Family appeals for access to new drug

Heath Weir looks and acts like a normal seven-year-old but a rare genetic disorder has meant his life is much tougher than most kids his age.

He suffers from cystic fibrosis, a disease that has recently become the battleground for a dispute between sufferers and the Federal Government’s Pharmaceutical Benefits Advisory Committee.

In December last year, negotiations stalled between PBAC and Vertex Pharmaceuticals, which has developed a new drug, Orkambi, to treat the underlying cause of CF.

Heath’s mother, Allison Bruce, and thousands of families across the country are calling on the Government to fast-track access for the expensive drug in Australia, which is available in the US and Europe. “I look at this and I see a future in my son,” she said.

“When I discovered the drug and started reading about it I was excited because I’ll try anything to give Heath a better chance at life.”

CF affects every organ in the body but is mainly present in the lungs, where it leads to a build-up of sticky mucus that can cause infection, frequent hospitalisation and permanent lung damage.

Current treatments include a punishing regime of daily exercise, nebulisers and up to 40 tablets a day, which Ms Bruce says leaves Heath feeling exhausted.

Cystic Fibrosis WA wants the Government to make Orkambi available now and negotiate the price later, as has been done in Germany.

CFWA chief executive Nigel Barker said access to the drug was the first step in helping people who suffer from the disorder to limit the damage it causes.

“Research has shown that 25 per cent of patients who experience exacerbations never regain their previous lung function levels,” he said.

“So any delay in accessing Orkambi can have significant lifelong repercussions.

“We have seen from the trials that (this drug) can significantly reduce exacerbations for some CF patients who, like Heath, carry a particular gene mutation.”’

A rally was to take place last night outside Federal Parliament in Canberra, where hundreds of families were expected to call for access to the drug. Federal Health Minister Greg Hunt was approached for comment.