Government seals deal with industry for rare cancer therapy

Pharma News: Australian children and young adults diagnosed with high-risk neuroblastoma will continue to receive free access to a promising, life-saving medicine, DFMO, under a new arrangement secured by the government.

Neuroblastoma, a rare cancer affecting around 50 children in Australia each year, presents a particularly high-risk variant in about half of those cases. Analysis of international clinical trial data has shown that adding DFMO 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.

The cost of DFMO treatment, which ranges from $500,000 to $700,000, has placed an enormous financial burden on families. However, with the government’s continued support, eligible patients will be able to access this drug free of charge.

Initially, the government provided interim funding to cover the cost of DFMO, reimbursing state and territory governments while Norgine worked to establish an Expanded Access Program (EAP). This program, which began yesterday, allows Clinigen, on behalf of Norgine, to provide the drug at no cost to patients.

Mark Butler, Minister for Health and Aged Care, reaffirmed the government’s commitment saying, “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.”

Under the new arrangement, treating clinicians can now register their patients online to access the EAP. The decision to prescribe DFMO remains with the clinician, integrated into the patient’s existing treatment plan.

DFMO has already received approval from the U.S. Food and Drug Administration (FDA) and, in Australia, the Therapeutic Goods Administration (TGA) has designated the drug as an ‘orphan’ medicine. The TGA is currently reviewing Norgine’s application for DFMO to be formally registered on the Australian Register of Therapeutic Goods (ARTG).

While the drug works its way through the approval process, Minister Butler assured that all patients currently receiving DFMO will be seamlessly transferred to the new EAP.

“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,” Minister Butler confirmed.

Norgine also plans to seek approval for listing DFMO on the Pharmaceutical Benefits Scheme (PBS), a move that would further broaden access to the treatment in the future.

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