Childhood Cancer Foundation is delighted to see that “Dinutuximab Beta”, an innovative immunotherapy drug for the treatment of High Risk Neuroblastoma, has been approved for reimbursement in Ireland. To date, this has only been available as part of clinical trials. This ground breaking drug will now become part of the frontline “standard of care” for Irish children with High Risk Neuroblastoma. This puts Irish children on a par with children receiving the most up to date front line treatments available across the world.
Childhood Cancer Foundation played a small role in this success. Working hand in hand with the Oncologists from St. John’s Ward in Our Lady's Children's Hospital Crumlin, we participated in the formal NCPE application process. The role of Childhood Cancer Foundation in this application was to provide the Patient Organisation Submission of Evidence. This was included in full in the final report to the HSE and helped to ensure a complete picture of the impact of this High Risk Neuroblastoma and the benefits of access to best in class medicines was presented to the decision makers. This is directly linked to one of our strategic goals which is to provide children in Ireland access to innovative medicines.
We must remember that none of this would be possible without the participation of Irish children in well controlled, internationally cooperative clinical trials - this is their win!
High Risk Neuroblastoma affects approximately 6 children a year in Ireland and generally affects children under 5. It is a cancer of the sympathetic nervous system and leads to cancer of the bone, bone marrow, blood, soft tissue and potentially the central nervous system.
The treatment regimen is recognised as one of the harshest used in the field of children’s cancer. It involves chemotherapy, surgery, stem cell transplant, radiotherapy and immunotherapy. Despite this, it has a significant rate of relapse with post relapse survival rates of less than 20%. Inclusion of this product in the treatment regimen has been shown to improve the rates of survival of children with High Risk Neuroblastoma.