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It's time to close the paediatric gap

Innovation has been slow to reach the youngest patients. Thankfully, that is changing

By Pierre Meulien, IMI Director

kids pills steth
Image by 9dream studio Shutterstock 


In late 2020, we held our annual IMI stakeholder event - online for the first time, thanks to COVID-19 restrictions - where we hosted a discussion on the topic of paediatric cancer. We chose this topic because it’s a challenge that best benefits from cross-sectoral collaboration, where different disciplines - imaging, digital tools, data analytics, diagnostics, and medicines - are forced out of their silos. This makes for a good case study for preparing the future post-IMI partnership. It also aligns with the European Commission’s ambitious cancer mission.

But more than that, we felt the need to talk about it because medical innovation has often been slow to trickle down to children and adolescents. A fraction of the number of anti-cancer medicines for kids are authorised compared to those for adults, for example, and half of new medicines are not tested in children. In the absence of reliable information that would come from clinical trials, paediatricians have traditionally administered medicines off-label; dosing is guesswork, with per-kilo values used as a marker.

But children are not simply small adults, as one panellist at our event remarked. They don’t metabolise drugs in the same way adults do. Legislative initiatives around the world that mandate testing of drugs intended for children have boosted the availability of paediatric medicines. However, it remains challenging, particularly in paediatric oncology, where too few drugs targeting paediatric cancers are currently under development.

Which way forward?

As we’ve learned, whenever something is imposed by the regulators, everyone looks for a way to navigate this new reality. Increasingly in Europe, the vehicle is IMI. Paediatricians of course, have been very supportive of legislative efforts to mandate clinical testing of drugs intended for children, but still nobody could agree on the how. Pharma risked doing clinical trials costing millions of euro only to be rebuffed by regulators who might conclude that there was not enough data or subjects involved. The private sector can’t do this without the public sector, academic experts in paediatrics, social scientists, and of course, regulators. What was needed was proper infrastructure where people could align. It took a decade, but it became clear that IMI could once again be that pre-competitive safe space.

And so that’s the background to ITCC-P4, the only IMI project focused on children’s cancer medicine. The project has embarked on a mammoth task to significantly improve and accelerate science-driven new oncology drug development for children by providing high-quality biological and preclinical evidence that a particular regime would be safe for use in children with cancer. It has proven extremely difficult to create animal models of children’s tumours because the tumours are so heterogeneous, manifesting so early in life that environmental factors are unlikely to be the cause. The consortium has recruited some of the best minds in the paediatric cancer field to tackle this problem, and their objective is to create a testing platform with 400 new preclinical models of high-risk paediatric solid tumours that can be used to carry out early drug testing. 


To further build a favourable paediatric clinical research environment, in 2018 the project c4c was launched, seeking to establish standards for the best way to carry out clinical trials that involve children. They’ve recruited advisory groups made up of young people with different diseases who, for example, have helped draw up child-friendly consent forms.

Other IMI projects are teaming up with c4c to make sure they align. They include EU-PEARL, which is working on the design of adaptive platform trials, and INNODIA, a diabetes project with clinical trials involving children. c4c are also collaborating with AIMS-2-TRIALS, a project on autism (which mostly rears its head in early infancy and for which early diagnosis can make a big difference) and other paediatric research efforts in the area of infectious diseases: PERISCOPE and RESCEU. CONCEPTION, meanwhile, is doing for pregnant and breastfeeding women what c4c is doing for kids; this is another group for whom clinical information on the safe use of medicines is urgently needed. 

Genetic newborn screening – a tricky new frontier

IMI is also moving into research that concerns genetic newborn screening for rare diseases. Again, though this topic has been under discussion for decades, it is fraught with ethical concerns. Diagnosing a newborn with one of thousands of diseases we can’t treat – only about 1 % of rare diseases have a specifically-approved treatment – raises an ethical dilemma. On one hand, knowing a newborn has de novo mutations that might lead to certain diseases would help our scientific understanding and speed up diagnosis (one of the well-known challenges facing rare disease patients is getting a diagnosis in the first place; it takes an average of eight years). On the other hand, where does that leave the child’s family?

Given the kind of infrastructure and that would be needed to build such a screening platform, and given the challenges in terms of acceptance and safety, ethics, policy, rights and the law that it would surface, a public-private partnership seems to be the best option. Reaching any kind of consensus on such a topic will require a lot of cooperation with partners inside and outside of the IMI world. Nobody can do it alone, and it’s crucial that we get it right.


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