Concern has been raised by health professionals 1 2 and politicians3 regarding the lack of scientific data on the use of medicines in children. Such data are usually obtained from clinical trials by the pharmaceutical industry and used to obtain a product license, and these trials are almost invariably conducted entirely in adults. Studies throughout Europe have shown that health professionals are forced to use medicines that are either not licensed for use in children or used at a different dose, for a different indication, or by an alternative route from that recommended (“off label”).4 Two thirds of children in hospital4 and 90% of sick newborn infants5 receive medicines that are unlicensed or off label. Little information is available about the risk of such prescribing, but one study has suggested that there is an increased risk of toxicity.6

The pharmaceutical industry has been reluctant to study medicines in children for various reasons. These include the limited financial returns, the difficulty of organising clinical trials in children, and concerns about possible toxicity.7 …