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Substantial uncertainty about benefits of neuroblastoma drug – health officials

The rare type of cancer affects around 100 children each year in the UK.

A drug will not be made available to patients with a rare type of cancer that mainly affects babies and young children due to its high cost and “substantial uncertainty” about its long-term benefits, health officials have said.

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.

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 was currently too expensive to do so.

It said although there was “still substantial uncertainty about its long-term benefits”, evidence suggests dinutuximab beta could increase overall survival compared with current treatments.

The company needs to demonstrate that the drug has at least the potential to be cost-effective before we can consider recommending dinutuximab beta Meindert Boysen

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

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.

The main aim of current treatment is to extend survival, but experts have called for a cure.

Until 2009, maintenance therapy with isotretinoin was considered standard care in the NHS for people with high-risk neuroblastoma. Since then, dinutuximab beta has been available through a clinical trial and free supply from the company.

NICE said its draft recommendation was not intended to affect treatment with dinutuximab beta started in the NHS before the guidance was published.

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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