Cystic fibrosis patients plea for Pharmaceutical Benefits Advisory Committee to list Orkambi on the Pharmaceutical Benefits Scheme

Ms Gollan, 25, lives with cystic fibrosis, a chronic condition that affects the lungs and digestive system.

Since she was granted compassionate access to the new drug in January, she has seen her lung capacity grow from 35 per cent to a high of 50 per cent, her energy levels increase and her health stabilise.

“All those mundane tasks that people take for granted – I can now do them without having to take a nap,” she said. “I have not needed to call in sick, which means I can hold down a job and not let the team down.

“I did the City 2 Surf. I feel bad taking it when everyone else can’t access it too, but I feel I owe it to all the other CF patients to take it and make my health as good as I can so I can be a voice for them.”

Ms Gollan travelled as a Cystic Fibrosis Australia patient representative to Canberra last month to share her experience with members of the Pharmaceutical Benefits Advisory Committee (PBAC).

PBAC is meeting this week to determine whether to recommend Orkambi for listing on the Pharmaceutical Benefits Scheme.

Orkambi targets the cause of cystic fibrosis, not just the symptoms, and has been shown in clinical trails to improve lung function and reduce hospitalisation and antibiotic use.

Members will take into account its clinical effectiveness, safety and value for money compared with other treatments. PBAC previously decided after its March meeting not to recommend listing “based on 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.”

Ms Gollan said she was prepared for the drug to be rejected, but remained hopeful. She said Orkambi had the potential to change the lives of 1000 people, but costs about US $260,000 without subsidy each year.

“This drug gives you hope for the future and I will never give up fighting for it,” she said. “If I could say anything to the decision makers it would be that our lives matter and every patient with cystic fibrosis deserves the right to access the best medications we have. To take the hope away from all these patients is cruel and unfair.

“It’s in your power this week to extend our life expectancy [beyond 37], reduce our lung decline and ultimately provide stability and good health, which is priceless.”

A Department of Health spokesperson said it will announce its decision on December 16.

Cystic Fibrosis Australia CEO Nettie Burke said every day a person was denied access to Orkambi was another day of potential life limiting lung damage.

“CFA is committed to holding our politicians and bureaucrats’ consciences to the fire so they don’t shy away from the tough decisions,” she said in a statement.

This story first appeared on The Newcastle Herald.