'Dream come true': Hope after receiving life-changing drug
ALMOST three months ago, this Sunshine Coast family found out they would have access to a drug that would change their lives.
Now, they want to thank the community for helping make it happen.
Evie Marshall, 12, was diagnosed with cystic fibrosis at six weeks of age.
She was taking a cocktail of 44 tablets a day, plus 10 hours with a feeding tube into her stomach to help maintain her weight.
But on August 17, a drug that would add at least 20 years to her short life expectancy was accepted onto the Pharmaceutical Benefits Scheme.
"It was like no feeling I've ever felt before," Evie's mother, Sonia, said.
"To be told that your daughter has another 20 years to live, it's a life-changing moment.
"It's just a dream come true, it really is."
Orkambi, a drug that treats people suffering from cystic fibrosis with the genotype Delta 508, costs about $250,000 per patient per year. But under the PBS it would cost the Marshall family under $7 a month.
Ms Marshall said Evie had already had a "fantastic" response to the drug, feeling the effects after just two hours of taking her first pill.
Her lung function has increased by 16 per cent, she no longer needs the two antibiotics she was taking daily since the age of four, her need for physiotherapy and nebulised medications has halved and she hasn't needed insulin for her CF-related diabetes.
Evie has gained weight, been at school more often and had lots of energy.
"I'm honestly feeling a lot better," Evie said.
"Straight away ... I could just run. I wouldn't feel as out of breath when I ran."
The Nambour Christian College student said not having to take as many pills and not being in hospital as much was "pretty awesome".
"I'm a lot happier," she said.
For the first time in Evie's life, she's starting to imagine a future that doesn't end at age 37 and is already planning to study at university.
"It's really fun now, I can actually think of a future," she said.
The Marshall family have credited the Sunshine Coast community for the continued support in making Orkambi accessible.
"They signed petitions, they wrote to their federal members, they really got behind us," Ms Marshall said.
"That together is a pretty loud voice, and that's what made the difference."
About half of cystic fibrosis sufferers in Australia are still without a "magic pill" like Orkambi, but Vertex Pharmaceuticals recently announced a new drug that would cover 90 per cent of the different forms of the disease.
'Grateful" mother's heartfelt letter
Dear Sunshine Coast Daily,
I want to say a huge thank-you to you for your continued support for those living with cystic fibrosis and your coverage of their struggle to gain affordable access to a new life-extending medication called Orkambi.
The drug company was asking $250,000 per patient per year for the medication. The Australian Government had rejected it three times for Pharmaceutical Benefits Scheme listing due to the cost, and the drug company had considered pulling out of Australia.
After a lot of campaigning by the CF community and media coverage by the Sunshine Coast Daily, the Australian Government finally listed the medication on the PBS.
The lives of more than 1300 Australian children and young people living with CF have now been extended by about 20 years. This includes my own 12-year-old, Evie.
Since starting the medication six weeks ago Evie's health has dramatically improved. Her lung function has increased by 16 per cent, she no longer needs the two antibiotics she was taking daily since the age of four, her need for physiotherapy and nebulised medications has halved, she hasn't needed insulin for her CF related diabetes, she has gained weight, been at school and had lots of energy.
The medication is a not a cure, but it is nothing short of a miracle. The Sunshine Coast Daily was a huge part of changing Evie's life and the lives of more than 1300 others with CF.
Sonia Marshall (Evie's very grateful mother)