Orkambi, the revolutionary Cystic Fibrosis drug which treats the disease’s underlying cause, has been recommended by the Pharmaceutical Benefits Advisory Committee to be listed on the Pharmaceutical Benefits Scheme.
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.
Prior to being on the PBS, it cost $250,000 a year for patients.
Busselton woman Jackie Hodson, who has been on a Orkambi compassionate scheme, credits the ‘life-changing’ drug with her her health remaining stable over the last three years.
“I don’t get as as sick as often and when I do get sick I recover quicker,” she said when talking to the Mail in May.
“It’s closest thing we have to a cure, you are not taking an antibiotic treating you for being sick, it’s fixing the problem. It is the first drug to treat the underlying cause and not just the symptoms.
She, along with Bunbury mother Taryn Barrett, have been long-time campaigners for the drug to be made accessible to 1300 Australian’s living with Cystic Fibrosis who are eligible for the medication.
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.
Ms Barrett’s three-year-old son Connor has Cystic Fibrosis.
It is estimated if Connor was to begin taking Orkambi now it would add 23 years to his life.
In May, Jackie Hodson and Taryn Barrett spoke to the Mail about Orkambi going before the Pharmaceutical Benefits Advisory Committee in July.
At the time, Ms Barrett described the fight 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.”
Although Ms Hodson had access to the drug, she was still in limbo, unsure of how long Orkambi would be provided to her – especially if it was again refused for the PBS.
“If the government denies it again, what’s the point in the company even making it. They may question whether they should bother trying if governments don’t approve it,” she said.
In October, Ms Hodson turns 27, the life expectancy for people in her generation with Cystic Fibrosis.
This November, she marries the love of her life, Aidan Fraser.
She said reaching the milestone of 27 would bring up mixed emotions for the pair.
“It will be good to have made it, I’m just hoping to keep going,” she said.
“I think when I turn 30, that will feel really good.
“Ultimately, it is what it is. We don’t get anywhere by dwelling on it. It is hard to see what other couples are going through, we just have to hope that it’s not going to be us.”
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