Every Australian child and young adult with high-risk neuroblastoma will have free access at Australian hospitals to a promising medicine not otherwise available in Australia, known as DFMO, thanks to funding from the Australian Government.
Around 20 Australian children have high-risk neuroblastoma, which is a cancer which affects the nervous system and is most common in children and babies under 5 years old.
The medicine known as DFMO (difluoromethylornithine) was approved by the medicines regulator in the United States, the Food and Drug Administration, in December 2023.
It has been shown to reduce the risk of relapse and improve survival of patients suffering high-risk neuroblastoma.
This has led some families to seek treatment overseas in the U.S., which can cost up to $500,000, while other children have missed out because they couldn’t afford the treatment or were too sick to travel.
After engaging with the drugmaker Norgine over a number of weeks and advocating on behalf of Australian children and their families, the Albanese Government has secured an undertaking that Norgine will make DFMO available to Australian patients for free, while it pursues the necessary approvals for a PBS listing.
Until Norgine establishes its compassionate access scheme, the Albanese Government will provide funding to ensure that major paediatric hospitals across Australia can provide DFMO to every Australian child with high-risk neuroblastoma, for free and with no payment required.
The Commonwealth will provide funding to state and territory governments to support them to administer DFMO to eligible patients, free of charge, through the Drug and Therapeutics Committees of major public hospitals.
This will ensure that patients with high-risk neuroblastoma can obtain the medication without expense and overseas travel.
Around 20 patients are expected to benefit each year over the next two years.
The fully Commonwealth-funded scheme can begin immediately, as soon as the Committees of the major public hospitals approve the administration of DFMO in their hospitals.
The one-off funding will remove the financial barriers to DFMO access while the company’s compassionate access scheme is established and the application is considered by the Therapeutic Goods Administration.
The Government has committed to reimburse states and territories for the costs of purchasing the medicine for eligible patients and is further supporting treatment through the national public hospital funding agreement.
Quotes attributable to Minister Butler:
“The Government is committed to supporting the best health outcomes for all children and young adults, including those with life-threatening cancers.
“We understand that DFMO offers the only hope to some patients who are desperately ill from neuroblastoma.
“This one-off funding ensures that they can get this new and promising treatment, without the huge price tag, while proper approval processes are followed.
“I am delighted that the company sponsoring the medicine for use in Australia is also looking to establish a compassionate access scheme to provide DFMO to those patients in need.
“In the coming days, I will be writing to my state and territory health colleagues to ask them to consider providing DFMO on the basis of the Government’s commitment to reimburse them for the cost of purchasing this medicine.”
