NBUK statement on NICE decision not to fund Dinutuximab beta for children

Press Statement 4 May 2018

Embargoed until 8.00 AM, Friday 4th May 2018

Neuroblastoma UK has expressed its disappointment at today’s announcement by the National Institute for Health and Care Excellence (NICE) that it has not approved Dinutuximab beta for funding in the NHS. This will be devastating news for the families. This decision potentially denies their children, who are newly diagnosed with Neuroblastoma, a life-threatening condition, access to a drug that is readily available within Europe and the USA, where a similar treatment is provided.

Dinutuximab beta is used to treat high-risk neuroblastoma, a rare and life-threatening disease affecting around 50 children in the UK each year.

NICE have accepted that the clinical trial using Dinutuximab beta is effective in increasing survival for these patients but does not currently meet its incremental cost effectiveness ratio.

Chair of Neuroblastoma UK, Tony Heddon said "We are disappointed at the decision and the uncertainty that this will bring to families. Although understanding the rationale, we feel that as high-risk neuroblastoma is a rare disease, it should be assessed as such, and either a higher financial threshold should apply or an alternative appraisal procedure used. We must seek to find a UK solution to this problem so that parents do not feel the need to seek treatment abroad at great personal expense."

NICE recognise that Dinutuximab beta is a promising drug but feel more data is required. We will therefore be looking to see how, on behalf of neuroblastoma children and their families, we can work with EUSA, the drug’s manufacturer, NHS England and NICE to come up with an alternative solution, such as the Cancer Drugs Fund. We would press all parties to be flexible in determining the value of the drug to find a swift solution, which will allow us, to gain more accurate/definitive data to meet the NICE requirements, and also enable children the time for new therapies to be developed.

Tony Heddon, Chair of Neuroblastoma UK added “It is imperative that children have access to the latest treatments and that we continue to invest in research into this terrible disease. Through doing so we will improve survival from 40% to 85% as has been achieved in childhood leukaemia.”


Notes for Editors:

Dinutuximab beta is a monoclonal antibody used as second-line treatment for children who have high-risk neuroblastoma, which has an increased chance of coming back. The drug works by binding to GD2, a glycolipid, which is present in high amounts on the surface of neuroblastoma cells, but not normal cells. When Dinutuximab beta attaches to the neuroblastoma cells, it makes them a target for the body’s immune system, which then kills the cancer cells.

Dinutuximab beta was approved by the European Medicines Agency in May 2017.

NICE has stated that 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, which is higher than what NICE normally considers to be a cost-effective use of NHS resources (that is, between £20,000 and £30,000 per QALY gained).

Neuroblastoma UK is a charity funding research into neuroblastoma to find a cure and deliver new, effective, and kinder treatments.

  • Neuroblastoma is an aggressive childhood cancer, with 1 in 6 of all children’s cancer deaths due to Neuroblastoma.
  • Neuroblastoma is the second most common solid tumour in childhood and the most frequent single type of cancer in the first year of life.
  • Research for a cure is largely dependent upon funding provided by charities such as Neuroblastoma UK, who is a major funder of research into Neuroblastoma, and has funded £5M worth of research in the UK.
  • Since Neuroblastoma UK (formally The Neuroblastoma Society) was established (1982), 5-year survival of children aged 1 – 14 years old with Neuroblastoma has doubled from 34% to 68%.


For Neuroblastoma UK:       Alastair Whitington      media@neuroblastoma.org.uk

                                             Tony Heddon                chair@neuroblastoma.org.uk