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State of paediatric medicines in the European Union
  1. Stephanie Kohl
  1. Policy & Advocacy, European Association of Hospital Pharmacists, 1200 Brussels, Belgium
  1. Correspondence to Stephanie Kohl, European Association of Hospital Pharmacists, Boulevard Brand Whitlock 87, 1200 Brussels, Belgium; Stephanie.Kohl{at}

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State of Paediatric Medicines in the EU

At the end of October, the European Commission presented a report to the European Parliament and the Council on the progress made in children’s medicines since the entry into force of the Paediatric Regulation. The report highlights the positive advances in paediatric medicine development which, thanks to the Paediatric Regulation, has become an integral part of the overall development of medicines in the European Union (EU).

Since 2007, the Paediatric Regulation aims at encouraging and enabling high-quality research into the development of medicines for children, ensuring over time that most medicines used by children are specifically authorised for such use with age-appropriate forms and formulations and increasing the availability of high-quality information about medicines used by children. By setting up a system of obligations, rewards and incentives, the Regulation tries to encourage manufacturers to research and develop medicines for children’s specific therapeutic needs.

The report on the State of Paediatric Medicines in the EU showed an increase of research into paediatric medicines and that the Regulation works best in areas where the needs of adult and paediatric patients overlap. In relation to the paediatric investigation plans (PIPs)—development plans aimed at ensuring that the necessary data are obtained through studies in children to support the authorisation of a medicine for children—it was noted that the number of agreed PIPs surpassed 1000 in 2017. PIPs were mostly completed in relation to immunology/rheumatology (14%), infectious diseases (14%), cardiovascular diseases and vaccines (each 10%), while oncology (7%) was found on the lower end of the agreed paediatric investigation plans.

Despite a lot of positive developments, the report also acknowledged that the concept of a paediatric use marketing authorisation (PUMA), which aims at stimulating research in existing compounds that are off-patent and/or helping transform known off-label use into authorised use, has failed to deliver. To …

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  • Competing interests None declared.

  • Provenance and peer review Commissioned; internally peer reviewed.