Children with rare cancer given free access to treatment at home, after previously being forced to travel to US for $500,000 drug

Posted by author July 10, 2024 Travel

Children with rare cancer given free access to treatment at home, after previously being forced to travel to US for 0,000 drug

In short:

The Commonwealth will fund interim arrangements to make a medication to treat neuroblastoma freely available in Australia.

The drug was not available in Australia and required families to spend hundreds of thousands to pursue overseas.

What’s next?

Hospitals will be able to make the medication available almost immediately, as an interim measure while it awaits PBS approval.

Australian children and young adults with neuroblastoma, a cancer that affects the nervous system, will be given a desperately needed lifeline to access medicine previously only available by travelling overseas.

There are about 20 Australian children with high-risk neuroblastoma, which most commonly affects babies and children under 5 years old.

Last month, the ABC reported the story of two-year-old Luna, who had endured five rounds of chemotherapy, surgeries, a bone marrow transplant and immunotherapy as part of her treatment for the rare cancer.

Luna’s family and others had pleaded for access to a promising medication that could help to treat the cancer, but which was exorbitantly expensive and not yet approved in Australia.

The medication, known as DFMO (difluoromethylornithine), was approved in the United States at the end of last year, but because it is not available in Australia, some families were spending as much as $500,000 to pursue treatment in the US.

Other children missed out because they couldn’t afford treatment or were too sick to travel.

Health Minister Mark Butler announced on Wednesday morning after engaging with the drug maker Norgine over several weeks, the government had secured an undertaking that the company would provide DFMO to Australian patients for free, while it pursued local approvals for the medicine to be listed on Australia’s Pharmaceutical Benefits Scheme.

Mr Butler said until that compassionate access scheme is established, the government would fund major paediatric hospitals to provide DFMO to children for free.

“We understand that DFMO offers the only hope to some patients who are desperately ill from neuroblastoma,” Mr Butler said.

“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.”

About half of children diagnosed with neuroblastoma do not survive, and more than half of those who are successfully treated have recurrences that lower survival rates to just 5 per cent.

DFMO was found in trials to reduce the risk of neuroblastoma recurrence by 50 per cent, helping about three-quarters of children diagnosed to survive the disease.

‘Huge relief’ for uncertain families crowdfunding treatment

Kalee Abdollahi’s nine-year-old son Zai was diagnosed with high-risk stage 4 neuroblastoma in March last year.

He has been undergoing intense treatment ever since, including eight rounds of high-dose chemotherapy, major surgeries and a stem cell transplant.

Despite his positive response to the treatment, Ms Abdollahi said there was a 50 per cent chance he could relapse.

Kalee Abdollahi said the funding announcement was a huge relief.(Supplied)

So, alongside friends, they had been fundraising to get enough money to take Zai to the US later this month where he could access DFMO to decrease his chance of falling sick again.

“If Zai is to relapse, his survival rates are just 5 per cent,” she said.

Ms Abdollahi said today’s announcement was “life changing” and a “huge relief” because they would no longer need to travel to the US for the treatment, and instead could get help from their current doctors.

“Safe and affordable access to this drug is so important so I don’t need to risk Zai’s health by travelling overseas multiple times to access this drug,” she said.

“The announcement today just takes the pressure off us and the uncertainty of the next step for Zai’s treatment.”

Zai Abdollahi’s family had been crowdfunding to access medication to treat neuroblastoma.(Supplied)

Shadow Health Minister Anne Ruston said the passionate advocacy of families who had experienced the heartbreak of a diagnosis had led to today’s announcement.

“We recognise the many parents across the country who have fought for this outcome,” Senator Ruston said.

“The Coalition calls on the states and territories to now facilitate this treatment without delay.”

Posted 10 Jul 2024Wed 10 Jul 2024 at 1:49am, updated 10 Jul 2024Wed 10 Jul 2024 at 2:20am