NICE Approves Dinutuximab Beta for Child Neuroblastoma

Nicky Broyd

July 17, 2018

The National Institute for Health and Care Excellence (NICE) has approved dinutuximab beta (Qarziba, EUSA Pharma) for NHS funding in England for children with high-risk neuroblastoma.

NICE had previously rejected the drug for funding on cost grounds but the final draft technology appraisal guidance for the immunotherapy drug came after an NHS discount price was agreed with the manufacturer.

Before any discounts a course of treatment would cost around £152,200.

Destroying Cancer Cells

Dinutuximab beta received its European marketing authorisation in March 2017. The immunotherapy drug gets attached to molecules on the outside of neuroblastoma cells alerting the immune system to destroy them.

Although long-term research is not yet available NICE cites trial modelling giving 3-5 years of additional survival time compared with isotretinoin treatment.

Neuroblastoma is diagnosed in around 100 children a year in the UK - most of them under 5-years-old. The cancer develops from neuroblast nerve cells left behind after the child's early development.

Current treatments for high-risk neuroblastoma include:

  • Chemotherapy

  • Radiotherapy

  • Stem cell transplant

  • Surgery

  • Isotretinoin

NICE estimates that dinutuximab beta infusions could be an appropriate treatment in around 60% of cases.

The criteria include patients being at least 12 months old, and after the cancer has partially responded to first stage chemotherapy, myeloablative therapy, and stem cell transplantation, but who have not received anti-GD2 immunotherapy drugs.

'Important Treatment Option'

In a statement, the director of the NICE Centre for Health Technology Evaluation, Meindert Boysen, 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."

Dr Juliet Gray, Associate Professor in Paediatric Oncology at the Cancer Immunology Centre, University of Southampton, called the decision "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."

Commenting on the decision, Cancer Research UK's policy manager, Rose Gray, said "It's great to see NICE, NHS England, and the company working together, finding a way to make this drug routinely available on the NHS."


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