Belinda Cunningham says it is heartbreaking to know a drug that could extend the life of her three-year-old granddaughter sits on a pharmacy shelf but remains out of reach.
The 45-year-old from Perth is one of more than 50 people meeting in Canberra today to convince the Federal Government to fund Kalydeco, a cystic fibrosis medicine that could improve lung function and increase life expectancy by 10 years or more.
Ms Cunningham's granddaughter Aaliyah Byrne is one of about 240 Australians with a rare form of the disease, caused by the gene mutation G551D, who could benefit from the new drug.
But the high cost - up to $300,000 a year for each patient - has resulted in a stand-off between the drug's maker Vertex and the Pharmaceutical Benefits Advisory Committee over who should pay.
The committee has recommended funding Kalydeco on the Pharmaceutical Benefits Scheme for patients aged six and over but on the condition that the results prove to be as good as those from Vertex's clinical data.
If the drug does not work, the manufacturer would be forced to repay money.
Ms Cunningham said yesterday she understood the drug was expensive and the Government needed to do its homework, but in the meantime the health of patients was going backwards.
"Aaliyah is not eligible to be on the drug until she's six but we want to know it will be there for her as soon as she needs it," she said.
"While we have the luxury of a few years yet, there is still no guarantee she will be able get it and in the meantime other children are already missing out.
"Despite all the breakthroughs, very young people are still dying. It's really getting to me that people are suffering irreversible damage unnecessarily."
People with cystic fibrosis lack an essential protein in their cells, causing sticky mucus to build up in organs such as the lungs and pancreas.
Average life expectancy is 37 but some people die as children or young adults.
Cystic Fibrosis WA chief executive Nigel Barker said Kalydeco was the first drug of its type to treat the underlying cause of the condition rather than the symptoms.