Life-changing medicine approved

By Lachlan Ellis

A treatment for those with cystic fibrosis dubbed a ‘miracle medication’ will be subsidised starting next week, saving families hundreds of thousands of dollars.

The Federal Government announced earlier this month that the drug Trikafta would be listed on the Pharmaceutical Benefits Scheme (PBS) for eligible children aged 6 to 11 years from 1 May – cutting the cost from up to $250,000 a year, to just $30 per script.

Eight-year-old Ben McLean from Bacchus Marsh has the condition, and his parents Angus and Ginnie are two of the many Australians who have backed the campaign to get Trikafta subsidised.

Last year the medication was approved for the PBS, but only for those aged 12 and older – leaving younger people like Ben still unable to access it.

Angus McLean said the announcement was something that his family, and families across the country, have been eagerly awaiting.

“Early prevention for cystic fibrosis is the key factor in keeping kids out of hospitals, and reducing damage to their lungs. This new drug is the best one that’s been conceived yet for the majority of people with CF, and it’ll do exactly that – reduce lung damage, and keep them in the best possible condition they can be in,” Mr McLean told the Moorabool News.

“We’ve been anxiously waiting, there’s 33 countries that have already added this to their government-funded medication list, including New Zealand – so Australia was pretty slow to jump on it, but we have now, which is fantastic. I think there’s about half a dozen eligible kids between Ballan up to Ballarat.”

Peak patient body for people with cystic fibrosis, Cystic Fibrosis Australia, says 530 children have been waiting for the PBS listing, with the private cost of Trikafta making it inaccessible for the majority of families.

Cystic Fibrosis Australia CEO Jo Armstrong said they can celebrate as more people can access transformational therapies and as a result will have longer, healthier lives.

“This is a huge win for the cystic fibrosis community. There are over 500 children that will benefit from this decision,” she said.

“We have been advocating for this all year and I am so grateful that all stakeholders have accelerated the process. I had grave fears it could take 6 months or longer for the PBS listing and I sincerely thank the Minister for Health, Mark Butler, MP, Vertex Pharmaceuticals, the PBAC, our Federation members, and everyone in the community who has been advocating.”

Cystic fibrosis is a progressive, genetic disease that causes persistent lung infections, and is caused by genetic defects limiting the flow of chloride and water through cell membranes, creating a build-up of mucus in the lungs, pancreas, and other organs.

Trikafta combats this by improving chloride and water flow in patients with a certain genetic defect, helping improve lung function and breathing.

There is no cure for cystic fibrosis, and over one million Australians carry the cystic fibrosis gene. Every four days a baby is born in Australia with cystic fibrosis – 1 in every 2500 babies.