Delay in life-saving treatment could halve Aussie girls life

A subsidy for a drug that could double the lifespan of thousands of Aussies, including 14-year-old Victorian Rita De Bon, is cruelly delayed.

Exclusive: Rita De Bon’s lifespan will be slashed in half, she will struggle to breathe, suffer sleepless nights and cough up blood when the money for a life-saving drug runs out in October.

The 14-year-old from Lorne in Victoria who dreams of becoming an Olympic horse rider suffers from cystic fibrosis – a genetic condition that clogs her lungs and body with sticky mucus that makes it hard to breathe.

Her life expectancy is just 37 years of age.

Since April, she has been using a breakthrough $277,000-a-year drug called Trikafta which resulted in her lung function improving by 25 per cent in just eight weeks.

The breakthrough new treatment allowed her to put on weight and lead an almost normal life for the first time.

It’s also allowed her to come off multiple antibiotics and steroid drugs.

“This drug is as close to a cure as we’ll possibly ever see, it is saving her life,” Rita’s mother Bhakti De Bon said.

“She is now watching the Olympics, and saying, for the first time she actually has a real chance to live a life that could get her to the Olympics.”

Rita de Bon is 14. Picture: Supplied

Rita with her horse Puff. Picture: Supplied

The Year nine student said since taking the medicine she has not had to go to hospital for two week-long “tune ups” that kept her breathing.

“I’m pretty sad because it means I won’t be able to live as long and do as much,” Rita said of the prospect she will have to stop using the treatment later this year.

About 1200 Australians suffering from the devastating condition are desperate for the subsidy which would slash the price of the drug to just $40 per month.

This medicine is available to cystic fibrosis patients in 17 other countries.

The money for Rita’s high cost medicine was made available by an extremely generous family friend and was meant to tide Rita over until a federal government committee subsidised the medication earlier this year.

Instead, the drug subsidy process has become a stalemate between a pharmaceutical company charging an exorbitant price for a lifesaving drug and bureaucrats quibbling over how many people will use the drug that could cost taxpayers $1 billion.

The Health Department wants to provide a single bucket of funding for the treatment but it estimates 700 fewer people will continue using the treatment than pharmaceutical company Vertex estimates.

Rita’s life expectancy is just 37. Picture: Supplied

The government’s independent advisory committee that decides which medicines get a subsidy has twice deferred a decision on whether to subsidise the drug and is expected to announce a further delay later this month.

Cystic Fibrosis Australia CEO Nettie Burke said her organisation has offered to run a data registry on patients using the drug and report to the government every six months.

“This drug is outstanding. Women and men with CF are having babies and they are living longer,” Ms Burke said.

“A child born today using this drug in all likelihood will live into their late 60s and 70s when five years ago they lived on average until they were 37,” she said.

Rita’s parents are pleading with Health Minster Greg Hunt to intervene to fast track the subsidy for the medicine.

“We’ve got it now to October and then I guess she has to come off it and go back to a life where she has to entertain the idea that she won’t live,” Mrs De Bon said.

“The most common genetic disease. In Australia, why would you be asking them to hold your breath a bit longer throat because for a better price,” Rita’s father Ralph De Bon said

Originally published as Delay in life-saving treatment could halve Aussie girls life