A mother whose son lives with cystic fibrosis says young people are dying while waiting for a “life-saving” drug to be rolled out to all people living with the genetic disease that shortens life expectancy.
- Drug Trikafta Approved For People With Cystic Fibrosis With A Specific Mutant Under Drug Benefit Plan
- Mother of son with cystic fibrosis says thousands of patients without the mutant have missed out
- She said young people were dying while waiting for the “life-saving” drug
Cystic fibrosis primarily affects the lungs and digestive system due to dysfunction of the exocrine system, which is responsible for the production of saliva, sweat, tears, and mucus.
On three occasions, Vertex Pharmaceuticals has requested that its drug, Trikafta, be approved under the Pharmaceutical Benefits Scheme (PBS).
The drug currently costs Australian patients almost $ 300,000 per year.
While his requests had already been denied twice, on Friday the drug was made available to people with a specific cystic fibrosis mutation as part of PBS, making it more affordable, but only for those with a specific CF mutation. eligible people.
Vertex has not yet accepted the recommendation of the Pharmaceutical Benefits Advisory Committee (PBAC).
In a statement, the company said the “narrow recommendation” would exclude 80% of the CF community in Australia.
Thousands are missing
Bunbury’s wife Taryn Barrett – whose third son Connor was only three weeks old when diagnosed with cystic fibrosis – said there were nearly 400 patients with the specific mutation who would now receive ugly.
But, she said, around 1,800 patients in Australia, including her now six-year-old son, missed the point.
“People are irreversibly damaged and people are dying and that is not acceptable in a country like Australia.”
Under the PBAC’s recommendation – which would allow people aged 12 and over to access the drug – Ms Barrett’s son would not yet be eligible.
However, she said, once he got old enough the treatment would mean he “could age with it,” compared to his current life expectancy of 38 years.
A revolutionary drug
When the drug was approved by the Food and Drug Administration in the United States in 2019, it was called a breakthrough.
It can treat 90 percent of patients with cystic fibrosis and 20 countries have approved its use for all patients with cystic fibrosis.
“We need it for our little ones, to give them prevention and slow down this disease. My son will probably be able to age with it and, for adults, they need it now.”
Access denied ‘harmful’
In a statement, Vertex said it was disappointed with the PBAC’s recommendation:
“We do not agree with the restriction on access due to the reluctance of the PBAC to recognize the long term benefits of our reimbursed drugs. These services only delay access to Trikafta to the detriment of the CF community. “
– Vertex Pharma
The company said it is committed to ensuring patients have government-funded access to the drug as quickly as possible.
The Federal Ministry of Health has been contacted for comment.