10 year old Cystic fibrosis sufferer Evie Marshall and her mum Sonia are hopeful the federal goverment will approve new drug listing that would radically improve the young girl's life.  Photo Patrick Woods / Sunshine Coast Daily
10 year old Cystic fibrosis sufferer Evie Marshall and her mum Sonia are hopeful the federal goverment will approve new drug listing that would radically improve the young girl's life. Photo Patrick Woods / Sunshine Coast Daily Patrick Woods

'How can a price be put on my daughter's life?'

A SUNSHINE Coast mother has failed in her attempt to have the Federal Government list on the PBS a drug that would vastly improve her 10-year-old daughter's life.

The decision, based primarily on cost, condemns Year 5 Nambour Christian College student Evie Marshall to an uncertain future.

Each year at home, the cystic fibrosis patient undergoes 1100 hours of physiotherapy and nebuliser use for lung function, takes 15,500 tablets, attends more than 70 doctor appointments, endures 730 insulin injections and misses between 40 and 60 days of school.

In a decision released last week by the Pharmaceutical Benefits Advisory Committee, PBS listing of the drug 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".

The drug known commercially as Orkambi is produced by Vertex, a United States company under fire last year for paying out $US29m in retention bonuses to its five senior staff.

Treatment cost per patient based on the company's last offer to the Australian Government was about $259,000 a year. There are 1000 cystic fibrosis patients in Australia, 50 of whom live on the Sunshine Coast. The PBAC noted the net cost to the PBS would be more than $100 million in each of the first five years of listing.

It found that at Vertex's requested price, the drug was not sufficiently cost-effective to enable recommendation for PBS listing.

Additionally, the PBAC considered that the estimated incremental cost-effectiveness ratio per quality-adjusted life year was likely to be underestimated.

"The PBAC considered that, given the more modest clinical benefit, the price was too high to result in acceptable cost-effectiveness, even if it was recommended in conjunction with risk-sharing and pay-for-performance arrangements," the committee found.

Evie's mum Sonia flew to Canberra in March to give testimony into Orkambi's effectiveness and what it would mean to her daughter's quality of life.

Sonia said results of 100 Orkambi trials worldwide - 10 of which were conducted in Australia - led to a 61% decrease in hospitalisations, a 56% reduction in antibiotic use, improved nutrition and weight gain, improved mental health and sustained improvement in lung function.

"I feel utterly devastated at this decision,'' she said. "I feel like my daughter's future has been taken.

"How can a price be put on my daughter's life? How can those with cystic fibrosis on the Sunshine Coast, and my child, be denied affordable access to this life-changing and lifesaving drug?

"I challenge our government health bodies and Vertex to come to a compromise quickly. The CF community, and my daughter, should not be penalised because of a breakdown in commercial agreements.

"Every day a young person is denied access to Orkambi is potentially another day of irreparable and life-limiting lung damage."

Sonia praised the Nambour Christian College that held a fundraiser which gifted the youngster two top-of-the-range nebulisers and a glucose monitor to help manage cystic fibrosis-related diabetes.

Analysts believe ultimately Vertex will make billions of dollars from the treatment. Last year the company's chief executive Jeffrey Leiden was paid a total of $US36.6m including a $US14.9m retention bonus.



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