1.1 Dinutuximab beta is recommended as an option for treating high-risk neuroblastoma in people aged 12 months and over whose disease has at least partially responded to induction chemotherapy, followed by myeloablative therapy and stem cell transplant, only if:
they have not already had anti-GD2 immunotherapy and
the company provides dinutuximab beta according to the commercial arrangement.
1.2 This recommendation is not intended to affect treatment with dinutuximab beta that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop. For a child or young person, this decision should be made jointly by them or their parents or carers and their clinician.
Why the committee made these recommendations
Neuroblastoma mainly affects children and young people. Treatments for high-risk neuroblastoma include chemotherapy, radiotherapy, stem cell transplant, surgery and isotretinoin. Dinutuximab beta is an important, potentially curative option for maintenance treatment of the disease.
An indirect comparison with isotretinoin suggests that dinutuximab beta increases survival and the length of time before the disease progresses, compared with current treatment.
Dinutuximab beta does not meet NICE's criteria for a life-extending treatment at the end of life. Also, the range of cost-effectiveness estimates presented is higher than what NICE usually considers a cost-effective use of NHS resources. But taking into account the uncaptured health-related benefits, the rarity and severity of the disease and the potential lifetime benefit for children with neuroblastoma, dinutuximab beta can be recommended for high-risk neuroblastoma.
Dinutuximab beta also has a marketing authorisation to treat relapsed or refractory disease. This indication was not considered in this appraisal because it is not relevant to current NHS practice; most people with relapsed or refractory disease have already had dinutuximab beta.