Local cystic fibrosis sufferers now have access to life-changing treatment following the landmark listing of Trikafta on the Pharmaceutical Benefits Scheme (PBS).
On April 1, Trikafta became available on the Pharmaceutical Benefits Scheme (PBS) for cystic fibrosis sufferers aged 12 years and older who have at least one common disease mutation.
Minister for Health and Aged Care, Greg Hunt, said the listing of Trikafta was life-changing for many Australians with cystic fibrosis and their families.
“Without PBS subsidy, around 1,900 Australians would pay more than $250,000 a year for access to Trikafta,” Hunt said.
“This treatment is out of reach for most Australians, and now that it’s available on the PBS, patients will only pay a maximum of $42.50 per script, or as little as $6.80 with a concession card.
“This listing will make access to treatment within reach for thousands of Australians with cystic fibrosis each year.”
A 25-year-old Newcastle woman, Meghann Gibbs, who suffers from cystic fibrosis, said that Trikafta worked towards correcting defects associated with the disease, allowing her to lead a “relatively normal life”.
“Trikafta is a big step up. There’s no comparison to the drug I’m on now,” Gibbs said.
“While my current drug has allowed me to remain healthy, Trikafta is almost like a path towards normality.”
Cystic fibrosis is a progressive genetic disease that causes persistent lung infections.
The condition is caused by genetic defects that result in a build-up of mucus in the lungs, pancreas and other organs.
Over time, cystic fibrosis limits the ability of breathing and makes sufferers more susceptible to infection.
Trikafta works by improving the flow of chloride and water in patients with a specific genetic defect, resulting in improved lung function and breathing.
Without medication, Gibbs said it was difficult to get out of bed in the morning due to shortness of breath and coughing fits.
“Some mornings, I throw up just from the severe exertion,” she said.
Gibbs said her symptoms limited her ability to work in an office and attend social engagements, such as breakfast with friends.
“People aren’t sure from your physical appearance that you’re suffering … but there’s difficulty in everyday tasks that a normal person doesn’t experience,” Gibbs said.
“Cystic fibrosis for a person with normal respiratory capabilities is compared to breathing through a straw with every breath.”
Gibbs said sufferers spend hours doing airway clearance exercises and taking numerous medications a day to ensure lung function.
“It makes it really hard to live a normal life as a 25-year-old,” she said.
Gibbs is subject to 30 plus treatments and medication intakes per day depending on her health and has been recently battling hard to keep her lung function from declining.
“Trikafta can dramatically reduce the number of hours I would need to spend on these treatments and medications to keep myself alive,” she said.
“It’s a symptom improvement in being more normal, active and contributing to society with a job without constant sickness.
“Trikafta gives me some hope of returning to work full-time from an office and not flaking on my friends when social engagements come up … things like that would be such an improvement.”
In Australia, one in 2,500 babies are born with cystic fibrosis, and there is currently no cure.
The median life expectancy for Australians with cystic fibrosis is 47 years, significantly lower than the average Australian.
“We have been told our whole lives that our life expectancy is lower, and from someone approaching their mid-twenties, that’s pretty scary to think about,” Gibbs said.
“My future is always on my mind…is it worth going to university? Is it worth getting a job? Should I be putting a bucket list together?
“Trikafta means I won’t have to dwell as much on my future and what comes next in that context.”
Gibbs hopes to start Trikafta as soon as her doctors deem it safe.
In the meantime, she sympathised with suffers who were yet to gain access to the drug.
“While accessing Trikafta is amazing for me and has given me hope, there are still many people who aren’t able to access this drug,” she said.
“I hope that in the future, we can continue to fight for access to drugs for all people with cystic fibrosis so they can experience some relief.”
She said the drug is currently approved for around 90 per cent of people with cystic fibrosis and their genotypes, meaning around 10 per cent of people with the disease cannot access the drug yet.
Sufferers aged six to 12 are currently waiting on Therapeutic Goods Administration (TGA) approval for the drug before proceeding to the Pharmaceutical Benefits Advisory Committee (PBAC), which determines if it will become available for the age group on the PBS.
“Our PBAC process is unfortunately quite slow compared to other countries,” Gibbs said.
“I hope that the government and PBAC can work on expediting future applications to ensure that cystic fibrosis patients of all genotypes can access this life-changing medication and future medications that are even better at helping people live a normal life.
“It’s definitely not over – there are many people still out there fighting, and I hope the government can continue funding so all people with cystic fibrosis can access lifesaving treatments.”
Maia O’Connor