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A step forward in our campaign to increase children’s access to new drugs

13
Aug
2015

One policy issue that the ICR is involved in is discussing the regulatory barriers that our researchers face in running clinical trials in children – we have been pushing for changes to the way EU regulations are implemented.

Posted on 13 August, 2015 by Professor Paul Workman

Since taking on my job as Chief Executive and President here, I’ve increasingly found myself drawn into discussions on the complexities of science and health policy.

I’ve spent considerable time thinking about ways to improve the policies and systems that surround science and health – to help our own research here at The Institute of Cancer Research, London, and because I see it as part of my role to help move the whole field of cancer research and treatment forward.

At the ICR, we’ve been involved in the debates over how to make innovative new drugs available on the NHS more quickly, how to create a sensible system for allowing researchers access to patient data, and what more the Government could do to encourage academic institutions and private companies to work together.

But there has been one policy issue that has taken up our time and attention in particular, because it has such a direct impact on our ability to do research that benefits patients: the regulatory barriers that our researchers face in running clinical trials in children.

The ICR has been pushing for changes to the way EU regulations are implemented, with our aim being to prevent pharmaceutical companies from being able to avoid conducting clinical trials of new cancer drugs in children when those drugs could be beneficial.

Under the current system, when companies develop new cancer drugs for adults they often get exemptions from having to conduct expensive testing in children too – even where a drug’s molecular mechanism of action suggests it could work in kids.

As a result there are significant delays in new drugs becoming available for children, and some drugs may never be trialled and licensed in children at all.

It has been extremely frustrating for our researchers that they find it so difficult to run clinical trials of new drugs for paediatric patients – and so over the last 18 months, we’ve done a lot to highlight the importance of the issue.

We’ve worked with others in the paediatric oncology community to raise the problem with politicians and policy makers – working with MEPs and MPs to keep the pressure on the European Commission through Parliamentary Questions, and responding to two EU consultations.

We’ve also worked to raise awareness of the problem more widely– running a press briefing at the Science Media Centre, and coordinating a joint letter in The Telegraph from cancer charities. The issue has been picked up in many news articles, and also by organisations such as the Nuffield Council of Bioethics, which highlighted it in its recent report on ethical issues in clinical trials for children.

I’m pleased to say that those messages have been heard. European regulators have now announced changes to the way that the EU Paediatric Regulation governing clinical trials is implemented.

The European Medicines Agency (EMA) has changed its rules on granting pharma companies ‘waivers’ from having to assess drugs in children, to make it harder for companies to opt out of doing so.

Under the revised rules, companies will not be able to gain a ‘class waiver’ for an adult cancer drug simply because the precise condition it was developed for doesn’t occur in children.

Instead, class waivers will only be available for a small number of drug types where there is good evidence they are unlikely to work in children.

For all other drugs, companies will have to run clinical trials in children unless they can convince an EMA panel of oncologists that there is a particular reason why the treatment is unlikely to benefit paediatric patients – in which case they could be granted a product-specific waiver.

The change is intended to ensure a more evidence-based decision-making process, which should take into account key issues such as the mechanism of action of the drug in question.

So for example if a drug acts on the effects of a faulty cancer gene, then it could be evaluated in children whose cancers have that same defect – even if the name and location of the cancer happens to be different from in adults. This is much more logical and scientifically justified.

It’s always hard to predict the exact effect of changes in the complex EU rules governing research, but it should mean that children do eventually get access to an expanded palette of targeted cancer drugs.

The new guidance comes into force in 2018, and will not be applied retrospectively to drugs already granted a waiver, so it’s not all good news. But it is an important interim step in widening access to new cancer treatments for children, while discussions continue over reforming the EU Paediatric Regulation itself.

We’re very pleased that our efforts – working with the children’s cancer community – have helped to deliver some important changes to the process for running clinical trials of new drugs.

The EMA cited the ICR’s campaign on the issue – spearheaded by Professor Louis Chesler here – as one of the key justifications for making the change. Its report said that the fact that academic stakeholders such as the ICR and paediatric patient representatives had repeatedly raised the issue had prompted concerns about how to serve public health objectives in children.

It’s vital that new cancer drugs are assessed in children whenever there is evidence that they could be effective. The revised guidance from the EMA is an important step forward in making sure that happens.

But it’s not the end of the matter. The class waiver system has been reformed, but not so radically to ensure that all potentially valuable cancer drugs are likely to be tested in children as well as adults.

So we will continue to work with the children’s cancer community and the EMA to push for changes to the EU Paediatric Regulation itself. That way, it should be possible to realise our ultimate aim – by making sure that children benefit just as much from the new wave of targeted cancer treatments as adults.

As I said at the beginning of this post, I’ve become more involved personally in a range of policy issues affecting cancer research and treatment. This success with improving access to drugs for children’s cancers convinces me that research organisations like the ICR have an important role to play in policy debate – and can help effect change.

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