By Luke Voogt
Government haggling over a new wonder drug is putting Ocean Grove youngster Brody McPherson at risk of irreversible lung damage, according his mother Kate.
The three-year-old has been on 40 tablets a day since being diagnosed with cystic fibrosis (CF) at four weeks of age.
He is one of 30 children in Australia under six years old with a rare form of the disease.
“We can’t have a day without thinking about CF,” Kate said.
“It’s just a constant battle – you don’t get a moment off.”
The “brave, cheeky” three-year-old endures three nebuliser treatments a day and has been hospitalised for weeks at a time, Kate said.
But a new wonder drug – Kalydeco – could add decades to Brody’s life and prevent irreversible lung damage.
“It would change our lives completely,” Kate said.
“It would just give him a chance at being a normal kid.”
The drug works by treating certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which account for 5 per cent of CF cases.
“It’s absolutely amazing,” Kate said.
“All the drugs he’s on now just treat the symptoms of the disease. This drug actually treats the cause.”
“Forty tablets a day is a lot for a three-year-old to be taking, when it could all come down to Kalydeco morning and night.”
The only problem: the Federal Government and Vertex Pharmaceuticals, the company which produces the drug, are still haggling over the price.
“It’s been proven to be safe and effective,” Kate said. “It’s literally down to dollars and cents.”
The Government recently approved the drug for children over the age of six via the Pharmaceutical Benefits Scheme (PBS) after a three-year campaign by CF advocates.
But the drug is not available on the PBS for children between two and five years old.
“The Government just needs to list this medication as an absolute priority,” Kate said.
“We need to stop the disease in its tracks.”
The drug costs more than $200,000 a year outside of the PBS, Kate said.
“To me his life’s priceless but no one can afford that.”
As only 30 children under six have Brody’s condition in Australia, the expense for the Government would be relatively small, Kate said.
“Given the amount of medication he’s on, if they could offset that it would save them a lot of money.”
Jane O’Brien has CF and was behind the campaign to make the drug available for children older than six.
“Lives should come before profit,” she said.
“Every day these children are denied this medication they are at risk of irreversible lung damage.
“Kalydeco … has already changed the lives of a few hundred Australians.”
The Pharmaceutical Benefits Advisory Committee deferred recommending PBS listing for Kalydeco for patients aged two to five years at its November meeting.
A Federal Health Department spokesperson and a Vertex spokesperson said their respective parties working towards a resolution as “a matter of urgency”.
