New treatment on PBS gives hope to kids with Cystic Fibrosis

Twelve-year-old Cystic Fibrosis patient Nick has become one of the first paediatric patients to receive the life-changing treatment, Trikafta, following its listing on the Pharmaceutical Benefits Scheme (PBS).

Nick was diagnosed with Cystic Fibrosis (CF) in utero at just 16 weeks gestation via a prenatal procedure called amniocentesis. CF is an inherited disorder that causes severe damage to the lungs, digestive system and other organs in the body.

“I don't know what life is like to not have cystic fibrosis because I have always had it. I have always taken tablets, done physiotherapy and spent a lot of time in the hospital,” Nick said.

It was thought this would be the management plan for the rest of Nick’s life, it was his normal, but after Trikafta became available, he was given a new beacon of hope.

While CF has no cure, Trikafta is the next best thing. The treatment works by improving breathing, reducing the risk of lung infections, and improving weight gain.

Nick’s mother, Helen, cried tears of joy when she heard the news.

“I felt overwhelming relief and for the first time, I could breathe. When you have a baby you have all these hopes and dreams for them and with a diagnosis such as CF all those hopes and dreams are taken from you and you grieve the life you had visioned for your child,” Helen said.

“Now Trikafta has been put on the PBS, I feel like we had been given a second chance to have that life.”

The life-changing treatment has previously been nearly impossible for most families to afford, costing more than $250,000 a year, but now the treatment will cost families a maximum of $42.50 per script, or as little as $6.80 with a concession card.

With the treatment becoming more accessible, patients like Nick will no longer need to have lengthy hospital stays or breathing treatments, and will finally be able to participate in the things they love without the fear of getting sick.