NICE Technical Assessment of Dinutuximab beta

The National Institute for Health and Care Excellence (NICE), which is responsible for providing evidence-based guidance on which treatments and drugs will be available in the NHS, is about to undertake a technical appraisal of APN311 (Dinutuximab beta) immunotherapy for treating high-risk neuroblastoma following myeloablative therapy and autologous stem cell transplant.

Following a number of interviews with parents whose children have been affected by neuroblastoma, Tony Heddon, NBUK Trustee, will be attending the NICE appraisal meetings as a patient expert, to represent their views.

The outcome measures to be considered include overall survival, progression free survival, adverse effects of treatment, health-related quality of life and the cost effectiveness of the treatment. The appraisal is expected to reach a decision in May 2018. In order to be provided on the NHS, Dinutuximab beta needs to be approved by NICE. It has already been approved by the European Medicines Agency and in the United States, where a similar antibody is available.

APN311 is a chimeric monoclonal antibody that targets GD2, a glycolipid overexpressed in certain tumours such as neuroblastoma. The immunotherapy drug recognises cells expressing a receptor (GD2) on their surface, and signals for the immune system to destroy these cells. Because the drug has to make contact with tumour cells, this immunotherapy is thought to be suited to mopping up small populations of residual cells rather than penetrating the primary tumour or large metastases. Hence immunotherapy is used to help eradicate any residual disease, and hopefully increase the chances of keeping children in remission.

APN311 has been available in the UK over the last few years as part of the SIOPEN (an international research collaboration) High Risk and Long Term Infusion trials. Patients with high risk disease who have achieved a sufficiently good response at the end of surgery, intensive chemotherapy and stem cell transplant, and have completed this treatment within specified time lines, have been eligible to receive the anti-GD2 antibody as part of the High Risk study. Those children who haven’t been able to receive the antibody through the High Risk study, including those children with relapsed neuroblastoma, have generally been eligible to receive antibody through the Long Term Infusion study.

The SIOPEN Long Term Infusion study closed in January 2017, and the High Risk Trial closed to recruitment of new patients in June 2017. While the NICE process for Dinutuximab beta evaluation is underway, immunotherapy will continue to be provided within the High Risk trial to patients already enrolled who respond well enough to receive the antibody as part of the trial.

Guy Blanchard, Chair, NBUK, stated “It is vital for UK patients that the provision of anti-GD2 immunotherapy is not interrupted so that children in the UK benefit from the latest therapies in the same way as those in Europe and the US. Immunotherapy is a rapidly evolving research field and it is hoped that improvements in it, alongside other drugs, will in the near future improve long-term survival for neuroblastoma patients.”