NICE approves neuroblastoma drug

Press Statement 12th July 2018

NBUK statement on NICE decision to fund Dinutuximab beta for children

Neuroblastoma UK has welcomed today’s announcement by the National Institute for Health and Care Excellence (NICE) that it has approved Dinutuximab beta for funding in the NHS.

Dinutuximab beta is used to treat high-risk neuroblastoma, a rare and life-threatening disease affecting around 50 children in the UK each year. In its initial assessment, published in April 2018, NICE had accepted that the clinical trial using Dinutuximab beta was effective in increasing survival for these patients, but that the treatment did not meet its incremental cost effectiveness ratio and that more data was required to assess its effectiveness. Unless the cost was reduced the treatment could not be funded in the NHS.

Following NICEs initial assessment NBUK has worked with EUSA, the manufacturer, NHS England and NICE, encouraging all sides to be flexible so that an agreement could be achieved.

Chair of Neuroblastoma UK, Tony Heddon said, "This is fantastic news for the families of newly diagnosed children and removes the uncertainty about the treatment they will receive. We would like to thank NHS England, NICE and EUSA, the drugs manufacturer, for their commitment to finding a solution that will enable children who are newly diagnosed with neuroblastoma, a life-threatening condition, access to this new treatment. I would particularly like to thank Dr Juliet Gray, Associate Professor in Paediatric Oncology, Southampton and Dr Martin Elliott, Consultant Paediatric Oncologist, Leeds, and Nick Bird who joined me in providing expert advice to the NICE appraisal committee.

Although understanding the rationale for the assessment of new drugs, we feel that NICE's current one size fits all approach needs review and that rare diseases, such as high-risk neuroblastoma, should be assessed as such, and either a higher financial threshold should apply or an alternative appraisal procedure used.”

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.

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  07939 507798

                                             Tony Heddon