Neonatal drug trials: impact of EU and US paediatric regulations

Abstract
The rational use of medicines in neonates is limited by the lack of scientific evidence for their use, as most medicines used in neonates are either unlicensed or off-label.1 In order to improve this situation, legislation has been passed in Europe and the USA to encourage the pharmaceutical industry to study medicines in the paediatric and neonatal populations. In order to evaluate the impact of these paediatric regulations in the neonatal population, we analysed the drug trials registered in the Clinicaltrials.gov database. Among all (138 948) trials registered, 30 912 (22%) were paediatric trials, and only 288 (0.2%) involved neonates. The number of trials registered grew steadily over time. From 1999 to 2012 trials …