People with cystic fibrosis and their loved ones are nervously waiting to discover if a ‘game changing’ treatment, which now costs $250,000 a year, will be put on the Pharmaceutical Benefits Scheme later today.
It is the fourth time the treatment, Orkambi, has been considered for subsidy on the PBS, with the Pharmaceutical Benefits Advisory Committee and creator Vertex unable to reach a price agreement on three previous occasions.
Carissa Marheine, who studies psychological science at Latrobe in Wodonga, has spent much of her 18 years in and out of hospital with the condition.
She said it’s simply not fair a treatment is available but inaccessible to many.
“I don’t know why it hasn’t been put on the PBS sooner,” she said. “It’s really able to help people. It shouldn’t take four times to be approved.
“It doesn’t make sense and it’s not fair on people who need it – it will make people live longer.”
Miss Marheine, 18, said she was diagnosed with cystic fibrosis as a baby and was lucky enough to enter a drug trial six years ago of Ivacaftor, which she calls her ‘miracle drug’.
Ivacaftor, which is on the PBS, is made by the same company that produces Orkambi.
“It’s a genetic condition which affects your lungs, digestive system and sweat glands,” she said.
“It affected my breathing, you have a build up of mucus in your lungs and pancreas. I do pyshio and take antibiotics and enzymes for pancreatic insufficiency whenever I eat anything.
“I used to have regular hospital admissions, normally for about 10 to 14 days on constant antibiotics and high insensitivity physio.
“It’s a bit different for me now because I’m on the miracle drug but every three or four months I used to have to go to hospital and travel to Melbourne – it was hard.”
Miss Marheine explained Orkambi treats the mutation which most commonly causes cystic fibrosis so would help a large amount of people in the same way Ivacaftor successfully treats her.
“It kept me out of hospital, I’ve gone 12 months without admission. It’s the longest I’ve gone without going to hospital in my life,” she said.
Miss Marheine said being out of hospital has allowed her to embrace life more, which others with cystic fibrosis who need Orkambi should be able to also do.
“I’m able to breathe a lot better, I don’t have to take as many medications and it’s kept me out of hospital what has been great,” she said.
“I’ve got more time to study spend time with family and look after animals.”