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RELATED: We've given up hope on CF drug
A GOORNONG family is in a state of happy shock after the federal government announced the addition of the drug Kalydeco to the Pharmaceutical Benefits Scheme.
Glenyce and Tim McCrohan, whose daughter Amelia, 7, suffers from cystic fibrosis, have been campaigning for the drug to be subsidised by the PBS.
Kalydeco is a new treatment for cystic fibrosis in patients with a specific gene mutation and is the first medicine to treat the underlying cause of the disease.
It reduces respiratory and gastrointestinal complications, improves lung function and leads to fewer hospitalisations.
Kalydeco will transform Amelia's quality of life, from one of constant illness and 30 daily medications, to that of a healthy, happy girl.
"We'd given up hope about seeing (Health Minister) Peter Dutton list it," Mr McCrohan said.
"I'm still in a state of shock. We didn't think it was going to happen."
"We’ve been fighting for it for so long, it still seems unreal," Mrs McCrohan said.
Without being on the PBS, the drug was unattainable, costing $300,000 a year per patient.
The government has approved $174.5 million to pay for it to be available for the next four years.
The PBS listing of Kalydeco will help more than 250 Australians affected by the cystic fibrosis G551D gene mutation.
"This drug will turn off Amelia’s CF. She’ll be able to function relatively normally and not have the symptoms causing trouble for her," Mrs McCrohan said.
"It’s not a cure. And it will only help a certain percentage of the cystic fibrosis population but what it does is it turns off the defective gene," she said.
"To a certain degree, she will be able to forget about the fact that CF is there and it won't rule her life the way it does now.
"It gives her the opportunity to aim as high as she wants to. She can do whatever she wants without the limitations that she had before."
Amelia won't start taking Kalydeco until December 1, because the drug has only just been listed and the family's doctor can't prescribe it yet.
Once she starts taking it, Amelia's parents hope to wean her off the many medications she takes each day.
"The best possible outcome would be that she could come down to two tablets a day," Mrs McCrohan said.
The McCrohans have been heavily involved in a social media campaign to create awareness about the need for Kalydeco.
"There's been letters written to politicians to get as much help as possible. There was a petition presented to parliament last week which was on behalf of the 'yes to Kalydeco' community," Mrs McCrohan said.
"We have been quite a vocal committee and have been chasing Peter Dutton for a long time."
Mrs McCrohan said Australia was one of the last developed nations to approve the drug.
"All those other countries in a worse financial situation have had the drug to use. It’s been quite frustrating waiting," she said.
Mr McCrohan encouraged other people fighting for certain drugs to be provided through the PBS to "keep up the fight".
"Eventually they will get listed," he said.
