A Tarneit couple are hoping a new cystic fibrosis drug will be added to the Pharmaceutical Benefits Scheme sooner rather than later, prolonging the life of their son and the lives of hundreds of children battling the disease.
In December, the pharmaceutical benefits advisory committee recommended that Kalydeco be placed on the PBS and subsidised by the federal government. The drug, which costs a single user about $300,000 a year, has been found to significantly improve the lung function of cystic fibrosis patients aged six and older.
The committee will meet next month to decide when the drug should be listed and how much it will cost.
Brody, the son of Kate and Steve MacPherson, was diagnosed with cystic fibrosis in December when he was four weeks old. Doctors discovered he had the rare gene G551D, which does not allow salt and fluid to flow through his body.
The result is a build-up of sticky mucus in his lungs and digestive system.
About 8 per cent of cystic fibrosis patients in Australia, or about 200 people, have the gene. Mrs MacPherson said they knew something was wrong with Brody when he was constantly feeding and unsettled and wouldn’t put on weight.
But they never imagined it could be cystic fibrosis.
“It was out of the blue. We have no family history of cystic fibrosis. We knew nothing about it before he was diagnosed,” she said.
While Brody won’t be able to take Kalydeco until he is six, Mrs MacPherson hopes the government will subsidise it so the family can afford to buy it when the time comes.
“If we can keep him as healthy as possible for the next six years, there will be something to help him,” she said. “Kids can lead a normal life on Kalydeco..”
Cystic Fibrosis Australia chief executive David Jack said Kalydeco was the most important development in the treatment of the disease since the discovery of the cystic fibrosis gene in 1989.
He said he was confident that once pricing negotiations concluded, Health Minister Peter Dutton would deliver a listing date.
