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Brexit could bar child patients from cancer trials

Close EU loophole now, experts urge

Jo Carlowe

Thursday, 09 March 2017

Cancer treatments are not being trialled in children because of outdated European regulations which are in urgent need of reform, say cancer experts.

Experts from The Institute of Cancer Research (ICR), London, and The Royal Marsden NHS Foundation Trust have set out their concerns in their response to a European Commission consultation on the future of the EU Paediatric Regulation. 

They are urging European officials to reform the rules on clinical trials in children now “or risk cementing the current outdated approach for years to come”.

They also warn of a risk of UK children being denied access to European trials once the UK has left the EU. If the UK leaves the European Medicines Agency (EMA) as proposed, children could also wait years longer for drugs to be licensed for use, they say.

The experts say it is vital that new reformed rules are applied across Europe so children in the UK do not lose out after leaving the EU.

Moreover, the researchers and doctors say there are “nowhere near enough” cancer medicines being trialled in children or licensed for paediatric use.

European regulations currently allow drug companies to opt out of running clinical trials of new cancer drugs in children even where there is good evidence that the drugs could be beneficial, they state.

A new analysis by the ICR of EMA data shows that over the last five years (2012-2016) pharmaceutical companies were granted waivers from having to trial cancer drugs in children for 33 of 53 cancer treatments (62%), which were ultimately approved.

The EU Paediatric Regulation was introduced in 2007 with the aim of improving children’s access to new treatments.

But it allows companies to apply for a waiver from having to trial a cancer drug in children if it targets a cancer that does not affect children, such as lung cancer – even if the drug’s mechanism of action means it could be effective for them.

For example, in the past five years the drugs axitinib and obinutuzumab have received waivers by regulators despite having a mechanism of action that is potentially relevant to children’s cancers.

Axitinib could potentially be effective in childhood neuroblastoma, hepatoblastoma, medulloblastoma, Wilms tumour and ependymoma. It could also have a role in rhabdomyosarcoma and high-grade glioma. Obinutuzumab may be relevant for lymphoma, states the ICR. 

The ICR and The Royal Marsden are 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.

The organisations say the consultation could be the best opportunity for years to urgently deliver the reforms needed.

Professor Paul Workman, chief executive of the ICR, said: “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.

“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. It’s vital that whatever deal the UK does preserves access to Europe-wide clinical trials for children with cancer, and avoids creating even longer delays in children accessing the latest cancer medicines.”

Alongside a series of further recommendations, the ICR and The Royal Marsden are calling for a lower age limit for adult trials so researchers can include adolescents where clinically appropriate. The experts also believe we need more flexible age limits for paediatric trials so researchers can include young adults who have paediatric-type cancers.

And they believe stronger economic incentives are required to compensate drug manufacturers for the financial challenge of developing treatments for children, in whom cancers are much rarer than in adults.

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