Temora nurse Renae Golder has two children with Cystic Fibrosis, a debilitating disease that requires extensive treatment and greatly reduces lifespan. 

Until recently, doctors could only manage the symptoms, but a drug called Orkambi targets the underlying genetic condition in some sufferers, reducing the severity of the condition.

The only problem is that the drug costs between $260,000 and $300,000 per year.

There have been two failed attempts to list Orkambi on the Pharmaceutical Benefits Scheme (PBS), which would put the drug within the reach of the more than 1000 patients who would benefit from the treatment.

Mrs Golder said she was willing to be a voice for her children who were being denied access to the medicine, so she will join a protest on the lawns of Parliament House in Canberra on Tuesday.

“Medicine’s no good if it’s not being used,” Mrs Golder said.

“This is a business to (the drug companies), they have the power and can put a price on it, but I need it for my kids.

“It frustrates me to no end that all of that research and effort has gone into finding a treatment and we can’t afford to use it.”

Clinical trials show Orkambi has cut relapses of Cystic Fibrosis by 39 per cent, meaning a 61 per cent fall in the number of hospitalisations.

It has also led to a 56 per cent decrease in the need for intravenous antibiotics among sufferers. 

Orkambi is available under subsidy in the US, Germany, France and Austria – but has been rejected in Australia.

When it was first rejected by the PBS, a Department of Health spokesman said it was because of an “unacceptably high and uncertain incremental cost-effectiveness ratio at the requested price by the sponsor and uncertainty around the impact on long-term improvements in lung function and survival … The net cost to government was more than $100 million in each of the first five years of listing.”

If listed on the PBS, it is estimated Orkambi would cost between $200-$1500, depending on financial circumstances. 

Mrs Golder pointed out that ongoing hospitalisations also put a strain on the health system and asked why a treatment wasn’t a better option.

“If this drug contains their lives, why not use it?” she said.

“For the kids there’s a lot of hard work, a lot of routine, a lot of medication every morning, physiotherapy, sometimes life gets put on hold to go to the treating hospital in Sydney.

“I’ve spent months sitting by their bedside, it’s a slow deterioration for them, there’s no cure, we just try to make the most of what we’ve got and our time together.”

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