20 May 2016 - West Pymble six-year-olds Charli and Jasmine are twins, but their future lives look very different.

Unlike her typically developing sister, Charli is living with both autism and cystic fibrosis – a disease that means she will be lucky to survive into her 30s.

But alongside the 1000 Australians diagnosed with two copies of the F508del cystic fibrosis gene mutation, Charli’s parents are facing a cost of $250,000 a year if they want to access a revolutionary new drug that improves the condition’s symptoms.

Her mother, Vivienne, has slammed the Federal Government’s decision by the Pharmaceutical Benefits Advisory Committee to reject the PBS listing of ­Orkambi.

In a decision handed down last month, PBS listing of Orkambi was refused “based on an unacceptably high and uncertain incremental cost-effectiveness ratio at the requested price by the sponsor, and uncertainty around the impact of the drug on long-term improvements in lung function and survival”.

Cystic Fibrosis Australia chief executive Nettie Burke said trials had found that patients on Orkambi experienced improved lung function and reduced exacerbations, hospitalisation and antibiotic use.

“It’s criminal and morally bankrupt to have a drug that can save lives and people aren’t able to get access because of funding,” she said.

More than 30,000 Australians have signed an online petition on Change.org calling for the government to “reach an agreement” with drug manufacturer Vertex to allow Orkambi’s approval on the PBS.

Orkambi will be resubmitted to the PBAC with more information in ­November.

For more details, go to: http://www.dailytelegraph.com.au/newslocal/north-shore/sydney-mother-calls-for-cystic-fibrosis-drug-orkambi-to-be-accessible-for-daughter/news-story/746b1f600a87be99fb27150e6da3ecd2