In October 2017, the European Commission presented to the European Parliament and the Council a comprehensive report on progress made in children’s medicines 10 year after the Paediatric Regulation came into force. The Paediatric Regulation, adopted in 2006, aims to address a gap in knowledge on how medicines should best be used by children. The scope is to reduce the level of off-label use and increase the number of medicines specifically developed and tested for children. In 2013, the Commission published a first report on the Paediatric Regulation and, although it revealed some promising signs of progress, it found that, due to the length of medicinal product’s development, it would take at least 10 years to gain a full understanding of the situation. Finally, in 2017 the Commission released the second report that assessed the Regulation’s impact on public health and business. One of the first progress described in the report is the increase of the number of new authorized medicines for children. In 2007-2016 over 260 new medicines for use by children (new marketing authorizations and new indications) were authorized most of them linked to the Regulation’s requirements. Furthermore, the number of agreed Paediatric Investigation Plans (PIPs) surpassed 1000 in 2017, of which 131 were completed at the end of 2016. On the other hand, the analysis performed by the Commission, underlined that there is a category of paediatric-only developments, which the Regulation tries to generate specific interest in, but has so far failed. It introduced, in fact, the concept of a Paediatric Use Marketing Authorization (PUMA) to stimulate research in existing compounds that are off patent. PUMA tool should also help to transform known off-label use into authorized use that is safer and better framed through the marketing authorization. To date, only three PUMAs have been granted, revealing a result being clearly below expected levels. Another relevant result achieved after the Regulation adoption, concerns the increase of the number of clinical trials carried out in children prior to the authorization of medicines. The proportion of clinical trials in the European clinical trial database EudraCT that include children has increased by 50 % in 2007-2016, from 8.25 % to 12.4 %. Moreover, research with previously neglected paediatric subpopulations has risen considerably. Prior to the Regulation, research with neonates was almost nonexistent in medicine development. Overall, this analysis demonstrated that The Paediatric Regulation had a considerable impact on the development of paediatric medicines in the EU. It ensured that paediatric medicine development became an integral part of the overall development of medicines.
