THE family of a boy with a life threatening disease is battling authorities to get cheaper access to a potentially life saving drug.
Little Nixon Hoyle has cystic fibrosis.
The five-month-old wears a vibrating vest to dislodge excess fluid from his chest but there could be a better treatment.
The drug Orkambi, developed by North American company Vertex Pharmaceuticals, is designed to stop mucus production in patients with a specific genetic mutation of the disease.
At $250,000 per year, it is too expensive for Nixon’s family and requests for its inclusion on the Pharmaceutical Benefits Scheme have been rejected three times, most recently in July last year.
Nixon’s grandmother Kathryn Slater said patients’ lives were being risked because of the huge costs and lack of government help.
“It is a power struggle and profit struggle,” she said.
“...There are more medications to treat a headache than there are to treat this life threatening disease.”
A Vertex Pharmaceuticals spokesperson said the company continued to negotiate with the Australian government.
The spokeperson said billions of dollars had gone into the drug’s development over the past 15 years, with price reductions offered to countries which made the medicine available to all eligible patients.
The Pharmaceutical Benefits Advisory committee cited cost-benefit grounds as to why Orkambi was rejected but the decision cannot be scrutinised by tax payers. Vertex’s asking price and prescription costings have been censored.
Nixon’s mum Ashleigh Slater said she would continue to advocate for the drug’s listing.
She said the drug’s potential to help her son and others like him gave her cause for hope.
“I just hope that we all make it in the time that it takes for us all to get a fair shot with them,” she said.
In a previous hearing, the $100 million net cost per year for five years of PBS subsidy was considered too expensive.
While the committee refuted Vertex’s claims that the drug slowed lung decline beyond six months of treatment, it noted patients treated with Orkambi had less pulmonary problems and put on weight.
“There is a clinical place for this medicine at a price commensurate with its clinical benefits,” the committee concluded.
The Slaters have contacted health minsters Greg Hunt and Steven Miles to get Orkambi and another Vertex drug considered.
Mr Hunt did not directly respond.
A statement from the PBA committee said: “The government and the Minister for Health cannot pre-empt or interfere with the decision making process of the PBAC.”
To donate to Nixon’s cause, visit here.