Success of Paediatric Regulation inspires us to go further
21.09.22
15 years ago, the EU Paediatric Medicines Regulation was introduced because we had too few medicines for treating children, leaving the youngest patients in need. The Regulation sent a clear signal that action was needed in paediatric medicines development and introduced a framework of obligations and rewards to make it happen.
In the years since the introduction of the Regulation, more than 290 new paediatric medicines and indications for children have been approved in the EU [1]. Paediatric medicine development is now an integral part of the overall development of medicines[2], and the proportion of clinical trials that include children increased by 50% between 2007 and 2016. For example, new oncology treatments and trials are delivering tangible progress for young people with cancer.
It is important to recognise progress when we see it: children across Europe have benefited from a European policy and law designed to accelerate the development of paediatric medicines. Policymakers and stakeholders identified a pressing problem, designed a solution – and it has borne fruit.
Now, as the European Commission prepares to revise this successful Regulation, attention turns to the future. The review comes in the context of the implementation of the Pharmaceutical Strategy as part of the European Health Union and it presents an opportunity to think hard about how we can build on the progress delivered since 2007.
We should be bold. Inspired by past successes – and by the real unmet need that remains in the paediatric community – we must all prepare to do more to deliver for young patients.
Children at the centre
There are currently around 600 new paediatric medicines in development. This is due in large part to companies undertaking Paediatric Investigation Plans (PIPs) – research and development programmes for childhood medicines agreed with regulators. In acknowledging the success of the PIP approach, policymakers can stimulate further innovation by offering the right mix of scientifically well framed obligations and rewards.
So, what exactly do we want to improve?
The wider trend towards patient-centred medicines development must apply to children as well as adults. Any improved regulatory system for paediatric medicines should shift from the current ‘adult-centric’ approach to become more ‘child-centric’. To achieve this, it is vital to encourage ‘first-in-child’ innovation.
Introducing the concept of ‘first-in-child’ innovation will require rethinking the definition of what constitutes a disease or a condition. Thanks to advances in science, the underlying cause of many diseases, including several cancers, is now better understood. This allows treatments to be designed to target the cause of the disease.
The mechanism of action (MoA) of a medicine can make it suitable for use in more than one condition if each condition can be shown to be associated with the same cause. The definition of a disease or a condition should therefore include the underlying cause if this is supported by current scientific evidence.
EFPIA is proposing a framework grounded in robust science, for PIPs that can be based not on the adult indication, but on a paediatric need that the treatment can address based on its MoA. In practice, that would allow a therapy developed for an adult illness to be studied in a different childhood disease because both have the same cause.
This child-centric PIP approach would require significant efforts and present new scientific challenges. It will be critical to ensure that it is based on robust science and is framed in a way that ensures that such child-centric PIPs are scientifically and clinically meaningful, doable, and do not place undue burden on innovation,
Boosting innovation
Now is the time for all partners to work together to seize the opportunities arising from a once-in-a-generation rethink of how we can boost innovation in paediatric medicines in Europe.
To that end, I am pleased to be participating in a webinar organised by the European Society for Paediatric Oncology and the Association of European Cancer Leagues in cooperation with Childhood Cancer International – Europe. With input from MEPs, the European Commission, Member States, civil society and industry, it represents a positive step in our collective conversation on the future of paediatric medicines. (See the agenda and register here)
With the right policy measures, we can have a real and lasting impact on the lives of Europe’s youngest citizens.
Find out more here
Find out more here
