Twenty three years – that’s the difference Cystic Fibrosis drug, Orkambi, would make to Bunbury’s Connor Barrett’s life.
The two-year-old’s parents, Taryn and Adrian, hope the life-changing drug will be approved by the Pharmaceutical Benefits Advisory Committee when they meet in July.
Currently, the drug isn’t on the Pharmaceutical Benefits Scheme and costs $250,000 a year for patients.
People with Cystic Fibrosis develop an abnormal amount of mucus within the lungs, making it difficult to breathe.
The life expectancy of children born with Cystic Fibrosis today is 37.
Ms Barrett said extensive trials showed Orkambi reduced hospitalisations by more than 60 per cent, lung damage by 40 per cent and, if taken by age six, will add 23 years to life expectancy.
She described the fight for the drug as frustrating, and one that shouldn’t be battled in a first-world country like Australia.
“It’s one we have to win because there are fears that this drug company may leave the country if its fourth submission to the PBAC fails,” she said
“The price negotiations process for Orkambi, lasting two and a half years already, has been deadly.
Every day children with Cystic Fibrosis are suffering irreversible damage.
“Our federal government will not pay the price to reduce their suffering and extend their lives and Vertex hasn’t yet shown its ability to negotiate fairly.
“Hopefully they will both come to their senses soon so our sons can remain blissfully unaware of the the gravity of this situation and our youngest son doesn’t have to endure preventable suffering.”
The family travelled to Sydney to protest for the cause on April 30.
The demonstration made national headlines and the family were interviewed on morning program Sunrise.
A spokesperson for the federal Department of Health confirmed Vertex Pharmaceuticals had made its fourth submission to the PBAC to list Orkambi on the PBS for the treatment of certain cystic fibrosis patients aged 12 years and over, plus its first submission for patients aged six to 11.
The outcomes from the July meeting will be made public on August 17.
