It’s almost unimaginable that children and adults with a debilitating disease like cystic fibrosis can be denied a drug that could quite literally help save their lives because of cost.

Sadly, for the 1,000 young Australians with the cystic fibrosis gene mutation F508del homozygous, this is the case.

Orkambi goes back to the Pharmaceutical Benefits Advisory Committee (PBAC) for the third time in July 2017 and it is difficult to understand any sense behind this quarrel over dollars.

Australia is a country that prides itself on an equitable and fair health system but sadly the relevant parties, the government’s PBAC and the multinational pharmaceutical company Vertex, have been unable to come to an agreement on costs for this life changing drug.

The CF community must act and put pressure on both the drug company and the government to come to an agreement before lives are lost. Register your support at

The Federal Health Minister Greg Hunt has been supportive but we still are denied access to this life-changing drug.

Orkambi is designed to treat the underlying cause of CF and clinical trials in Australia and across the world provide solid evidence that Orkambi improved lung function and reduced exacerbations, hospitalisation and antibiotic use. 96-week trial outcomes clearly showed that lung damage was slowed on average by 40%, preserving lung function and maintaining lung health.

Exacerbations reduced by 39%, hospitalisations were reduced by 61% and antibiotic use by 56%. These statistics alone are massive improvements for cystic fibrosis sufferers used to lengthy hospital confinement and taxing drug regimes.

Trial participants reported improved nutrition and subsequent BMI increases, both of which have the capacity to stave off diabetes. Orkambi had a positive flow-on effect on the mental health of people with CF leading to social inclusion and a reduction in personal and family stress. Given the toll CF takes on people born with the disease, this emotional, physical and financial burden on families is equally devastating.

The PBAC must realise that Australians with CF need access to life changing drugs.

If you would like to tell your story to the media or our politicians in Canberra contact Vanessa at CFA by email

Yours sincerely,

Nettie Burke