EFPIA Welcomes Paediatric Medicines Regulation Report and Reiterates its Commitment to Progress in this Area

EFPIA welcomes the publication of the European Commission’s Report on the State of Paediatric Medicines in the EU and its acknowledgement of the significant progress achieved since the entry into force of the EU Paediatric Regulation.

Entitled: “10 years of the EU Paediatric Regulation,” the report furthermore recognises correctly the continuous commitment made by the pharmaceutical industry to develop medicines suitable for use by children – indeed, over 260 new medicines have been authorised for use in children since 2007.

EFPIA agrees with the findings of the Report that the Paediatric Regulation has been instrumental in creating a balanced legal framework that has succeeded overall in stimulating more clinical trials in children and has led to significant progress in paediatric R&D. This has been to the benefit of both young patients and society as a whole. Furthermore, this progress is continuing.

Nevertheless, it remains clear that serious unmet medical needs remain in the field of childhood diseases, including rare conditions in paediatric oncology. EFPIA member companies continue to work on addressing many of these unmet needs. This is illustrated most effectively by the significant number of Paediatric Investigation Plans (PIPs) that have already been agreed across a wide range of therapeutic areas with the European Medicines Agency (EMA). These are ongoing at present and will deliver additional positive results for children in the coming years.  

As a result of the long development cycle of new paediatric medicines, the full impact of the Regulation on the introduction of new treatments is not yet fully apparent.  However, the report also concedes that some of the availability issues identified, relating for example to market access procedures, are outside of the remit of the Regulation.

The Report recommends a further, joined evaluation of the Paediatric and Orphan Regulations, aimed at driving medicine development more effectively in sub-populations of particular need. EFPIA notes that, while these two Regulations have different purposes, it will be interesting to better understand and compare their impact on the development of treatments for rare paediatric diseases.  EFPIA looks forward to contributing to this analysis.

As noted by the Report, more progress can be made in stimulating paediatric medicines development, within the current framework. EFPIA believes that the implementation of the Paediatric Regulation can be further improved through a number of pragmatic measures, building on all stakeholders’ experience and the lessons learned.

EFPIA remains ready and willing to work with all stakeholders on pragmatic and concrete proposals that could achieve this important healthcare goal.