‘The dial has shifted in the right direction, need to keep it pointed that way’, says CEO of Rare Cancers Australia

Pharma News: Fierce lobbying from politicians, including Senator Anne Ruston, Shadow Minister for Health and Aged Care, and organisations such as the Australian and New Zealand Children’s Haematology and Oncology Group (ANZCHOG), Australian paediatric oncologists, and Neuroblastoma Australia, has pressured the government into committing to fund childhood cancer therapy.

High-risk neuroblastoma presents a daunting challenge with survival rates as low as 10–20%, particularly for those experiencing relapses. The lengthy process of DFMO (eflornithine) registration and funding has forced families to either forgo the drug in treatment plans or embark on costly fundraising efforts to seek treatment overseas, where the cost ranges between $500,000 and $700,000 per child – figures well beyond the means of most families.

Following weeks of engagement with drugmaker Norgine, the Albanese Government has reached an agreement with the company to make DFMO available to Australian children for free, while it pursues the necessary regulatory approvals for a PBS listing.

Until Norgine establishes its compassionate access scheme, the government will provide funding to ensure that major paediatric hospitals across Australia can offer DFMO to every child with high-risk neuroblastoma.

The fully Commonwealth-funded scheme can begin immediately, as soon as the Drug and Therapeutics Committees of major public hospitals approve the administration of DFMO in their hospitals.

Lucy Jones, CEO at Neuroblastoma Australia, who tragically lost her daughter when she was just two and a half years old, commented, “This funding will ensure every child needing it here in Australia can access it for free, avoiding the need to fundraise hundreds of thousands of dollars and travel overseas with immuno-compromised children. This action represents a big step forward, recognising the need to be more agile and responsive in order to help children with rare cancers.”

Mark Butler MP, Minister for Health and Aged Care, stated, “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.

“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.”

Christine Cockburn, CEO of Rare Cancers Australia (RCA), welcomed the news, saying it would change the lives of many families burdened by this rare and ruthless childhood cancer, several of whom RCA directly supports.

“We are thrilled for the families. The fact that they will no longer have to fundraise to travel overseas and access this life-changing treatment for their children is an excellent outcome,” Cockburn stated.

“To see that human lives were prioritised over policy, and that the complexities of the system did not remain a barrier to interim access. This outcome is a great response and beacon of hope for so many cancer patients having to self-fund their treatments that aren’t available yet in this country.

“The dial has shifted in the right direction, we now need to keep it pointed that way,” she emphasised.

Analysis of clinical trial data has shown that adding DFMO to treatment for high-risk neuroblastoma significantly reduces the chance of relapse, in some instances by 50%, and improves survival over the first few years after treatment completion.

In reimagining healthcare across the entire patient journey, Health Industry Hub^TM is the only one-stop-hub uniting the diversity of the Pharma, MedTech, Diagnostics & Biotech sectors to inspire meaningful change.

The Health Industry Hub^TM content is copyright protected. Access is available under individual user licenses. Please click here to subscribe and visit T&Cs here.