature review on off-label drug use in children


Pandolfini C; Bonati M


European Journal Of Pediatrics




Child; Humans; infant; Adult; Age Factors; Hospitals; Prescriptions; adolescent; Preschool; infant; Newborn; Clodronate; Community Health Services/statistics & numerical data; Drug Labeling/statistics & numerical data; Drug/statistics & numerical data; Legislation; Pediatric/statistics & numerical data


The aim was to compare results of studies performed in different settings worldwide and identify common therapeutic areas to allow for focused interventions, because off-label drug use can be a measure of the lack of knowledge concerning paediatric treatments. A secondary objective was to provide a brief review of efforts to date. A literature review of articles on off-label and unlicensed drug use in children involving general prescription samples was performed using Medline and Embase. In all, 30 studies from 1985-2004 were included. Eleven involved paediatric hospital wards, seven neonatal hospital wards, and 12 the community setting. The off-label and unlicensed classification methods varied, making results difficult to compare. In general, off-label/unlicensed prescription rates ranged from 11%-80%, and higher rates were found in younger versus older patients and in the hospital versus community settings. On the paediatric hospital wards, off-label/unlicensed prescriptions ranged from 16%-62% and most often concerned acetaminophen, cisapride, chloral hydrate, and salbutamol. In the neonatal wards, rates ranged from 55%-80% and often involved caffeine. In the community setting, rates ranged from 11%-37% and the most commonly implicated drugs were salbutamol and amoxicillin. Conclusion:A lack of harmonization between the evidence, the information available to doctors, and its use in clinical practice exists and this is part of the reason off-label therapies are so common. Attempts have been made to improve knowledge concerning paediatric treatments, but more focused interventions are needed, also taking into consideration this lack of harmonization.


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Pandolfini C; Bonati M, “A literature review on off-label drug use in children,” Pediatric Palliative Care Library, accessed October 2, 2023, https://pedpalascnetlibrary.omeka.net/items/show/13784.