NICE has recommended dinutuximab beta for treatment of high-risk neuroblastoma – a rare type of cancer that mainly affects children and young people – in final draft technology appraisal guidance published today.
It will be given to patients aged 12 months or over whose disease has at least partially responded to first stage chemotherapy, followed by myeloablative therapy and stem cell transplant but only if they have not received anti-GD2 immunotherapy.
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 five, and is estimated to affect 100 children each year in the UK.
Evidence suggests dinutuximab beta (Qarziba, EUSA Pharma) increases overall survival compared with current treatments. While there is uncertainty around the long-term clinical benefit of the drug, the appraisal committee believed the potential survival gain offered by its use was substantial.
The committee took into account other factors, including health-related benefits not captured, the rarity and severity of the disease and the potential to increase survival for children with high-risk neuroblastoma. This allowed the NICE appraisal committee to recommend dinutuximab beta.
An agreement has been reached with EUSA Pharma to allow the drug to be made available to the NHS at a confidential discounted price. The average list cost of a course of treatment for a three year old is £152,200.
Treatments for high-risk neuroblastoma include chemotherapy, radiotherapy, stem cell transplant, surgery and isotretinoin. The main aim of current treatment is to extend survival, but experts have called for a cure.
Meindert Boysen, director for the NICE Centre for Health Technology Evaluation, said: “Dinutuximab beta is an important treatment option for children and young-people with high-risk neuroblastoma in particular, and has shown the potential to increase their survival. We are grateful to the company for responding to our evaluation by setting a price which is cost-effective to the NHS.”