Calls for “life-changing” drug to be added to the PBS
Cystic Fibrosis sufferers are calling for a “life-changing” drug to be added to the Pharmaceutical Benefits Scheme (PBS) so more of the 3500 affected Australians can access the treatment.
Trikafta, produced by Vertex, currently costs Aussies $300,000 a year, meaning it is out of reach for most, including Ella Sawyer’s daughter, Evie.
Diagnosed with the condition six weeks after she was born, Evie is now 12 and manages her condition with various medications, supplements, and twice-daily breathing exercises that help clear her chest.
She is prone to long-lasting infections, takes enzymes with every meal and has increased calorie needs.
Though Trikafta is already available and reimbursed in 17 countries, the decision to recommend its listing on the PBS has been deferred while experts on the Pharmaceutical Benefits Advisory Committee engage further with Vertex.
In the meantime, the drug is available free of charge to critically ill patients with no other medicinal options, subject to specific eligibility criteria and on request by their doctor.
Evie isn’t one of the 200 Australians who currently qualify, so she and her family are waiting for change.
“It’s really, really frustrating that if you’re born with CF [cystic fibrosis], your access to the best treatment can be limited depending on where you are in the world,” her mother Ella Sawyer said.
“If Ellie were to get the flu or another infection, she could have a big loss of lung function quite quickly whilst we wait for price negotiations to go on,” she added.
“It’s just very frustrating when the quality of someone’s life comes down to a price.”
The lack of access to Trikafta is putting lives at risk, according to Cystic Fibrosis Australia chief executive Nettie Burke.
“We talk about having one of the greatest health systems here in the world,” Ms Burke said.
“I think we do, but we can’t profess that when we can’t get drugs that have been in America for two years.”
Better quality of life
Ms Burke said recent trials of Trikafta have shown it to be a “life-changing” treatment.
“People’s lives have definitely been extended, but their quality of life has been increased dramatically as well,” Ms Burke said.
“One of the wonderful things we’ve seen from people overseas, or from those on trials, is that there’s a whole lot of babies being born.
“Before, that wasn’t necessarily going to be the case because people were too unwell to have a family, but we’ve seen a big increase in babies.
“So we know that Trikafta is working …. It’s incredible.”
While negotiations continue between the PBAC and Vertex, Ms Burke and families of people with cystic fibrosis are calling for compassionate access to Trikafta to be given to all eligible patients.
“We could save lives by getting access immediately,” Ms Burke said.
The next update from the PBAC is due in August.
Image: Nettie Burke / Twitter