Wonder kids

By Rebekah Devlin

*Editor’s note – since this article was first published, Trikafta is now available for hundreds more children living with cystic fibrosis in Australia. As of 1 May, 2023 the drug has been added to the Pharmaceutical Benefits Scheme for eligible children age 6 and up reducing the cost from upwards of $250,000 to just $30 a script.  

Cystic fibrosis is the most common life-threatening genetic conditions in Australia, impacting about 4000 Australians.

It causes bodily fluids to become thick sticky mucous affecting many organs, but especially the lungs and pancreas, clogging them with thick mucus. The mucus blocks the tiny air passages in the lungs, trapping bacteria and leading to repeated infections. It can make breathing very difficult. The lungs become irreversibly damaged by repeated infections and blockages.

The pancreas is also unable to release enzymes needed to digest food.

And for those living with CF – the realities are stark.

The median life expectancy is just 47 years.

Traditional treatments can require daily use of a ventilator and up to 80 tablets to be taken each day. All this in addition to intensive physiotherapy to remove the mucus from the lungs.

Up to eight per cent of people with cystic fibrosis will require a lung transplant, with 70 per cent of those transplanted living for more than five years.

While the gene responsible for CF has been identified, currently there is no cure.

In Australia, all babies are screened at birth for CF – and one in 2500 are born with the condition. More than one million Australians are carriers of CF genes.

Miracle medicine Trikafta is changing lives in the CF community.

Developed on the back of other CF modulators including Orkambi and Symdeko, Trikafta is a new triple combination modulator therapy that addresses the cause of CF.

It was given FDA approval in the US in October 2019 for people aged 12 and older who have at least one F508del mutation.

For many people, the results have been life-changing, and as a close to a cure for cystic fibrosis as is currently possible.

So Cystic Fibrosis Australia went to work to bring the drug here.

“We believe that all people with CF should have the opportunity to lead full, productive, and valuable lives with access to the best treatments and high-quality clinical care,” says CFA chief executive officer, Jo Armstrong.

“One of the critical ways we support the community is through our relentless advocacy, research, and clinical improvement funding. CFA is committed to improving clinical practice and patient outcomes and experiences.”

In Trikafta’s case, it meant lobbying the Federal government and decision makers behind the Pharmaceutical Benefits Scheme to ensure the drug was subsidised and not financially inaccessible for families.

And they won!

In March, Trikafta was made available on the PBS for people aged 12 and over, who have at least one F508 Del mutation, which is the most common mutation.

“Without PBS subsidy, around 1,900 Australians would pay more than $250,000 a year for access to Trikafta,” then Health Minister, Greg Hunt, said at the time. 

“This treatment is out of reach for most Australians, and now that it will be available on the PBS, patients will only pay a maximum of $42.50 per script, or as little as $6.80 with a concession card.”

While this was cause for huge celebration for the CF community, the strict guidelines around age limits and gene mutation meant that many missed out on accessing the wonder drug, including children aged six to 11.

“This was a great step, but we know, there are many more people who have the potential to benefit from this medication who are yet to have access to it,” Jo says.

“We have already seen and heard the amazing impact Trikafta has had on the lives of those who are taking it in the 12-plus age group, and now we want more people to benefit. The stories are incredible and bring hope to the community.

“We know that the sooner the listing is made, the sooner young Australians with CF can benefit. Prevention is critically important, especially to minimise damage to the lungs. “

And so CFA is gearing up for the fight once again.

Trikafta for six to 11-year-olds may be on the horizon

Internationally, Trikafta has already been made available to children aged 6 and over and CFA wants Australia to follow suit.

Trikafta has been included on the November Pharmaceutical Benefits Advisory Committee (PBAC) agenda for this age group – a key step in it being subsidised here in Australia.

In late September, submissions to the PBAC were closed and more than 270 submissions are now being considered, including one from Cystic Fibrosis Australia.

“As the peak consumer body for Australians living with cystic fibrosis, it is our role to lead this conversation and ensure all Australians get access to the medications they require – without delay,” Jo says.

While the breakthroughs with Trikafta are life-changing, there remain many people in the CF community who will not respond to it or may not be eligible to access it.

“This means that our work must continue so that everyone has access to the medicine and therapies they need to have a full life,” Jo says. “We invest in research, national Standards of Care, Peer Review, advocacy, and the Australian Cystic Fibrosis Data Registry to help support all Australians with CF.”

Cystic Fibrosis Australia is committed to improving clinical practice and patient outcomes with the aim of extending life expectancy from 47 to 55-years by 2026.

“Increasing life expectancy and quality of life is central to our work at Cystic Fibrosis Australia and we will be relentless to achieve this important goal,” Jo says. “Every day we strive to do all we can so that people living with cystic fibrosis can have a full and meaningful life.”

For more information on the work of Cystic Fibrosis Australia, head to https://www.cysticfibrosis.org.au/

Read ‘Our miracle Trikafta girl’ article here.