Children with cancer are being denied new, potentially life-saving drugs, because EU rules are enabling pharmaceutical companies to trial some drugs only in adults, according to the UK’s Institute of Cancer Research (ICR). The ICR has called for urgent modifications to the European Union Regulation on Paediatric Medicine to make it more difficult for pharmaceutical companies to avoid testing drugs in children.
The regulation, which came into force in 2007, was intended to promote the development of medicines for children; at the time, over half the medicines used in children were prescribed ‘off-label’ and not specifically tested for use in this age group. Under the regulation, companies are offered longer market exclusivity if they test their drugs in children.
The proportion of clinical trials including children has increased since the regulation was implemented. Nevertheless, pharmaceutical companies often gain exemptions or ‘class waivers’ from carrying out expensive testing of cancer drugs in under 18s if the adult cancer targeted does not occur in children.
ICR chief executive Alan Ashworth said the way EU rules are implemented fails to take into account that modern cancer treatments are often targeted at genetic features of the tumour that may be common in a cancer affecting a completely different part of the body in children.
The ICR and the France-based European Consortium for Innovative Therapies for Children with Cancer (ITCC) have found that of 28 cancer drugs approved for adult marketing authorisation in Europe since 2007, 26 have a mechanism of action relevant for paediatric malignancies, but 14 have been waived from being tested in children. As a result there are significant delays in new drugs becoming available for children, and some drugs may never be formally licensed for paediatric use, according to the ICR.
‘The issue is not as straightforward as the ICR makes it to be,’ points out Yoon Loke, clinical senior lecturer at the University of East Anglia. ‘In some instances, there may not be any immediately apparent target for the drug in children today, although there may be a slim possibility of it becoming of use five to 10 years later. Is any drug company prepared to invest in such a slim prospect that may only arise many years ahead? Moreover, trials in children carry inherent risks of harm for drug toxicity. Should today’s children be exposed to such risks if there is no clear therapeutic pathway for benefit in future?’