A new initiative aims to support children and young adults with high-risk neuroblastoma, a rare cancer affecting the nervous system predominantly in children under five years old. 

Through new funding, patients will receive free access to the promising medicine DFMO (difluoromethylornithine) in Australian hospitals. 

Neuroblastoma affects approximately 20 children annually in Australia. 

This form of cancer poses a high risk of relapse and has driven families to seek treatment overseas, particularly in the United States, where DFMO was approved by the Food and Drug Administration in December 2023. 

Treatment costs can escalate up to $500,000, a prohibitive expense for many.

The Australian Government has negotiated engagements with Norgine, the pharmaceutical company responsible for DFMO. 

It says it has successfully secured an agreement from Norgine to provide the medication free of charge to Australian patients while the company seeks approval for a Pharmaceutical Benefits Scheme (PBS) listing. 

Until Norgine's compassionate access scheme is operational, the government will ensure major paediatric hospitals can administer DFMO without any cost to patients.

Funding from the Commonwealth will be allocated to state and territory governments to support the administration of DFMO through the Drug and Therapeutics Committees of major public hospitals. 

This funding will facilitate immediate access to the drug, eliminating the need for overseas travel and associated costs.

The government's one-off funding will cover costs until the compassionate access scheme is in place and the Therapeutic Goods Administration reviews the PBS application.