‘Revolutionary’: CF drug improves lung function 10-15 per cent in a month

Over 100 Cystic Fibrosis patients in Tasmania could benefit if a highly efficacious treatment drug is granted PBS listing, taking the annual cost of the drug down thousands of dollars.

Up to 100 of the 120 Cystic Fibrosis patients in Tasmania could benefit if a highly efficacious treatment drug is granted PBS listing, taking the annual cost down from $300,000 to $79.

Pressure is mounting to gain patients quick access to Trikafta, as price negotiations continue with no confirmed end point between the federal government and Vertex Pharmaceuticals which would then allow the drug to be listed on the PBS.

Independent member for Clark Andrew Wilkie has written to federal Health Minister Greg Hunt urging a timely resolution be made and Cystic Fibrosis Tasmania Executive Officer Paula Wriedt said there was no reason why the price could not be finalised in a four week period.

Andrew Wilkie in Hobart. Picture: RICHARD JUPE

Trikafta is reported to improve lung capacity by 10-15 per cent in just four weeks and reduce some of the complications of the disease which contribute to overall illness, which would improve the length and quality of life for patients who have an average life expectancy of 30 years.

“There is a sense of urgency for patients, they are the human face behind this squabbling over money,” Ms Wriedt said.

“It can’t drag on for six or 12 months as people’s lives are literally at risk.”

If PBS listed, the price of a monthly prescription will be $6.60 for health care card holders and $41.30 for non-health care card holders, with an annual respective cost of $79.20 or $495.60.

Cystic Fibrosis Tasmania (CFT) annual awareness and fundraising campaign launch, CFT executive officer Paula Wriedt speaks to the media

This is a stark contrast to the current annual cost of $300,000.

“Every day that eligible Australians are denied affordable access to this drug is another day spent coughing and struggling to breathe while their condition deteriorates,” Mr Wilkie said.

On December 10 last year the independent Pharmaceutical Benefits Advisory Committee recommended a PBS listing of Trikafta for Cystic Fibrosis patients aged 12 years and older.

“The government will not hesitate to list Trikafta on the PBS once the pharmaceutical company has agreed to the terms of listing,” a federal Department of Health spokesperson said.

“Negotiations with the company are ongoing.”

Cystic Fibrosis drug offers hope for young Tasmanian sufferer

Getting through a normal working day without a persistent cough, dripping nose, stomach pain and lethargy is one dream for Cystic Fibrosis sufferer Lizzie van Dongen.

But for the 25 year old to live beyond the average life expectancy of age 30, have her own children and grandchildren is an even bigger reason why she immediately needs access to a “revolutionary” treatment drug called Trikafta.

The Pharmaceutical Benefits Advisory Committee made a submission last Friday to request a Pharmaceutical Benefits Scheme listing for eligible Cystic Fibrosis patients aged 12 years or older to gain subsidised access to Trikafta.

The drug would otherwise cost users $300,000 annually, which is out of reach for most of the 100 Tasmanians who would have dramatically improved lives if they could access it.

It is a nerve-racking time for the West Hobart woman as she waits for Trikafta to be included in the PBS, and for the price negotiations between drug company Vertex Pharmaceuticals and the Federal Government, but once this is completed it is as simple as going to the doctor and having a script written.

“In just four weeks it can increase lung function by 10-15 per cent and reduce permanent lung damage,” Miss van Dongen said.

Cystic Fibrosis sufferer Lizzie van Dongen is awaiting the pharmaceutical companies to set the price and start distributing the new drug Trikafta. Picture: Chris Kidd

“Declining lung function is a key indicator of morbidity and this is quicker as you get older so just imagine what this drug can do for your life expectancy.”

But time is of the essence for Cystic Fibrosis sufferers to access this drug to have the opportunity to live a long life free of other complications.

“The comorbidities affect your digestive system, liver, kidneys, pancreas and you may also get diabetes, osteoporosis, reflux and bowel problems along the way,” she said.

Right now Miss van Dongen battles her severe form of the disease with a gruelling regimen of daily physiotherapy and medication, a cumulative six months of antibiotic use annually for repeated infections and several hospital admissions every year.

Despite these challenges, Miss van Dongen has established a successful career as a muscular-skeletal physiotherapist, has travelled extensively including trekking through Nepal and has many more dreams on the horizon.

“I think that was just the way I was brought up, my parents did a really good job of not making me feel different, they just encouraged me to live a healthy and happy life,” she said,

“In December I ran my first (65 kilometre) ultra-marathon and next I would really love to do 100 kilometres.

“I would love to become a wife and mum and just to travel; there is a whole world out there I am champing at the bit to explore.”

Her plea to the PBS, Vertex and the Federal Government is of the utmost urgency.

“This is a permanent illness you spend a lifetime trying to fight and it progressively gets worse,” Miss van Dongen said.

“This highlights the time sensitive delivery needed for us.”