New European Union (EU) paediatric regulations to encourage more research on children’s medicines became law on 26 January 2007.¹ The main proposals are as follows. First, there will be incentives to encourage the pharmaceutical industry to test products for use in children. Secondly, the European Agency for the Evaluation of Medicinal Products (EMEA) will host an expert paediatric committee to oversee and assess paediatric investigation plans for developing medicines for children. Thirdly, a European clinical trials network will be set up to foster collaboration on paediatric studies throughout the EU. Fourthly, medicines licensed for paediatric use should be sold with a special label indicating that fact.

This legislation has the potential to have an enormous impact on how paediatric medicines, both old and new, are studied and used. Is this good news for Europe’s 140 million children?

THE DRIVERS FOR THE NEW EUROPEAN LEGISLATION

Evidence-based prescribing for children is compromised by lack of sufficient data on many drugs.^2–4 Although there is an elaborate system for testing drugs before they are used in adults, many drugs given to children have not been tested in this population at all, even though children can react differently to drugs.⁵ Moreover, it is often financially unrewarding to conduct research on medicines for children because the children’s market is smaller than that of adults.

If evidence for the safety, efficacy and acceptable risk/benefit of a drug exists, then that drug can be licensed for use as a medicine with indications, doses and side-effect warnings. However, prescribing can also be unlicensed (eg, the medicine is given as a liquid whereas the licence is for a tablet) or outside the terms of the licence (off-label; eg, a different dose for a different …