Public consultation on the EU Paediatric Regulation

The European Commission (EC) is consulting on the Paediatric Regulation seeking feedback from stakeholders and interested parties. The information and views received will help the Commission prepare its second report on the Regulation, assessing the impact of the Paediatric Regulation on public health and the pharmaceutical industry. The final report is expected sometime in 2017.

The EU Paediatric Regulation legislation came into effect almost ten years ago on 26 January 2007. Its objective is to improve the health of children in Europe by enabling the development and availability of medicines for children aged 0 to 17 years. Key objectives of the Regulation are to ensure that medicines for children are of high quality, ethically researched and appropriately authorised and to improve the availability of information on the use of medicines for children. Achievement of these aims should not subject children to unnecessary clinical trials or delaying the authorisation of medicines for use in adults.

When it became law, the Regulation significantly changed the regulatory landscape for paediatric medicines in Europe. One of its early impacts was the establishment of the Paediatric Committee (PDCO), which is responsible for coordinating the EMA’s work on medicines for children. The PDCO’s main role is to determine the studies that companies must carry out on children as part of paediatric investigation plans (PIPs).

This EC consultation is based on an EMA/PDCO report on experience with implementing the Paediatric Regulation. The report showed that there has been an improvement in paediatric medicine development in Europe over the past ten years but it also highlighted challenges to be addressed. One such challenge will be the low uptake of paediatric-use marketing authorisations (PUMAs) by companies.

So, what improvements have there been?

There are now more medicines authorised for use in children. From 2004 to 2006, prior to introduction of the Regulation, 31 new medicines and new indications were authorised for paediatric use via the centralised procedure. From 2012 to 2014, 68 new medicines and new indications were centrally approved.

Considering children’s needs is now an integral part of medicine development. Somewhat cynically, one might say this is at least in part due to the fact that new marketing authorisation applications (MAA) must have an approved PIP, or PIP deferral or waiver but the evidence is that for whatever reasons, the Regulation is working. By the end of 2015, the PDCO had adopted 860 opinions for PIPs. Clinical trials in children initiated as part of an agreed PIP now represent about a third of paediatric trials recorded in the EU Clinical Trials database

Without a PIP or a PIP deferral or waiver, an EU MAA will not pass validation so if you require some support with one of these documents, your paediatric regulatory strategy or a PUMA, please contact one of the Regulatory team at Regulis.

The EC consultation is open until 20 February 2017. Click here to access the consultation page.