Concerns about involving children in clinical trial having a material impact on their access to new treatments for cancer and other diseases, says a new report.
The study – by the Nuffield Council on Bioethics in the UK – concludes that children are missing out on treatment advances that are helping adults and makes a series of recommendations aimed at redressing the balance.
Doctors often have to customise adult medicines in order to provide a dose suitable for use in children – which is often based on an imperfect calculation based on body weight given the lack of clinical data in children – and not enough care is taken to make sure drugs are palatable and child-friendly.
The recommendations include a greater emphasis on partnerships between industry and public research networks, and giving greater consideration to the views of young people, their parents and child health experts when drawing up research proposals, taking into account prior experience of clinical trials.
Moreover, EU regulations on trialling new drugs in children also need to be revised to ensure cancer drugs developed for adults are also tested in children whenever they could be effective, says Nuffield.
In particular the report calls for a review of the use of waivers that allow sponsors developing drugs for ‘adult-only’ diseases to be exempted from testing them in children, because at the moment the system “is not working as intended.”
At the moment, sponsors can get a waiver from developing a paediatric investigation plan (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.
Waivers are however being issued for drugs even when their mechanism of action might be applicable to children with cancer, says the report.
The report has been welcomed by the Institute of Cancer Research (ICR), which has been campaigning for a change to the 2007 EU Paediatric Regulation for some time. Last year, the ICR reported that out of 28 new cancer drugs approved in the EU for adults since 2007, the 26 could potentially work in children but just 14 of these were exempted from paediatric testing via the waiver system.
Policy manager Eva Sharpe wrote in a recent blog post that she was pleased to see the recommendation to close the waiver loophole “as a matter of urgency, to ensure drugs are evaluated in children whenever there is evidence they could work.”
The Nuffield report also calls for incentives to be introduced for paediatric clinical research on off-patent medicines in preparation for the 10-year review of the EU regulation that gets underway in 2016.