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NICE queries long-term benefit of neuroblastoma treatment

But it is prepared to recommend it for inclusion in Cancer Drugs Fund, providing cost is lowered

Caroline White

Friday, 04 May 2018

The National Institute for Health and Care Excellence (NICE) is not minded to recommend a treatment for high risk neuroblastoma, on the grounds that its long-term benefits remain unclear.

But it is prepared to recommend it for inclusion in the Cancer Drugs Fund (CDF), providing its cost to the NHS can be reduced.

In preliminary guidance published today, NICE gives a provisional thumbs down to dinutuximab beta for this rare type of cancer that mainly affects children and young people.

Neuroblastoma is most common in children under the age of five, and is estimated to affect 100 children each year in the UK.

While the evidence suggests that dinutuximab beta (Qarziba, EUSA Pharma) could increase overall survival compared with current treatment, there is still substantial uncertainty about its long-term benefits, says NICE.

But because the treatment shows promise, the NICE appraisal committee would like to recommend it for managed access in the CDF. However, for this to happen, the company would need to reconsider the cost of the treatment to the NHS, says NICE.

The most plausible incremental cost-effectiveness ratio (ICER) is likely to lie between £62,300 and £79,900 per quality-adjusted life year (QALY) gained, which is higher than what NICE normally considers to be a cost-effective use of NHS resources─ between £20,000 and £30,000 per QALY gained.

Meindert Boysen, director for 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.”

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

This decision is now out to public consultation until Tuesday 29 May 2018.

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