BUNBURY families with children diagnosed with cystic fibrosis will have to pay $250,000 a year if they want to access a revolutionary new drug that improves the condition’s symptoms.
A fortnight ago the Pharmaceutical Benefits Advisory Committee announced a decision not to fund the new drug Orkambi.
The life-threatening genetic disease affects the lungs and digestive system with no current cure.
An open letter written by Cystic Fibrosis Australia CEO Nettie Burke said clinical trials of the drug provided solid evidence of improved lung function and reduced hospitalisation.
The drug is currently available in other nations for patients aged 12 and over with trials starting on children with cystic fibrosis aged six and over. Without the PBS, the drug costs users up to $5000 a week.
Bunbury parents Adrian Knapinski and Clare Yearsley said Orkambi promised a brighter future for their daughter whose daily treatment includes 25 doses of medicine.
“When you have a child with a chronic illness, you try your best to have faith in medicine,” Mrs Yearsley said.
“But what good are these advancements if the federal government then denies access?”
Fellow Bunbury parents Adrian and Taryn Barrett said the decision has them reconsidering future options for their son Connor who is nine months old.
“When Connor was diagnosed we were ready to jump on a plane and find the best care but were relieved to discover that was in fact Australia,” Mrs Barrett said.
“Considering Orkambi is widely accessible in America, now we are not so sure.
“It’s time for Federal Member for Forrest Nola Marino to stand up and help people with cystic fibrosis in her community by letting the government know this is not acceptable.”
Ms Marino told the Mail decisions made by the PBAC are done with the best advice and professional opinion available and it would not be appropriate to ignore the experts.
Orkambi will be resubmitted to the PBAC with more information later this year.