The National Institute for Health and Care Excellence has ruled against the provision of NHS funding for EUSA Pharma’s neuroblastoma treatment Qarziba.

Qarziba (dinutuximab beta) is a monoclonal chimeric antibody developed to target a specific antigen, GD2, on neuroblastoma cells, approved in Europe last year to treat the condition in children aged 12 months and above.

However, in draft guidelines, the cost watchdog said while the immunotherapy could increase overall survival compared with current treatment, there is still “substantial uncertainty” about its long-term benefits.

Because of its promise, the Institute’s appraisal committee stated that it would like to recommend the drug for managed access in the Cancer Drugs Fund (CDF), but only if company lowers its cost to the NHS.

Currently, 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, the Institute concluded, and thus much higher than what is normally considered a cost-effective use of NHS resources.

“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,” noted Meindert Boysen, director for the NICE Centre for Health Technology Evaluation.

In a statement, EUSA said it is pleased that NICE recognises the clinical value of the drug, but is "deeply disappointed in the draft guidance received by NICE which could now leave children with this rare, life-threatening cancer without access to a potentially curative treatment option.

"Approximately 40 children in the UK with high-risk neuroblastoma would benefit from treatment with Dinutuximab Beta. This would represent only 0.05% of the total NHS expenditure on medicines. Dinutuximab Beta is internationally recognised as standard care for this group of young patients. It is already available in several other countries across Europe where it has been deemed both affordable and a cost-effective use of health care funding."

The firm also stressed that the draft guidelines are not final."Every conversation we have had thus far with NICE and the clinical or patient community has been very supportive of reaching a positive outcome by the time the final appraisal is issued later this year. EUSA Pharma is committed to working with all parties to achieve this."

Neuroblastoma is a rare type cancer affecting around 100 children each year in the UK, which develops from specialised nerve cells called ‘neuroblasts’ left behind after a baby’s development.

The main aim of current treatment is to extend survival. Until 2009, maintenance therapy with isotretinoin was considered standard care in the NHS for people with high-risk neuroblastoma, but since then, Qarziba has been available through a clinical trial and free supply from the company.