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Child cancer drug approved for NHS use

60% of children with high-risk neuroblastoma can be treated with dinutuximab beta

Adrian O'Dowd

Thursday, 12 July 2018

Children with high-risk neuroblastoma - a rare type of cancer that mainly affects young people – can now be treated with a drug that has the potential to extend their lives.

A National Institute for Health and Care Excellence (NICE) appraisal committee has announced in final draft technology appraisal guidance published today that it is recommending that dinutuximab beta (Qarziba) be made available on the NHS.

Treatments for high-risk neuroblastoma – a cancer that develops from specialised nerve cells left behind after a baby’s development – include chemotherapy, radiotherapy, stem cell transplant, surgery and isotretinoin.

NICE said that modelled trials had estimated the survival gain of the dinutuximab beta drug was three to five years compared to the current treatment option of isotretinoin.

Neuroblastoma is most common in children under the age of five, and is estimated to affect around 100 children a year. NICE said that 60% of these patients would have access to dinutuximab beta as long as they met 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.

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 [EUSA Pharma] 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.”

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