Cancer experts urge reform of paediatric cancer trial rules

Cancer experts are calling for a reform of outdated European regulations which they are argue are preventing cancer treatments from being trialled in children.

The UK’s Institute for Cancer Research (ICR) says the rules on clinical trials in children must be reformed now or there is a risk of "cementing the current outdated approach for years to come". Experts have also stressed that it is crucial that any new rules are applied across Europe to include children in the UK after Brexit.

After the UK leaves the EU, paediatric cancer patients in the country may no longer have access to existing cancer trials, and could also have to wait "years longer" for medicines to be licensed for use if the UK leaves the European Medicines Agency (EMA) as proposed, the Institute is warning.

European officials are currently consulting on the future of EU Paediatric Regulation, which was introduced in 2007 to improve children’s access to new treatments.

As it stands, the regulations allow drug companies to opt out of running clinical trials of new cancer drugs in children, even if there is evidence that they could be beneficial to them.

A new analysis by the ICR of EMA data shows that over the last five years pharmaceutical companies were granted waivers from having to trial cancer drugs in children for 33 of 53 cancer treatments (62 percent), which were ultimately approved.

For example, axitinib and obinutuzumab were opted out of paediatric trials despite having a mechanism of action that is potentially relevant to children’s cancers, the Institute said.

The ICR and The Royal Marsden are now calling on the European Commission to revise the waiver system so that adult cancer drugs have to be tested in children whenever there is evidence that they could be effective.

Alongside a stream of other recommendations, they are also arguing for a lower age limit for adult trials so researchers can include adolescents where clinically appropriate, and more flexible age limits for paediatric trials so that they can include young adults who have paediatric-type cancers.

Stronger economic incentives are also required to compensate drug manufacturers for the financial challenge of developing treatments for children, in whom cancers are much rarer than in adults, experts stress.

Current EU paediatric regulation "is not fit for purpose," said Professor Louis Chesler, team leader in Paediatric Solid Tumour Biology and Therapeutics at the ICR and consultant in Paediatric Oncology at The Royal Marsden.

"By allowing pharmaceutical companies to use waivers to avoid trials in children so they can focus on adult treatments, the regulation is stifling progress and could be stopping children receiving a treatment that could save their lives."

"We’ve been urging decision makers to change the regulation for several years now, so that adult cancer drugs are tested in children whenever their mechanism of action suggests they could be effective," added Professor Paul Workman, chief executive of the ICR.

"This is a real chance for reform to prevent the current out-of-date approach from being cemented for a decade. It could also be the last chance to make meaningful changes that apply across Europe, including the UK, before we leave the EU".