‘I feel like I’m drowning’: Thousands with cystic fibrosis wait to hear if ‘life-saving’ drug will be listed on PBS – 9News

Matt Dickinson was struggling to breathe when his mother rushed him to an Adelaide hospital earlier this month.

The 20-year-old has cystic fibrosis, which causes mucus to form in the lungs, and an infection had made him fall dangerously ill.

"His body was producing so much mucus that he couldn't keep up with clearing it," his mother, Cassandra Day, said.

"He told me, 'I feel like I'm drowning' and I've never heard him say that before.

"He said, 'I feel like I have water in my lungs. I can't breathe. It's heavy. I'm struggling.' It was terrifying."

Adding to the family's distress, COVID-19 protocols meant Ms Day could not be with her son, who also has autism, while he was in hospital.

"It was petrifying because he was so sick," Ms Day said.

"And then the phone calls that I was getting from the doctors were of significant concernthey were sharing how concerned they were and I felt absolutely powerless.

"I had no idea how my boy was, I didn't know if he was going to come back from it."

Matt ended up spending weeks in hospital hooked up to IV antibiotics - including his 20th birthday - without his family.

Lengthy hospital stays are familiar ground for many of the 3500 Australians living with cystic fibrosis, the country's most common genetic disease.

The condition has no cure and recurrent infections cause irreversible damage, with lung failure the most common cause of death.

Around half of those with cystic fibrosis currently pass away by the age of 30.

Ms Day said watching her son's condition decline was even more devastating knowing there was a medication that could give him his life back.

While the drug Trikafta is available to patients in Australia, it is incredibly expensive, costing more than $300,000 per year.

Ms Day is one of many campaigning for Trikafta to be added to the Pharmaceutical Benefits Scheme (PBS) for everyone with cystic fibrosis.

A petition with 35,000 signatures calling for Trikafta to be subsidised under the PBS was presented to Federal Parliament in November by Labor MP and paediatrician Mike Freelander.

"Trikafta is a life-saving and life-changing treatment for people for people with cystic fibrosis," Mr Freelander told parliament at the time.

"We have seen people taken off the heart/lung transplant list. We're seeing people's lives saved."

In July last year, the Pharmaceutical Benefits Advisory Committee (PBAC) recommended Trikafta be listed on the PBS, but only for those aged over 12 and with a specific mutation of cystic fibrosis.

Genevieve Handley, who started the parliamentary petition, said the decision would only cover about 20 percent of the 2200 people waiting to access the medication.

Vertex Pharmaceuticals, which manufactures the drug, is also yet to proceed with the PBAC recommendation.

Listing Trikafta on the PBS was also discussed at a PBAC meeting in December, with the independent advisory panel's decision expected to be made public tomorrow.

Ms Handley said people's lives were depending on Friday's PBAC outcome and Vertex Pharmaceuticals agreeing to list Trikafta.

"More than 25 countries have provided access and reached agreements before Australia," Ms Handley said.

"There are 2200 Australians who need Trikafta now.

"The CF community desperately needs a 'yes' from the PBAC, followed by access soon afterwards."

Ms Handley, who has cystic fibrosis, said Trikafta had been a gamechanger for her after she was approved for Vertex's limited compassionate access scheme, three days before she was due to meet with a lung transplant team.

"It's improved my lung function from 34 percent to 55 percent," she said.

"My lung bleeds that had continued for months have stopped and I no longer need to see the transplant team.

"I can breathe without hearing crackles or wheezing, rarely cough and can walk along the beach and talk at the same time."

Ms Day said hearing positive stories about other people who were able to access Trikafta gave her hope that Matt would soon be able to benefit.

"He has been so sick but having access to this medication would basically give him his life back and help him be able to realise that that he can do more than just be the sick kid," she said.

"I think our kids deserve a future."

9news.com.au has contacted the Department of Health for comment.

A spokesperson for Vertex Pharmaceuticals said: "Vertex has always maintained its focus to ensure equitable access to Trikafta for all eligible Cystic Fibrosis patients in Australia."

"Trikafta has been available internationally for up to two years. There are now more than 25 countries, including Canada, the UK, France, Germany, and the Netherlands, that have recognised the value Trikafta brings to the CF Community and funded the therapy for all eligible patients. Australian patients deserve the same."

A spokesperson for the Federal Health Department said in September that Vertex had still not provided a formal proposal for the drug to be reconsidered and listed for the broader cystic fibrosis population.

The spokesperson for Vertex said the proposal was submitted at the December PBAC meeting.

Contact reporter Emily McPherson at emcpherson@nine.com.au

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'I feel like I'm drowning': Thousands with cystic fibrosis wait to hear if 'life-saving' drug will be listed on PBS - 9News