Subsidy a welcome relief
THE lives of thousands of Australian children and adults living with cystic fibrosis (CF) will improve following last week’s decision to make a revolutionary drug publicly available.
The Pharmaceutical Benefits Advisory Committee (PBAC) announced on Friday a deal with Vertex Pharmaceuticals to make a lifesaving drug called Orkambi accessible.
It was the pharmaceutical company’s fourth submission to have the expensive drug listed on the benefits scheme for patients 12 years and older and its first submission for patients six to 11 years old.
Both submissions were accepted and the announcement was met with celebration from families across the country who had been campaigning for the move.
Before Orkambi was on the scheme, it cost patients $250,000 a year.
Mt Helena parents Melissa and Justin Gibson rejoiced at the news, knowing their eight-year-old son Toby would now have hope of a longer life.
Toby, who was born with the rare genetic condition, has two copies of a gene mutation that Orkambi targets.
Mrs Gibson said the drug would be “life altering” for Toby, enabling him to experience a better quality of life through improved lung function and less hospital admissions, and prevent further lung damage.
Cystic Fibrosis WA (CFWA) chief executive Nigel Barker said the decision was a culmination of years of advocacy by people whose lives had been impacted by CF to force an agreement between the government and Vertex.
“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.”
CFWA president Caz Boyd, who was born with two copies of the gene mutation that Orkambi targets and had a lung transplant at 27, said she was grateful that the journey for youngsters living with CF would be different from hers.
“If this drug was available 24 years ago I wouldn’t have needed a lung transplant and witnessed the lives of many childhood CF friends cut short by this insidious disease,” she said.
Melissa Gibson, whose eight-year-old son Toby has CF, is overjoyed that Orkambi has been accepted into the Pharmaceutical Benefits Scheme.