As a five-year-old boy with cystic fibrosis, Alexander Dunlop’s life expectancy is just 37 years without Orkambi, which targets the cause of the disease but costs $250,000 a year.

Now his parents Simon Dunlop and Katrina Martin, along with more than 1000 other families, have a tense wait as negotiations continue to have it listed and made affordable.

Ms Martin said the drug’s benefits in preventing costly hospital admissions – let alone improving everyday life for people with the chronic illness – made it “worth every cent”.

“Orkambi will be life-changing for Alexander,” she said.

“We hope and pray that it is approved by the PBAC so he is able to live a long life – one where he achieves all his hopes and dreams and isn’t restricted by his health.

“He is a proper little country boy, and we hope he can stay healthy enough his entire life to not have to move closer to a treating hospital and away from what he loves.”

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Alexander’s disease means he needs salty and fatty foods plus two extra teaspoons of salt a day in gel capsules; pancreatic enzymes every time he eats; and two or three nebuliser sessions a day.

“There’s also plenty of physio, including running, jumping, playing – encouraging him to take big, deep breaths.”

This all adds up to about 30 tablets a day and at least 1.5 hours of physiotherapy – that time doubles when he is sick.

The Kindergarten student has been hospitalised five times due to chest problems and bowel issues, and visits Lady Cilento Children’s Hospital about every eight weeks for check-ups.

“He would have a lot more flexibility with his lifestyle: not having to stop in the middle of class to take salt tablets, not having to leave for two weeks and putting everything in his life on hold to go to hospital,” Ms Martin said.

Then there is the profound effect the drug could have on the length of Alexander’s life.

“It could increase by 23 years, approximately, which is huge,” his mother said.

“He would have time to do everything he could possibly want to do with his life; even have grandchildren.”

Along with signing petitions for Orkambi and Kalydeco – another Vertex-developed drug which is suitable for a smaller percentage of the CF community – Alexander’s family has fundraised for Cystic Fibrosis Queensland in conjunction with the Moree Rugby Union Club, where they raised $26,000.

The Pharmaceutical Benefits Advisory Committee (PBAC) will soon consider submissions from the drug manufacturer’s Vertex to list Orkambi on the PBS.

Vertex has indicated this will be its final attempt to negotiate, and there are fears the company will leave the country altogether.

“If Vertex pulls out of Australia, we don’t just lose access to drugs but also to Vertex’s clinical trials here in Australia,” Cystic Fibrosis Australia chief executive Nettie Burke said. 

“Knock-back after knock-back because of price has worn everyone down …

“Our government needs to know we are dissatisfied with their efforts to negotiate so far and Vertex needs to understand that the ‘drug hold back’ and unreasonable pricing is cruel and unconscionable.”

A rally was held in Sydney in recent days to show support for the “wonder drug”.

Source: Cystic Fibrosis Australia