Long term benefit of neuroblastoma treatment is too uncertain, says NICE

NICE has published draft guidance in which dinutuximab beta is not recommended as a treatment for high-risk neuroblastoma – a rare type of cancer that mainly affects children and young people.

Evidence suggests dinutuximab beta (Qarziba, EUSA Pharma) could increase overall survival compared with current treatment, but there is still substantial uncertainty about its long-term benefits.

Because dinutuximab beta has promising clinical benefit, the NICE appraisal committee would like to recommend it for managed access in the Cancer Drugs Fund (CDF). However, for it to be able to do so, the company will need to reconsider the cost of the treatment to the NHS.

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.”

Neuroblastoma is a cancer that develops from specialised nerve cells called ‘neuroblasts’, which are left behind after a baby’s development. It is most common in children under the age of 5, and is estimated to affect 100 children each year in the UK.

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.

NICE’s draft recommendation is not intended to affect treatment with dinutuximab beta that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop.

This preliminary decision from NICE, is now the subject of public consultation until Tuesday 29 May 2018.