It has long been known that 50–90% of medications used in children are “off-label” or unlicensed, and this is associated with an increased risk of adverse events 1–14. This means that some of the most vulnerable people in the European Union (EU), sick children, are being treated in a non-evidence-based, unregulated fashion. Even commonly prescribed respiratory medicines, such as short-acting β2-agonists and antipyretics, have not been adequately studied in young children 15. The risks of the present situation include: death or serious adverse events from overdosing; non-availability to children of important medications; under-dosing leading to loss of efficacy; and the use of poor-quality formulae. Side-effects that may not affect adults might be very important in children; the effects of inappropriately prescribed inhaled steroids on growth is well known 16, 17, but could the use of parenteral steroids affect alveolar development in the first 2 years of life 18, 19? Furthermore, there may be fundamental differences between adult and paediatric diseases; several studies have established that adult asthma is a chronic inflammatory disease, but many wheezing phenotypes in early childhood are not inflammatory and are thus poorly responsive to corticosteroids 20–29. Some welcome steps have been taken to address this, with the publication of the evidence-based Medicines for Children by the Royal College of Paediatrics and Child Health 30, and the first edition of a Paediatric British National Formulary 31 replacing the generic one. However, evidence-based therapeutics requires evidence and this has been lacking.
There are several likely reasons for this scandalous lack of evidence-based paediatric pharmacotherapy. Much paediatric disease improves over time (many wheezing phenotypes 32, for example), whereas adult disease is more often chronic, progressive and requires prescription medicines long term. Thus, there is …