International Childhood Cancer Day: why an EU law needs improving to help develop new treatments for young people

To mark International Childhood Cancer Day 2016, we’re launching a new campaign as part of our Cancer Research UK Kids and Teens fundraising initiative. This will help fund vital research into children’s cancers, and support our clinical trials looking to develop new treatments. But we’re concerned that an EU law could be putting potential progress in children’s cancer at risk, as we’ll explore below.

For children diagnosed with cancer today, the future looks a lot brighter than it did 40 years ago. Back in the 1970s, about a third of children diagnosed with cancer in the UK survived their disease for at least ten years. Now it’s jumped to around three-quarters.

But we can’t forget that this still means some 250 children will lose their lives to cancer each year, making it the biggest cause of death by disease in young children in the UK.

And it’s not just about survival. Treatments for children’s cancers can have side-effects that can last long into adult life. Although it’s good news that so many more children are surviving, this comes with the added challenge of finding kinder treatments and ways to manage these side effects.

So there’s more that should – and can – be done.

Our ambition is that all young people diagnosed with cancer will survive and go on to live long, fulfilling lives. This means funding research into the inner workings of cancer in young people, along with clinical trials to test new treatments. But it also requires support from the political world in how laws and regulations are put in place that control how this research is managed.

And we’re concerned that one European law in particular might not be up to scratch.

What we’re doing in the lab

Our research is radically shifting the way children, teenagers and young adults are treated.

We fund around 60 per cent of children’s cancer trials in the UK. For example, we led a series of trials that found much better treatments for a type of childhood liver cancer, boosting survival from 14 per cent in the 1970s to more than 80 per cent today.

In the 1970s, our scientists helped classify childhood leukaemia into four different types, paving the way for more effective and personalised treatments. As a result the number of children surviving the disease for 10 years or more increased from one in 10 in the late 1960s to around nine in 10 today.

Updating an EU law

We’re committed to our research into children’s cancer, but progress is only possible if the research environment is right. So to fully tackle the challenge, we also need to ensure that Governments both in the UK and in Europe help create the right policy environment for progress in children’s cancers.

The recent Cancer Strategy for England sets out ways of improving the treatment of children, teens and young adults as well as improving their access to clinical trials. So it’s important that these recommendations are acted on.

But right now, we’re also focusing on a European law, which could make a real difference to children diagnosed with cancer.

When it was introduced in 2007, the EU Paediatric Medicines Regulation – a law that establishes how all medicines for children are regulated – promised to support the development of cancer drugs for young people by, among other things, requiring that medicines approved for adult use are investigated for their potential benefits in children.

The problem is that companies can avoid carrying out these investigations (called ‘Paediatric Investigation Plans’, or PIPs for short) if they argue that the cancer type doesn’t exist in children.  Too often, companies are using these exemptions to avoid the time and cost of researching children’s cancers.

Ultimately, we believe the law has resulted in far fewer treatments being made available for children than there should be. In fact, of the 6,000 children and adolescents who die of cancer in Europe each year, less than 10 per cent have access to an innovative treatment. That’s just not good enough, so it’s vital that these problems are fixed.

What needs changing?

In early 2017, the European Commission – the EU body that drafts and updates new laws – will decide whether to revise the law or leave it as it stands. So this year we – together with patients, researchers and cancer organisations from across Europe – will be pushing hard to ensure it’s properly reviewed and crucial improvements are made.

We’ll be asking that Paediatric Investigation Plans are based specifically on how a drug may work in children and young people, rather than only being tested on adult tumours. We’ll also look if there are ways to simplify the process of developing these plans to speed up the whole process, and aim to strengthen existing incentives for drug companies, which aren’t going far enough.

This is a crucial period for research into children’s cancers. Our understanding of the basic biology has accelerated in the last 10 years allowing us to glimpse what’s happening ‘under the hood’ in these cancers. The more we understand the mechanics of what drives children’s cancers the sooner we are able to fix them, whether it’s using new treatments such as immunotherapy or drugs known to work in adults but have not yet been tried in children.

And that’s why it’s vital that the laws that govern research into children’s cancers encourage drug development, rather than creating more road blocks.

Catherine Guinard is a public affairs manager at Cancer Research UK

• If you’d like to support our research into children’s cancer, get involved with Cancer Research UK Kids and Teens