If you follow our blog, you’ll know that a major area of policy work for The Institute of Cancer Research this year has been how we can increase access to new drugs for children with cancer.
There is an urgent need to accelerate development of innovative treatments for children with cancer, and at the ICR we have been campaigning for changes to an outdated EU Regulation, which we believe is denying children access to the latest cancer drugs. A simple update could make a real difference to the number of new cancer drugs which enter clinical trials in children.
Earlier this year the ICR
responded to a European Commission consultation, calling on it to take into account advances in our knowledge of the biology of cancer, which mean that adult cancer drugs could often also be used to treat children.
This month, the European Commission has released updates to the processes involved, and although there are some small changes, there is nothing like what is needed to make a real difference.
So what is this all about?
At the moment, before pharmaceutical companies can gain marketing authorisation for a new drug in adults, they are obliged to develop a paediatric investigation plan (PIP), which describes how the drug will be studied for potential paediatric use.
A company can get a waiver from developing a PIP and trialling the drug in children if the product is unlikely to be effective or safe in children, if it does not represent a significant advance over existing treatments, or if the disease the drug is intended for does not occur in children.
It’s the final point here which causes the problems. We certainly wouldn’t want companies to be obliged to trial drugs in children when they are likely to be ineffective or unsafe, but judging whether a treatment is likely to work by where in the body a tumour is located is outdated. Medicine has moved on from simply categorising cancers as ‘breast cancer’ or ‘lung cancer’ - we can now look at specific molecular defects in an individual’s cancer, and use medicines specifically targeted at genetic features of that tumour. These genetic features may occur in a subset of patients across a number of cancers, rather than a single tumour type.
With this in mind, giving a company a waiver from testing a dug in children because the disease that it is intended to treat doesn’t exist in children now no longer makes sense. What matters when determining whether a new drug could be effective in children is its mechanism of action – not whichever adult cancers it can be used for.
The ICR would like to see the Regulation revised so that pharma companies can no longer be granted waivers from testing in children when there is reason to believe that the drug could be effective for a childhood cancer.
Looking instead at the mechanism of action of the drug would substantially increase the number of paediatric trials for potentially very important drugs for childhood cancers and could open to door to many new cancer drugs becoming available for children.
How can we achieve this change?
It seems that the Regulation itself can’t be updated for several more years, so we’ve been hoping for changes in the way that it is implemented. This year, the European Commission has been running a public consultation on the guidelines for developing PIPs, which included how companies apply for waivers.
We used this opportunity to call on the European Commission to make a change to the way the waivers are awarded, taking into account a drug’s mechanism of action. Looking at the consultation responses which the European Commission published, we were not the only organisation to be calling for this change – groups all over Europe, including the Teenage Cancer Trust here in the UK – were making the same call. And earlier this year, British MEP Glenis Willmott asked the Commission directly in a Parliamentary Question whether it would take this chance to update the guidance.
We have been waiting to see how the Commission would respond.
The updated guidance was published this week, and what’s changed?
I am really disappointed to say that there has been no move to a system taking into account the mechanism of action of drugs. The only real change to the waiver system is a recommendation that pharma companies should check with the European Medicines Agency whether they qualify for a waiver rather than assuming they will based on the criteria.
The Commission does provide a commentary, looking at what organisations called for in their responses to the consultation, and in some way, this answers the question of why there haven’t been bigger changes.
It considers the change we asked for to be out of scope of the consultation, and it seems to suggest the Regulation itself precludes any major changes to its implementation.
When the original Regulation has not kept up with advances in cancer science, this seems to be a real missed opportunity. If the Commission had used the PIP guidance document to update the way the Paediatric Regulation was implemented, it could have made a real difference now to the 15,000 children and adolescents diagnosed with cancer each year across Europe.
It’s hard to accept that a technicality in EU process should be allowed to deny children the same access to cancer drugs that is already afforded to adults, and we’ll be continuing our policy work in this area. Watch this space.
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