Pharmaceutical Benefits Scheme gives better access to cystic fibrosis drug Trikafta

West Australian

5 hours ago

More Australians living with rarer types of cystic fibrosis will be able to access treatment for the first time when expanded rules to the Pharmaceutical Benefits Scheme kick in next week.
From July 1, people who have at least one mutation related to the genetic disorder will have access to Trikafta on the PBS.
The drug, which would usually cost upwards of $20,000 a month, has been a life-changing therapy for people living with cystic fibrosis since it was first put on the PBS in 2022.
Almost 4000 people live with cystic fibrosis across Australia. It's a genetic disorder caused by mutations in the gene that regulates the movement of salt in and out of the cells.
It causes a thick, sticky build-up of mucus in the lungs and other organs. Over time it causes irreversible damage to the lungs, limiting the ability to breathe and increasing infection risk.
Trikafta has proven highly effective in treating cystic fibrosis by improving the flow of salt between cells, in turn improving lung function and breathing.
The expanded listing includes a further 271 mutations, giving more Aussies with rare types of cystic fibrosis access to the drug.
Among those rare cases is four-year-old Koah Hart, who has one of the rarest mutation combinations in WA.
The expanded eligibility criteria, in force from next Tuesday, includes both of his rare mutations.
Mum Brittney Hart, 27, said she was shocked to learn both were included.
'Seeing the news this morning, I honestly had tears in my eyes,' she said.
The mother-of-two said she and others in the cystic fibrosis community have advocated tirelessly to give those with rarer types of the disorder the same chance for treatment as those with more common mutations.
'We've worked so hard for this, to give my boy an extra shot at life. It's really exciting. I did cry a little bit,' she said.
'The outcomes and what it improves is . . . I have no words. It really honestly just gives us more hope that these young kids will reach their old age and reach their full potential.'
Koah was diagnosed at just four-weeks-old, two weeks before Christmas 2020 — the height of the COVID-19 pandemic. He's had six hospital admissions since.
'It was very isolating at the beginning, and having an immunocompromised newborn (during COVID) was really scary,' Ms Hart said.
The superhero-obsessed little tyke currently takes between eight and 10 tablets a day to manage his condition.
'His meds are called his superhero meds, and when we go for an admission it's his superhero (IV) line,' Ms Hart said.
She hopes Trikafta will reduce that number, as she's seen in other families.
'At the beginning of Koah's diagnosis we said we wanted quality of life over quantity, but this gives us hope for the best of both worlds,' she said.
The latest listing change is the third expansion since 2022. Those eligible will only have to pay $31.60 per script, or $7.70 with a concession card.
That will drop to $25 a script when the reduced PBS co-payment begins on January 1, 2026.
Cystic Fibrosis WA chief executive Lisa Bayakly said it was a 'great advocacy outcome for people with (cystic fibrosis) across Australia'.