A Busselton woman with Cystic Fibrosis and a Bunbury mother, whose son is living with the disease, have expressed their nerves ahead of Friday’s long awaited decision on whether a ‘life-changing’ drug will be made accessible.
In May, Jackie Hodson and Taryn Barrett spoke to the Mail about the drug, Orkambi, going before the Pharmaceutical Benefits Advisory Committee in July.
It was Vertex Pharmaceuticals’ fourth submission to the PBAC to list Orkambi on the Pharmaceutical Benefits Scheme for the treatment of certain Cystic Fibrosis patients aged 12 years and over.
The pharmaceutical company also made its first submission for patients aged six to 11 years.
As the drug isn’t on the PBS, it costs $250,000 a year for patients.
Ms Hodson and Ms Barrett have been vocal advocates for the cause, and as the public announcement draws closer, both again shared their feelings.
Ms Barrett’s youngest son, two-year-old Connor, has Cystic Fibrosis.
She said she was incredibly anxious as the decision loomed.
“It is hard to know what outcome will be,” she said.
“It is sounding a bit more positive this time around – Vertex has agreed to reduce the price and the minister has made some encouraging comments but we can’t get out hopes up too high.”
Ms Hodson is on a compassionate scheme to take the revolutionary drug.
She said she was anxious but hopeful the PBAC would grant others access to Orkambi, something she said would be a ‘dream come true’.
“Orkambi is the closest thing we have to a cure for Cystic Fibrosis and it is the only medication developed that treats the underlying cause, rather than just treating the symptoms of this dreadful disease,” she said.
Although she has access to the drug, Ms Hodson is still in limbo, unsure of how long Orkambi will be provided to her – especially if it is again refused for the PBS.
She expressed her fear of it being rejected for a fourth time.
“I’m scared to think of the sense of responsibility I’ll feel to continue the fight for Orkambi,” she said.
“(Being on the compassionate scheme) is an opportunity that comes with both gratefulness and frustration, that the other eligible 1300 people living with Cystic Fibrosis aren’t being given the same chance at life.”
People with Cystic Fibrosis develop an abnormal amount of mucus within the lungs, airways and the digestive system
This causes impairment of the digestive functions of the pancreas and traps bacteria in the lungs resulting in recurrent infections, leading to irreversible damage.
Lung failure is the major cause of death for someone with Cystic Fibrosis.
In an emotional Facebook post, the 26-year-old shared her heartbreak for the parents who had lost children to the disease, and for children who currently weren’t able to access the drug, who ‘may never know what grey hair is because the Australian government wouldn’t pay the price to improve their health’.
“Above all, I’ll be shattered that once again the Australian government will have failed us and the thousands of other chronically ill Australians waiting for access to treatment options. The people that deserve a chance. Those who are hoping with everything they have, that a positive decision comes before health deterioration and ultimately death,” she wrote.
Her post has already been shared more than 40 times.
The PBAC is expected to make the announcement between 10am and 1pm on August 17.
