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Can we be satisfied with progress on children’s cancer?


A new analysis of the impact of European regulations designed to bring through new treatments for children with cancer has expressed disappointment at the speed of progress. The ICR’s Dr Eva Sharpe finds out more.

Posted on 26 March, 2018 by Dr Eva Sharpe

Berlaymont EU commission building in Brussels with EU flags

Berlaymont EU commission building in Brussels. Credit: Flickr user Dimitris Avramopoulos (CC BY-SA 2.0)

A new article by leading childhood cancer experts has explored whether efforts by the European Union to make new treatments available for children have lived up to expectations.

In short, the answer is no.

There was a lot of hope that a long-awaited review of the EU’s Paediatric Regulation, which governs the development of paediatric medicines in Europe, would recommend substantial and immediate change.

Here at The Institute of Cancer Research, London, we hoped it would be an opportunity to close a loophole that we and many others in the paediatric research community have been arguing against for many years.

So it’s very disappointing that the loophole remains in place for now, and that we are no closer to seeing changes to regulations that could open up access to new treatments for many more children with cancer.

Exemptions from testing cancer drugs in children

This time last year, the European Commission carried out a large-scale consultation, gathering evidence for a new review of what the regulation had achieved in its first 10 years.

The fact is that while the Paediatric Regulation has had a positive impact on the health of children overall, the picture is quite different for children with cancer, and the Commission’s own report acknowledges this specifically. While there has been great progress in discovering targeted treatments for adult cancers, this has not been the case for children.

In particular, there are major concerns over a system that governs ‘waivers’ which allow companies exemptions from testing cancer drugs in children if the specific disease (eg breast or prostate cancer) doesn’t occur in children, even where a drug's mechanism of action suggests it could work for them.

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Widespread efforts to raise concerns

The ICR, alongside other cancer research organisations, the paediatric oncology medical community and many parent-led charities have been pushing for changes to the regulation for several years, to try and ensure that pharmaceutical companies will be obliged to make their promising new cancer drugs available for testing within clinical trials for children. 

We felt that European Commission’s consultation was our best chance to strongly advocate for the much needed changes to the regulation and, alongside many others in the paediatric research community, we sent detailed feedback to the European Commission as part of the process.  

So when the European Commission published its report, State of Paediatric Medicines in the EU, last Autumn, we were hopeful that it might have taken on board our concerns.

Delayed by further review

Sadly not. While the report acknowledged that there are problems with children’s access to new cancer drugs, it suggested a further review was needed before any updates to the current regulations could be made.

It suggested further investigation into how the Paediatric Regulation works alongside another piece of European legislation known as the Orphan Drug Regulation, which governs the development of ‘orphan drugs’ - pharmaceutical agents to treat rare medical conditions.

Because of their low incidence, all paediatric cancers are classed as rare medical conditions, and yet the Orphan Drug Regulation has not benefited children with cancer in the way it might have been expected to do.

The view from the experts

In a new comment piece for The Lancet Oncology, a group of paediatric experts from the ACCELERATE Platform working group – including Professor Andy Pearson of the ICR and Dr Lynley Marshall, a Consultant in Paediatric and Adolescent Drug Development at The Royal Marsden NHS Foundation Trust and ICR researcher, alongside parent-led charity representatives – reviewed the report from the European Commission and the impact it will have for children with cancer.

They concluded that while the Commission does highlight the relevance of a potential new approach based on the mechanism of action of new drugs, it is disappointing that the report doesn’t suggest any concrete proposals about how this could be implemented.

In contrast, the ACCELERATE group lay out a series of practical suggestions which could be undertaken now, and in fact some of which are already underway.

As well as suggested changes to the regulation itself, they recommend that we need an agreed list of the best drug targets for children’s cancers, joint projects between researchers, doctors and companies to help identify which drugs should be tested in children first, and molecular profiling of children at diagnosis and relapse to learn more about the causes of children’s cancers and determine which are the most suitable treatments.  

Urge to prioritise the review

The authors are concerned that the Commission’s further two-year investigation is likely to lead to further delays in drug development for children with cancer.

In their own analysis into how the Orphan Drug Regulation has been used for cancer treatments, they found that it has achieved little to encourage drug development for children’s cancers.

They found that only two per cent of the orphan drug designations for cancer drugs were for treatments specifically for children, and that of the 272 treatments that were applicable for both adult and childhood disease, 74 per cent had no information whatsoever about paediatric use when they received market authorisation. This is not a route that is successfully encouraging new drugs to be developed for children with cancer.

The authors state their disappointment; the hopes and expectations of the paediatric oncology and parent community have not been met by the report. They urge the Commission to prioritise reviewing the regulation more urgently than currently planned.

The approach in the US

One thing that comes clearly out for me is that the authors also believe that that a collaborative and international approach will be the best way forward if we’re to give children with cancer rapid access to new treatments.

These debates around mechanism of action are not just going on in the Europe – there have been parallel discussions happening in the US for several years.

And last year, the Research to Accelerate Cures and Equity (RACE) for Children Act was passed by the US Congress, which requires companies to test drugs in children where the mechanism of action could be applied to a paediatric drug target too. This act is precisely what we had hoped to see in Europe.

While this widens the gap between Europe and the US, we’re really hoping that the approach taken and the lessons learnt by the FDA will help inform policy decisions in Europe, resulting in greater alignment which is beneficial to children everywhere.

Next steps

Last year, the ICR and 15 other cancer charities wrote to Jean Claude Juncker, President of the European Commission, and Vytenis Andriukaitis, Commissioner for Health, setting out our concerns over the delays while the further review is carried out.

In a joint letter, the signatories called on President Juncker and Commissioner Andriukaitis to use their influence to bring about the changes much sooner than is currently planned. We were pleased that as a result, the ICR has been invited to join European Commission discussions on how to widen children's access to the latest cancer drugs.

A response from Commissioner Andriukaitis, on behalf of himself and President Juncker, said there would be a series of workshops on the Paediatric Regulation and invited the ICR to send a representative.

It’s good to be at the table, but what children really need is change – and the time is ripe for it. With the recent legislation from the US, the appetite for driving change forward internationally is clearly apparent, and it’s needed much more urgently than the European Commission has promised up until now. 


policy childhood cancer European Union
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