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Why now is our best chance to change rules on childhood cancer trials


The Institute of Cancer Research, London and The Royal Marsden NHS Foundation Trust are calling on the European Commission to update rules on testing new drugs in children. Dr Eva Sharpe, our Senior Policy and Engagement Manager, explains why.

Posted on 10 March, 2017 by Dr Eva Sharpe

Blister Pack of Pills

Since I joined The Institute of Cancer Research, London four years ago, there is one policy topic that we have focused on perhaps more than any other – the need to reform the rules around clinical trials for children’s cancers.

We now have our best chance to make a much-needed change to the EU regulation that governs childhood cancer trials.

I thought I’d take some time to explain what the problem is with the current system, and why we could finally have an opportunity to make some important changes.

The issue

Across the EU, drug development for children is governed by the Paediatric Regulation – a piece of legislation specifically set up to increase the number of drugs made available for children.

Since it came in to force in 2007, the consensus is that the Regulation has delivered some progress. But in its current form, it simply doesn’t work for the modern world of cancer medicine. Cancer research has moved on and the Regulation hasn’t kept up.

It’s all down to the rules on whether a drug should be trialled in children or not.

Developing drugs for small patient populations like children’s cancers is financially challenging and doesn’t offer big profits. So as a way of promoting this research, the Regulation obliges companies to submit a plan for studying a new drug in children, before they can gain marketing authorisation in adults.

However, companies can get a waiver from having to prepare and carry out this plan, if the treatment is unlikely to be effective or safe, or if it does not represent a significant therapeutic benefit over existing treatments.

Companies can also get a waiver – and this is the crucial bit – if a drug has been developed against an adult disease that children don’t get. That might sound logical enough, but in fact in cancer it has been a major problem and meant children have benefited from new treatments at a much slower rate than adults.

Why the Regulation doesn’t work for cancer medicines

In the last decade or so, medicine has moved on from simply categorising cancers as ‘breast cancer’ or ‘lung cancer’.

More and more now, doctors are looking at the molecular defects in a cancer, and choosing medicines that target specific mutations.

So a drug which is relevant for a lung cancer in one patient may be relevant in another patient’s brain tumour if they both have the same mutation driving the tumour. That means that a drug designed to target a particular mutation in an adult cancer could also work against the same mutation in children’s cancer.

Under the current system, potentially important cancer drugs are granted waivers against testing in children, even though they could be effective for paediatric cancers too.

We have been campaigning for several years to close this loophole so that wherever there is a case that a cancer drug may be beneficial for children, it is tested. This change could bring new types of drugs to the clinic for children and make a huge difference to survival rates in childhood cancers.

Voluntary trials

There are some companies which are trialling new drugs in children in cases where they had previously been granted a waiver. This is a positive step which we welcome.

But in a report to the European Commission, the EMA found only 14 cases where this has happened since 2008. So only a small proportion of the total number of drugs which might have been relevant for children with cancer are being voluntarily evaluated.

This suggests it is not enough to rely on voluntary action. The ICR has been arguing that the Regulation itself needs to be updated to really have an impact.

An opportunity for change

We and others have been calling for some time for this change. We were pleased to see the EMA propose an interim solution in 2015 that may reduce the number of waivers granted when it comes into force next year. But in truth, until the Regulation itself is updated, we’re not going to see the scale of increase in trials for children that we need.

Paediatric cancer medicine needs to incorporate advances seen in adult cancer treatments such as molecularly targeted drugs and the use of biomarkers, and this can only occur through more trials.

At the moment the European Commission is preparing a report for the European Parliament and Council on the Regulation, outlining the impact it has had on public health and business.

These are the two bodies that adopted the Paediatric Regulation – so this is a major opportunity to let EU decision makers know about the drawbacks in the current rules, and strongly push for them to be updated.

The ICR, and many other organisations with an interest in paediatric research, sent detailed feedback to the Commission while it gathered evidence for its report so we will wait for the report with interest to see if these points are picked up.

We see this as our biggest opportunity yet to make a change to the Regulation, and one that we really hope the Commission will take.

In its consultation, the European Commission specifically asked the sector about future scientific and medical trends that will need to be addressed in any new regulations.

To me this suggests that the Commission wants to learn the lessons from the current Paediatric Regulation, and avoid drafting anything that can get so badly outdated.

I’m optimistic that we will get the changes we have argued for – and that children will finally start to gain the full benefit from the latest advances in cancer medicine.

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