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.
The announcement was welcomed with tears and cheers from the Cystic Fibrosis community who tuned into an live online announcement.
Busselton woman Jackie Hodson, who has been on an Orkambi compassionate scheme, credits the ‘life-changing’ drug with her health remaining stable over the last three years.
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.
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 expectancy.
The pair both attended the Cystic Fibrosis Australia live online announcement and broke down when the news they’d been hoping for became a reality.
Ms Barrett and Ms Hodson have become the faces, and some of the loudest voices, for the Orkambi campaign.
Ms Barrett even travelled to Sydney earlier this year to protest for the cause.
After the announcement, she was immediately asked for her reaction.
Wiping away tears, she and husband Adrian expressed their happiness and relief.
“That's amazing,” they said.
“I am lost for words. It is amazing for everyone and the people who will get it really, really soon,” Ms Barrett said.
“It is historic for our community, it is the start of great things, maybe children who are born now will be able to live with this disease.”
Ms Hodson spoke with the Mail shortly after the announcement.
“I’m so bloody relieved, I cried so much,” she said.
“I’m so happy it is over and done. I’m so happy for little Connor.
“Now we know for sure it is not going to go anywhere, and this may mean it will be a smoother process for the next new drugs which are coming out.”
Ms Hodson was on her way to media commitments in Bunbury and said she would have a glass of bubbles with Ms Barrett to celebrate.
“I think we’ve earned it,” she said.
“We’ve had so much support from the media and the community has really gotten behind us – we are so thankful.”
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.
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.
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.
Cystic Fibrosis Western Australia chief executive officer Nigel Barker said the August 17 decision was a culmination of years of advocacy by people whose lives had been impacted by Cystic Fibrosis to force an agreement between the government and Vertex, the manufacturer of Orkambi.
“We are ecstatic that the Australian Health System is supporting better health outcomes and quality of life for those with rare diseases, but we’re also concerned that lives have been put on hold and lungs have incurred irreversible damage whilst we struggle to get a fair deal, years behind approval in the USA and Europe,” he said.
“There has to be a better way to fast track the process of drug approval in the future.”
Cystic Fibrosis affects nearly 4,000 Australian children and adults.
Read more: Anxious wait for PBS results
