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Paediatricians, neonatal/paediatric pharmacists, and chief executives of hospital trusts recently received a position statement on the use of unlicensed medicines, produced by the Joint Standing Committee on Medicines of the Royal College of Paediatrics and Child Health (RCPCH) and the Neonatal and Paediatric Pharmacy Group (NPPG).
The licensing of medicines
Before a pharmaceutical company can promote a drug, it must obtain a licence.1 Following the 1960s thalidomide disaster,2 “legislation was introduced to ensure that no new drug could be marketed until independent experts were agreed that it had been adequately tested and was safe”.2 The process differs between countries but the principles are that the company must show the safety, quality, and efficacy of the drug when given in the dose and for the disease and age group recommended in the Summary of Product Characteristics (SPC). Drugs are increasingly licensed on a European Union wide basis. In the UK, doctors can legally prescribe drugs without a licence (unlicensed, UL) or outside the terms of the licence (off label (OL)—for example, in a different dose, as a different formulation, or for a different disease or age group). Prescribing outside the licence is relatively common for hospitalised children.3-6 In a neonatal intensive care unit 90% of infants receive UL or OL drugs. In primary care, 11–33% of prescriptions for children are UL/OL.7 ,8
As paediatricians can still prescribe, is this a problem?
The current licensing arrangements ensure a rigorous assessment of most drugs used for adults. When a medicine is prescribed OL/UL, these safeguards are absent, extrapolation from adult data is necessary (despite the great biological differences between adults and children9 ,10 and between children of different ages11), and children may be given inadequate doses or exposed to unknown risks. There is also evidence which suggests that adverse drug reactions (ADRs) are more likely with UL/OL …