Every Australian child and young adult with high-risk neuroblastoma will continue to have free access at Australian hospitals to a promising medicine not otherwise available in Australia, known as DFMO, thanks to an agreement secured from the Albanese Government.
DFMO has been shown to reduce the risk of relapse and improve survival of patients suffering high-risk neuroblastoma.
Around 50 children are diagnosed with neuroblastoma in Australia each year, of which around half are classified as having high-risk neuroblastoma. The cost of treatment using DFMO ranges from $500,000 to $700,000.
Through time-limited funding, the Albanese Government has been reimbursing state and territory governments for the cost of buying the medicine. As of today, this funding has been replaced by new arrangements that will ensure eligible patients, new or existing, continue to have free access to the drug.
The Government provided the interim funding after Norgine, the supplier of DFMO in Australia, undertook to establish what is called an Expanded Access Program (EAP), while it pursues the necessary TGA approvals and PBS listing.
Through the EAP, which starts today, Clinigen on behalf of Norgine will continue to provide access to DFMO at no cost to patients.
To access the EAP, treating clinicians register their patients online. Any decision to prescribe DFMO remains one for the treating clinician, in line with the patient's existing treatment.
DFMO has been approved by the medicines regulator in the United States, the Food and Drug Administration. In Australia, the TGA has designated it as an 'orphan' drug, under a special program to develop and give access to drugs for small numbers of patients - such as children and young adults with the rare neuroblastoma cancer - which would otherwise not be financially viable for a sponsor.
Quotes attributable to Minister Butler:
"No child and family suffering through a high-risk neuroblastoma diagnosis will have to worry about receiving the world's best treatment.
"This is about giving families peace of mind during the toughest fight.
"It is a priority for the Government to ensure all patients who accessed DFMO through our reimbursement program will continue to receive it through the new EAP.
"All patients currently accessing the reimbursement program will be transferred to the EAP once enrolled by their treating practitioner.
"The TGA is in the process of evaluating an application from Norgine to register DFMO for the treatment of high-risk neuroblastoma on the Australian register of therapeutic goods.
"Norgine will pursue the necessary approvals for a PBS listing via the expert, independent Pharmaceutical Benefits Advisory Committee."
