Off-Label Drug Use in Pediatric Populations
Summary
Off‐label drug use in children refers to the prescribing of medicines outside the terms of their licensed marketing authorisation, whether by age, dosage, indication or route. It arises from a chronic shortage of paediatric clinical trials and age‐appropriate formulations, compounded by ethical, logistical and economic obstacles to studying medicines in younger age groups. As a result, clinicians often rely on extrapolated adult data, small observational studies or established clinical practice to guide therapy. Pharmacokinetic and pharmacodynamic differences across developmental stages heighten uncertainty regarding efficacy and safety, posing a risk of under‐ or overdosing as well as adverse drug reactions. Despite regulatory initiatives in Europe, North America and elsewhere to stimulate paediatric research, off‐label prescribing remains widespread, especially in neonatal and critical care settings. Globally, off‐label use underlines the imperative for robust pharmacovigilance, dose-finding studies and innovative formulations to deliver evidence-based therapies tailored to children’s unique physiological needs.
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Off-Label Drug Use in Pediatric Populations publication trend
The graph below shows the total number of articles in off-label drug use in pediatric populations across all publications each year (not limited to Nature Index journals).
Technical terms
Off-label use: Prescribing a licensed medicine outside the conditions of its approved marketing authorisation, including patient age, indication, dosage or route of administration.
Unlicensed medicine: Any product that lacks formal marketing authorisation for paediatric use, often resorted to when no authorised alternative exists.
Adverse drug reaction (ADR): An unintended, harmful response to a medicine administered at normal dosages for prophylaxis, diagnosis or therapy.
Summary of Product Characteristics (SmPC): The document approved by regulatory agencies that describes a medicine’s authorised uses, dosing and safety information.
Neonatal intensive care unit (NICU): A specialised hospital ward providing comprehensive care for ill or premature newborn infants, where off-label prescribing is particularly prevalent.
References
- Off-Label Prescribing in Pediatric Population—Literature Review for 2012–2022. Pharmaceutics (2023).
- Adverse drug reactions and off-label and unlicensed medicines in children: a nested case?control study of inpatients in a pediatric hospital. BMC Medicine (2013).
- Use of off-label and unlicensed medicines in neonatal intensive care. PLOS ONE (2018).
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