‘Transformational’ cystic fibrosis drug expanded on PBS

From 1 July, elexacaftor/tezacaftor/ivacaftor and ivacaftor (sold as Trikafta) will become accessible for more Australians living with cystic fibrosis (CF), thanks to its expansion on the Pharmaceutical Benefits Scheme (PBS).
 
The medication’s listing will be expanded to include patients with CF aged two years and older who have at least one mutation in the CF transmembrane conductance regulator (CFTR) gene and are responsive to treatment.
 
The expansion means more than 90% of the 3800 Australians living with CF now have access to a CFTR modulator.
 
It is a move expected to benefit hundreds of patients, with those diagnosed with rarer mutations of CF now able to access significantly cheaper Trikafta.
 
Cystic Fibrosis Australia CEO Jo Armstrong described the expansion as a ‘bold step forward’.
 
‘For those who’ve waited, hoped, and campaigned, this is more than a policy decision, it’s a transformational outcome,’ she said.
 
‘This decision means that hundreds of Australians living with CF will now have access to a therapy that was previously out of reach, a treatment that can dramatically improve quality of life and life expectancy.’

Trikafta was first listed on the PBS in April 2022 for patients aged 12 years and older who have specific gene mutations, and has since been expanded several times.
 
Without the PBS subsidy, patients can pay more than $250,000 for a year of treatment.
 
With the rollout CF medications, including Trikafta, as well as care and treatment options, the life expectancy for Australians with CF has risen from 47 to 60 years in the past two decades.
 
Federal Health and Ageing Minister Mark Butler described Trikafta’s expansion as ‘great news’.
 
‘It’s a wonder drug, it literally works within 24 or 48 hours to transform a person’s life, but it costs about $250,000 a year,’ he said.
 
‘Expanding this for another 200 or so Australians means that almost 80% of people living with cystic fibrosis now have access to this drug through the PBS.
 
‘Research is going on to make sure that, if at all possible, we can get 100% of cystic fibrosis patients in the same position that other patients have now, which is getting back to school, getting back to work and living a normal life.’
 
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