The Paediatric Regulation – a work in progress?

The Council’s report on the ethical issues raised by children’s involvement in clinical research was published in May 2015. The report draws on the contributions of over 500 children and young people, parents and professionals, and its recommendations are made accessible in both a magazine format and as an animation. 9 months on from its publication, this post focusses on progress with one of the key recommendations - a review of the European Medicines Agency class waiver system in order to promote research into new medicines for children.

In drawing up this report, the Working Party tackled not only the thorny issue of how children and young people can ethically be recruited to research, but also the more hidden ethical challenges that arise in connection with the ways in which research is prioritised and developed in the first place.

We started from the premise that ‘good’ research (a shorthand for ethically robust and well-designed research that asks questions that are important for the health of children) should be seen as intrinsically good, and as a natural and necessary part of any healthcare system. Building on this point, we made the strong claim that it is not an ethically neutral act to say ‘no’ to research proposals that meet these requirements and might potentially lead to better health care for children. There is similarly a strong ethical imperative actively to promote such research – and the European Paediatric Regulation, which mandates the inclusion of children and young people in clinical trials, unless a waiver has been granted, has a critical role to play in this arena.

The working party felt that the European Medicines Agency and the Paediatric Committee (the European institutions responsible for implementing the Regulation) should be commended for the very positive and proactive approach they have taken to their regulatory role. They have used it not only simply to police the system established by the Regulation, but have also sought actively to promote effective, collaborative, research with children and young people through a variety of practical means.

Nevertheless, much more can and should still be done – and our report picked out in particular the class waiver system, whereby medicines targeting ‘adult-only’ conditions have been exempt from the requirement to include children and young people in trials. We argued that this system was clearly not working as originally intended and strongly urged the Paediatric Committee (PDCO) to complete its promised review of the class waiver system as quickly as possible.

In particular, we argued that PDCO should ensure that where the ‘mechanism of action’ of a medicine is potentially relevant to children (for example for a different illness from that targeted in adults), research with children should go ahead. This recommendation has been echoed by others calling for research to improve healthcare for children and young people, such as the Unite2Cure campaign.

We therefore regarded PDCO’s announcement in July 2015 that they had revised the class waiver list, revoking eight such waivers, and updating fifteen more, as a “welcome step towards further promoting research with children”. This means that there are now fewer conditions where companies developing new medicines are automatically exempted from any requirement to develop a ‘paediatric implementation plan’ (PIP) setting out how they will involve children and young people in the study. However, under the Regulation, they are still potentially entitled to apply to PDCO on a case-by-case basis, and apply for a product-specific waiver, on the grounds that the condition being targeted by the trial medication does not arise in children. Thus, while PDCO will now be directly involved in discussing such cases with companies, they still cannot actually require them to produce a PIP – unless and until the Regulation is changed to give PDCO these powers.

The ‘ten-year review’ of the 2006 Regulation is coming up soon, offering a perfect opportunity to review the thinking behind the waiver system, and to ensure that PDCO has the powers it needs. We have written to the European Commission to urge the ethical imperative of such a change. In the meantime we urge all sponsors of research to consider the potential benefits of their research to children’s health – and where appropriate to include children and young people in trials on a voluntary basis.

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