Family’s joy at access to drug for son

A Bunbury mother says she has more hope for her son’s future following a recommendation to list a life-changing drug for children aged two to five with cystic fibrosis on the Pharmaceutical Benefits Scheme.

The Pharmaceutical Benefits Advisory Committee’s recommendation last week to fund Orkambi, a $250,000-a-year drug, could give Taryn Barrett’s four-year-old son Connor 23 extra years of life.

It comes after the drug was made available for ages six and up a year ago, when Mrs Barrett and her family of five vowed to continue their arduous and inspiring fight.

The drug had been rejected for funding three times before a stalemate over its price between the Commonwealth’s medicines regulators and the drug company Vertex finally came to an end.

Mrs Barrett said the family were “ecstatic” and full of emotions following last week’s milestone.

“Last year was obviously the biggest hurdle, to get it funded for those aged six plus after all the delays,” Mrs Barrett told the Bunbury Herald.

“Now that it’s going to be available for Connor in the next few months, that’s just really exciting and we’re a lot more hopeful for his future now.”

Mrs Barrett has been lobbying to get access to Orkambi for years.

“Next year Connor will start kindergarten, and just to know that he’ll be able to have this medicine that’ll just try and stabilise and slow the disease down when he’s taking that first big step into, I suppose the real world, that’ll be just peace of mind,” she said.

“Ultimately, hopefully he’ll get to grow old.”

She said while she was happy for Connor, it was also disappointing others had “died waiting” for access to the drug.

“That’s been pretty frustrating at times ... the fact that it took so long to get through, that was really distressing but it was worse for the families and for the people where the disease was more progressed,” she said.

“Some of them died waiting, so I’m so, so happy for Connor and the 180 other children that need Orkambi while they’re young but it’s really, really disappointing that it took so long.”

Orkambi could help stop irreversible lung damage in more than 1000 Australians with the most common mutation of cystic fibrosis.

About 180 of them are children aged two-five.

“Hopefully it will slow the disease now and we can wait for even bigger breakthroughs to come and ultimately it could be the start of it becoming a manageable disease for the children who are born now,” Mrs Barrett said.

Mrs Barrett vowed to continue fighting, with a similar drug — Symdeko — for people who have cystic fibrosis but a different gene mutation, being in pricing negotiations for more than six months.

“Orkambi is fantastic, but it’s not a cure, so we’re still going to keep fighting for research and hopefully there’ll be an even bigger breakthrough in the very near future,” she said.