Call for change in EU regulations that allow pharmaceutical companies to opt out of children's trials for some cancer drugs

Potentially life-saving drugs are being denied to children with cancer because they are not being put through the necessary clinical trials, say scientists who are calling for a change in European rules.

The Institute of Cancer Research says the current EU regulations allow pharmaceutical companies to opt out of children's trials if a new drug is intended for an adult cancer that does not occur in children.

But scientists say cancer drugs are increasingly aimed at molecular targets that may occur in many kinds of cancer. So, for example, a new drug for a type of lung cancer attacks a molecular defect that is also found in neuroblastoma – a devastating and often lethal children's cancer – but it will not be put through trials to make it available to children.

"Children do not have access to drugs that could be available in clinical trials even where scientists believe they could extend lives," said Prof Alan Ashworth, chief executive of the institute.

Since 2007, all but two of 28 cancer drugs given a licence for adult use in Europe had a mechanism of action which is relevant to a children's cancer. However, 14 have been given a waiver, allowing the company not to do a trial in children because they do not get that specific cancer.

"We believe the EU regulatory system is badly out of date and has not kept up with modern conceptions in cancer research," said Ashworth.

The original intention was well-meaning; it was designed to protect children from unnecessary treatment which might not benefit them, he said. But our understanding of cancer has moved on.

One of the adult cancer drugs that might help children is crizotinib, made by Pfizer and licensed to treat adults with non-small cell lung cancer. Dr Louis Chesler, a paediatric oncologist at the Royal Marsden, said the drug targets a genetic mutation that also occurs in some children with neuroblastoma.

"These drugs would not be available for children in any form because lung cancer does not exist in children," he said.

The institute, together with the European Consortium for Innovative Therapies for Children with Cancer, based in France, is lobbying for the EU to scrap the waiver option for drug companies, so that they are always required to plan clinical trials for children.

Paul Burstow, MP for Sutton and Cheam and former health minister, who recently co-chaired a summit meeting of leading oncologists, cancer researchers and health regulators to discuss children's and adolescents' access to clinical trials, said: "Our scientists are doing phenomenal work understanding the evolution of cancer and how to tackle it. However, that work is being held back by rules which harm patient care.

"Childhood cancer is a tragic reality for thousands of families, and it makes no sense to restrict research into potentially life-saving new treatments.

"It is important that we have rules to govern the ethical pursuit of new medicines, but these rules must be grounded in scientific reality and human need."