EU rules stop children with cancer benefiting from new drugs
Leading cancer experts are calling for a change in the European Union regulations on children's cancer drug trials. They argue that the current system denies children new, potentially life-saving drugs.
The UK's Institute of Cancer Research (ICR) says if the rules were changed, children would have access to drugs that are currently only being tested in adults.
At present, the European Union (EU) rules allow pharmaceutical companies to obtain exemptions from carrying expensive drug trials in patients under the age of 18 - even if there is evidence it might benefit them.
But, says the ICR, the ability of drug companies to use such "class waivers" causes significant delays in new drugs becoming available to the under-18s, and can even mean they are never formally licensed for use in children.
Many EU-approved cancer drugs denied to children
The ICR says 26 of the 28 drugs approved for use in adults in Europe since 2007 have anti-cancer mechanisms that could work in children, but 14 of them were exempted from testing in under-18s because the adult condition the drug was developed for does not occur in children.
Professor Alan Ashworth, chief executive of the ICR, says "the current system is failing to provide children with access to new treatments that could add years to their lives."
"It's essential that ground-breaking cancer treatments are tested not only in adults but also in children, whenever the mechanism of action of the drug suggests they could be effective," he urges.
"That means a drug developed for a cancer in adults could also be effective against a cancer affecting a completely different part of the body in children. The way EU rules are implemented fails to take this into account."
Call to scrap the current system of waivers and exemptions
The ICR has teamed up with the European Consortium for Innovative Therapies for Children with Cancer (ITCC), based in France, to urge the EU authorities to change the regulations and scrap the current system of waivers and exemptions to ensure the drug companies carry out more trials in patients under 18 years of age.
To support their case, they have produced an analysis of the impact of current EU rules.
One example they highlight is that of drugs approved for the treatment of adult cancers involving mutations in two genes: ALK and EGFR. The manufacturers of the drugs have been granted waivers so they do not have to test them in children, even though there is evidence that some mutations of these genes are involved in childhood cancers, say the ICR and ITCC.
The changes they are calling for affect the implementation of the 2007 European Union Regulation on Paediatric Medicine, which was brought in to improve children's access to new medicines.
Professor Gilles Vassal, chair of the ITCC European Consortium, says:
"The European Paediatric Medicine regulation significantly changed the landscape of drug development in children. However, there is an urgent need to change its implementation in order to meet the need for new innovative medicines to cure children and adolescents suffering life-threatening malignancies."
The ICR and ITCC analysis also shows that the European Commission's rules on improving children's access to cancer drugs for treating rare or "orphan" conditions is not working properly.
Of 25 drugs approved in the EU for the treatment of orphan cancer conditions, none was registered for use in children whose cancer is of a different type to that in adults.
Meanwhile in the US, the Food and Drug Administration (FDA) has implemented new procedures to ensure enough women take part in medical device studies.
Written by Catharine Paddock PhD
Copyright: Medical News Today
Not to be reproduced without the permission of Medical News Today.