Children have been described as ‘therapeutic orphans’, indicating a lack of authorised, age appropriate formulations of medicines to treat a variety of illnesses. Access to appropriate medicines can be particularly problematic in resource poor countries. There is some evidence that adverse drug reactions are more likely when using unlicensed or ‘off label’ medicines for children. Adherence and medication error may also be issues. In the UK, a national formulary for children was developed by paediatricians and paediatric pharmacists through the ‘Medicines for children’ project, and the British National Formulary for Children is now distributed to provide evaluated information to all doctors and pharmacists in the UK. This is complemented by a series of leaflets about unlicensed and off label medicines aimed at carers and is freely available on the internet.

Several initiatives are in place to improve the ‘orphan’ situation, with the USA starting the process with legislation designed to increase our knowledge of paediatric medicines and to improve the availability of those suitably authorised. …