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Decision on children’s cancer drug devastating, say charities

Charities say parents will have to try to raise hundreds of thousands of pounds for their children to receive the treatment overseas.

Cancer charities have said it is “devastating” that a drug will not be made available on the NHS to treat patients with a rare type of cancer that mainly affects babies and young children.

The National Institute for Health and Care Excellence (Nice) said it would like to recommend dinutuximab beta for inclusion in the Cancer Drugs Fund (CDF), but it is currently too expensive to do so and there remains “substantial uncertainty” about its long-term benefits.

Charities that support children with high-risk neuroblastoma – a cancer of the nerve tissue – said the decision was “immoral” and families will now have to try to raise thousands of pounds to send their children for the treatment overseas.

Stephen Richards, chief executive of Solving Kids’ Cancer, said: “Anti-GD2 antibody therapy given at the end of standard therapy represents the best chance of long-term survival for children diagnosed with high-risk neuroblastoma.

“It is recognised as standard of care by clinicians internationally.

“This is devastating for families – it takes away hope in the fight against high-risk neuroblastoma, and it’s immoral to deny children the chance of an essential treatment that could enable them to live longer or cancer-free.

“When we know that there is an effective drug available, it’s immensely frustrating for parents to be entangled within a system that leaves them no choice but to raise hundreds of thousands of pounds for their children to receive this treatment overseas.

“Having the extreme pressure of fundraising plus the anxiety and logistics of travelling abroad for treatment puts an enormous emotional and practical strain on families.”

We owe it to these vulnerable children to give them the best chance of survival Stephen Richards, of Solving Kids’ Cancer

Neuroblastoma develops from specialised nerve cells (neuroblasts) left behind from a baby’s development in the womb and affects around 100 children each year in the UK, most commonly under the age of five.

Mr Richards was also speaking on behalf of the charities Neuroblastoma UK, the Children’s Cancer and Leukaemia group, JACK, The Bradley Lowery Foundation, Hugs from Henry, Christopher’s Smile, Niamh’s Next Steps and Smile with Siddy.

Football mascot Bradley Lowery, six, from Blackhall, County Durham, died in July last year after being diagnosed with neuroblastoma when he was 18 months old.

The Sunderland fan was a mascot for his beloved club along with Everton and England as he battled the condition.

Nice said it was estimated dinutuximab beta would cost the NHS between £62,300 and £79,900 per quality-adjusted life year gained – higher than what is normally considered to be a cost-effective use of resources, which is between £20,000 and £30,000 per quality-adjusted life year gained.

Mr Richards added: “Nice has been directed to work within an appraisal route that is based entirely on complex health economics.

“This doesn’t take into account the nature of the disease and any of the unique contexts, challenges and considerations that exist within rare children’s cancers.

“Instead, a drug that saves the lives of some children and extends the lives of others has been appraised against metrics designed to evaluate treatments for chronic conditions affecting adults. The system’s one-size fits all approach is simply not fit for purpose.

“We owe it to these vulnerable children to give them the best chance of survival, and we will do everything in our power to keep fighting for these families.”

He said the decision leaves the UK lagging behind the United States and other European countries.

Meindert Boysen, director of the Nice Centre for Health Technology Evaluation, said: “Dinutuximab beta shows a lot of promise, but the evidence is uncertain and we must acknowledge this.

“There is opportunity for the company to collect longer-term data from the ongoing trials. This could make dinutuximab beta a candidate for inclusion in the Cancer Drugs Fund.

“However, the company needs to demonstrate that the drug has at least the potential to be cost-effective before we can consider recommending dinutuximab beta be included in the CDF.

“As such, we are keen to work with the company and NHS England to help them explore options.”

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