A LIFE for 10-year-old Nambour Christian College Year 5 student Evie Marshall and 50 others on the Sunshine Coast who share her illness will be determined by a decision to be made in March.
The Palmwoods youngster has already coped with more than most would deal with in a lifetime.
She knows her own life will be short unless she can access a revolutionary new two-drug therapy called Orkambi, which goes before the Pharmaceutical Benefits Advisory Committee and the Therapeutic Goods Association on March 16.
There, a determination will be made on whether the Commonwealth will subsidise the expected $300,000 annual cost per patient for a drug that offers a new life to cystic fibrosis sufferers.
That cost has been questioned by cystic fibrosis experts in the US who point to a price that is more than five times the average wage in that country.
Pharmaceutical company Vertex, the developer of the drug, justifies the cost on the basis of the small patient population, its clinical benefit, the time and cost spent in developing the treatment and the need to fund research and development to aid patients with different forms of the disease.
Evie's mum, Sonia, said the drug, which her daughter would not be able to begin taking until she was 12, would give her daughter a life.
Each year, Evie spends two weeks or more in a Brisbane hospital for sinus surgery, chest infections or dealing with the impact of cystic fibrosis-related diabetes.
She also undertakes more than 1100 hours of physiotherapy and nebuliser use for lung function at home, takes 15,500 tablets, attends more than 70 doctor appointments and visits to specialists in Brisbane, spends 3650 hours connected to feed pumps to deliver liquid food for nutrition, has 730 insulin injections and misses between 40 and 60 days of school.
Such is the array of medical equipment and medication needed to sustain her, the family has converted a room of their Palmwoods home as a virtual pharmacy to store, clean and prepare it all.
Sonia said if given access to Orkambi, Evie would have to take only two tablets a day and her quality of life would be vastly improved.
The cost is the issue. If the treatment is accepted under the Pharmaceutical Benefits Scheme, the cost to the patient would be $6.10 a month.
But with 1000 sufferers suitable for treatment across Australia the impact on the national health budget would amount to $300 million.
However, that cost could be offset against the hugely expensive treatment cystic fibrosis sufferers would otherwise require.
Sonia says it goes further than that.
"As her carer I can't work fulltime and can't work towards being a self-funded retiree,'' she said.
"I'm exhausted and am developing my own health issues. There are a lot of direct and indirect savings the Federal Government can make by approving the drug."
Sonia praised the support the family had received from Nambour Christian College.
"It's difficult for her but she receives wonderful support from her teachers and there is well-trained first aid staff. I can't speak more highly of them," she said.
Sonia said she wanted everyone on the Sunshine Coast to be aware of the ability of the drug to change and save lives.
"It's imperative that it be approved,'' she said. "I know there is a high cost and the impacts are significant, but everyone has the right to access life-saving medicine.''
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