Off-label prescribing in children: the policy problem that won’t go away
Doctors routinely give children medicines that were never licensed for them. It is often the best option available, and that is precisely the problem.
In brief
Off-label prescribing is routine in paediatrics, especially neonatal care. Why it happens, the risks for children and what policy can do to close the gap.
A medicine used off-label is prescribed outside the terms of its licence: for a different age group, condition, dose or route. In adult medicine this is relatively unusual. In paediatrics it is routine.
How common is it?
Studies across Europe and North America have consistently found that a substantial share of prescriptions in children’s hospitals are off-label or unlicensed. The proportion rises sharply in neonatal intensive care, where many of the medicines given to premature babies have never been formally studied in them.
Two decades of paediatric legislation have improved labels for many products, but the backlog of older medicines without paediatric data is large, and older off-patent drugs offer little commercial reason to go back and study them.
Why clinicians do it
Off-label prescribing is not reckless. It is usually guided by clinical experience, published studies and reference works such as the BNF for Children in the UK. For many conditions there is simply no licensed alternative, and withholding a well-understood medicine because its label lacks paediatric data would harm the child.
Why it still matters
Without formal studies, doses can be extrapolated from adults in ways that miss important differences in how children absorb, distribute and clear drugs. Formulations may not suit small children, leading to improvised preparations. Rare adverse effects specific to children can go undetected.
There are also legal and practical consequences. Clinicians carry more personal responsibility for off-label prescribing, and parents may not be told that a medicine is being used outside its licence.
Where policy can help
Incentives for studying off-patent medicines have had limited uptake. Publicly funded research networks, priority lists of medicines where paediatric data are most needed, and better use of real-world data are seen as the more realistic routes to shrinking the problem.
