Seriously ill children battling high-risk neuroblastoma – a rare type of cancer that mainly affects children and young people – are to be offered a drug which has potential to extend their lives.
A National Institute for Health and Care Excellence (NICE) appraisal committee has recommended dinutuximab beta in final draft technology appraisal guidance published today.
Modelled trials have estimated the survival gain of the drug is three to five years compared to the current treatment option of isotretinoin.
Meindert Boysen, director for the NICE Centre for Health Technology Evaluation, said: “We are pleased to be able to recommend dinutuximab beta. It 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.
“We welcome this new and effective treatment option.”
Dr Juliet Gray, Associate Professor in Paediatric Oncology at the Cancer Immunology Centre, University of Southampton, said: “Today’s decision by NICE is a vital step forward in the treatment of young children with this aggressive type of cancer.
“By harnessing the body’s own immune system, dinutuximab beta has shown it can target and attack this cancer very effectively in some patients.
“For some children this could mean extra weeks or months with their families, for others it may even lead to them becoming cancer-free for a long period of time.”
Neuroblastoma is most common in children under the age of five, and is estimated to affect around 100 children a year. Sixty per cent of these patients will have access to dinutuximab beta as long as they meet a number of criteria.
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.
The drug must also be provided by the company according to the commercial arrangement struck with the NHS, for treatment to be approved.
Neuroblastoma is a cancer that develops from specialised nerve cells called ‘neuroblasts’, which are left behind after a baby’s development.
Treatments for high-risk neuroblastoma include chemotherapy, radiotherapy, stem cell transplant, surgery and isotretinoin.