Friday 12 April 2024

Neuroblastoma Australia, together with the Australian and New Zealand Children’s Haematology and Oncology Group (ANZCHOG) and Australian paediatric oncologists, has written to request the federal government urgently consider providing interim funding for Australian children with high-risk neuroblastoma to access eflornithine, a recently approved US Federal Drug Administration (FDA) treatment.

Around 50 children are diagnosed with neuroblastoma in Australia each year, of which around half are classified as having high-risk neuroblastoma. This complex solid tumour accounts for a disproportionate number of childhood cancer deaths. In particular children with relapsed, high-risk neuroblastoma face low survival rates of between 10 – 20%.

Current treatment for high-risk neuroblastoma is prolonged, consisting of intensive chemotherapy, surgery, high-dose consolidation therapy, radiation therapy and immunotherapy. Reducing the rate of relapse for high-risk neuroblastoma is a crucially important avenue to improving survival rates.

Eflornithine, a drug that was approved by the FDA on 23^rd December 2023, has shown promising results in reducing the rate of relapse, and subsequently increasing survival for children with neuroblastoma. Analysis of international clinical trial data has shown that adding eflornithine to treatment for high-risk neuroblastoma significantly reduced the chance of relapse, in some instances by 50%, and improved survival for high-risk neuroblastoma over the first few years after treatment completion.  Based on these results eflornithine has been approved by the FDA to reduce the risk of relapse children with high-risk neuroblastoma in the United States.

Despite promising results internationally, eflornithine is not currently registered for use in Australia. A local pharmaceutical company is in the process of considering Australian registration and funding of eflornithine, however, registration is a time-consuming process which is likely to take at least a year. This leaves Australian families with current options of either not including eflornithine in their child’s treatment or fundraising to travel to the United States for treatment.

Eflornithine can be imported for individual Australian families using the Therapeutic Goods Administration Special Access Scheme.  However, the cost of treatment for an average size child is between $500,000 and $700,000, which is beyond the capacity for most families and health services to fund. Therefore, we have put an urgent request to the Health Minister proposing that the federal government funds local access to eflornithine for an interim period, until such time that it can be made available to Australian families through alternative means, such as TGA approval. Estimated costs include $5 million in funding in year one and a further $10 million in year two, dependent on when, and if eflornithine is registered in Australia. If the request is approved, it will mean every Australian family with a child with high-risk neuroblastoma will have equitable access to eflornithine, reducing the risk of relapse and improving survival rates for children with the disease.