Cystic fibrosis medication expanded on PBS

Children as young as two years old with cystic fibrosis (CF) will soon have greater access to treatment, with elexacaftor/tezacaftor/ivacaftor and ivacaftor (sold as Trikafta) to be expanded on the Pharmaceutical Benefits Scheme (PBS).
 
On Tuesday, it was announced the medication will be heavily subsidised for those aged between two and five years old who have at least one F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.
 
The subsidy will begin on 1 August and will see its cost drop from more than $250,000 each year to $31.60 per prescription.
 
Ashley Hayes, whose four-year-old son Heath lives with CF, said for her family, the change ‘means hope’.

‘Hope that every time he coughs, we don’t need to worry, “is two weeks in hospital coming? Do we need to do more treatment?”,’ she said.
 
‘Hope that no family will have to sit, when they have a newborn like Heath was, and worry will they live a long life.
 
‘We could never have imagined that we would be looking at a life where we could take three tablets a day.’
 
Trikafta was added to the PBS in May last year for children aged between six and 11 years old, but GPs can now prescribe the subsidised, ‘lifechanging’ medication much earlier.
 
With its expansion, it is expected to benefit hundreds of children each year, including some who will gain access to a modulator for the first time.
 
Cystic Fibrosis Australia Chief Executive Jo Armstrong said the benefits of adults and older children using Trikafta have already been proven, and now younger patients will be able to reap its rewards.
 
‘Access to early intervention is critical for young children,’ she said.
 
‘We know that it does slow the disease progression and improves the trajectory so that, ultimately, children will have longer, healthier lives as a result.
 
‘Trikafta is a very expensive medication, and it is not accessible without it being listed on the PBS, so today’s announcement means that it will be affordable for families, which is great news.’
 
CF is the most common, life-limiting genetic condition affecting Australians, with more than 3730 currently diagnosed.
 
And that number is continuing to rise, with the peak body revealing a ‘notable increase’ in diagnoses, up from 3446 around five years ago.
 
Health and Aged Care Minister Mark Butler said he was ‘delighted’ with the medication’s expansion.
 
‘It’s not unusual for a drug to go through a process of demonstrating efficacy and cost effectiveness for different cohorts and have the listing extended in a progressive way,’ he said.
 
‘[Trikafta] allows these children to go on to live long, happy lives and to enjoy their childhood, in the same way all their other school friends, who don’t have cystic fibrosis, are able to do.’
 
The expanded medication was listed on the PBS following a recommendation from the independent Pharmaceutical Benefits Advisory Committee.
 
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