A "pill of hope" could save Taryn and Michelle's sons. But the Australian government says no.

Connor Barrett is nine-months old. A gorgeous little boy with soft, brown curls and a cherubic face. Yet inside his tiny body a devilish disease is taking hold, a disease that means he only has a 50 per cent chance of reaching his 30th birthday. The disease is Cystic Fibrosis.

“One day I will have to tell him about his shortened life expectancy, that his hair will never turn grey nor his skin wrinkle,” his mother, Taryn, told Mamamia. “He will most likely have to retire just as his career takes off to focus on his health, and he may not be able to have children.”

But arguably more difficult than that heartbreaking conversation, will be the one in which Taryn must tell him that he had a chance for a better outcome. A chance the government took away.

Taryn and Connor Barrett. Image: supplied

As a young sufferer of Cystic Fibrosis, Connor stood to benefit from a revolutionary new trial drug that has been changing the lives of those with this cruel illness.

That drug is called Orkambi, and last week the federal government decided not to approve it for inclusion on the Pharmaceutical Benefits Scheme. For families like the Barretts, that would mean an impossible financial burden of $5000 per week, or roughly $260,000 every year.

For Taryn, and countless other parents around Australia, the decision represents diminished hope.

"I don’t know how I could look into his beautiful blue eyes and explain that the Federal Government has put a price cap on his life," said Taryn.

According to CEO of Cystic Fibrosis NSW, Michele Adair, it’s the most common of the rare genetic diseases. "Three thousand people nationally suffer with Cystic Fibrosis,” she told Mamamia. “In Australia one person is diagnosed with the condition every four days."

CF is a life-limiting, genetic disease that causes progressive damage to the lungs via the build-up of sticky mucus and chronic infection.

It also deteriorates the intestinal system meaning sufferers struggle to get the nutritional value from food. Because of this, individuals with cystic fibrosis need to take enzymes, and somewhere between 60 and 80 tablets a day in conjunction with steroid inhalers and intensive physiotherapy, said Adair.

It’s a terrible toll on a young body, and one that Orkambi was alleviating for CF sufferers like Connor Philpott.

Connor Philpott (right) and his sister both suffer from CF. Image: supplied.

The 15-year-old used to require a minimum of two three-week stints in hospital each year for his regular “tune ups” and more still when he contracted chest infections or head colds. But since being placed on the Orkambi trial 16 months ago that’s all stopped.

Within a matter of just eight weeks on the drug his mother Michelle watched his lung function improve, his appetite increase and his energy levels soar.

“He’s so vivacious now,” Michelle told Mamamia. “He’s so outgoing and lively and bubbly. We hadn’t seen that for fourteen years.”

Image: supplied.

With two of her three children suffering from the Cystic Fibrosis, Michelle sees Orkambi is a “pill of hope”. Hope that a cure will come along and that children like hers will be well enough to receive it.

But now, since the PBAC decision, the mood in their house has changed.

“We feel so cheated,” she said. “It’s so hard to believe that we live in a world where this amazing drug available, but the government says ‘No, you can’t have it’.”

Despite the health of as many as 1000 young people being at stake, the PBAC decided that the cost to the government – roughly $100million per year for the first five years of listing – would simply be too great.

This is despite worldwide trials, including ten here in Australia, which found that patients on Orkambi experienced an average three per cent improvement in lung function, as well as a 40 percent reduction in exacerbations, fewer hospital visits and less need for antibiotics.

There’s a specific reason for the apparent success of the drug: it’s the only one that treats the underlying cause of the disease, rather than just the symptoms. That’s why countries including the USA, France, Germany and Australia have all elected to subsidise the drug.

By refusing to negotiate with Orkambi’s manufacturer over the price of Orkambi, Michelle Philpott argues that the PBAC are failing to see the bigger picture.

“My children haven’t been in hospital for 16 months. How much money are we saving the government there?” she said. “We’re not taking up beds, we’re not relying on the health system. We’ve been at home and absolutely 100 per cent healthy. That’s huge.”

An active CF fundraiser and advocate, Michelle is working hard to ensure that her children’s voices are heard.

“The whole CF community is banding together on this,” she said. “It’s really good to know that we’ve got a bit of fight in us. We’re not going to be quiet about this. We’re not going to stop talking, until we let every single person know that we’ve got 1000 children in Australia that the government’s not helping.”

To help these mums and CF sufferers Australia-wide, visit www.cysticfibrosis.org.au for more information and contact your local Federal representative in Parliament to let them know the importance of Orkambi to so many lives.

You can also assist Michelle Philpott in her personal fundraising efforts by visiting this link.