Bella and Osiris, both 17 years old, were born with cystic fibrosis, a life-threatening genetic disease.

The disease affects the lungs, digestive system and other main organs as mucus builds up in the body blocking tubes, ducts and passageways. The life expectancy for someone with cystic fibrosis in New Zealand is mid-to-late 30s.

Both teenagers have a cupboard full of medication and have been confined to home or hospital for most of their lives. But only one of them has had the chance to try a life-saving drug.

Trikafta is a new drug manufactured by the US pharmaceutical company Vertex. Because it’s not funded by New Zealand’s drug-buying agency Pharmac, it costs $469,000 for one person for one year.

Bella was donated a three-month supply by cystic fibrosis expert Sir Bob Elliot, that he paid for himself at a cost of about $100,000.

“There’s an element of guilt there that I have it, yet there’s 500 other Kiwis who don’t and who I know are struggling,” says Bella.

Both Bella and Osiris are pleading with US drug manufacturing company Vertex and New Zealand drug buying agency Pharmac to do a deal which would bring the cost down.

“Without that drug,” says Osiris, “my life is gone.”

So what’s the hold up and what will happen to the price?

The drug is very new - it was only approved in the United States in October 2019. It was registered in Europe and the UK in August this year, and Australia is yet to register or fund it.

The first step to get it here would be for the US drug manufacturing company Vertex to apply for marketing approval to Medsafe, which is the part of the Ministry of Health that regulates the safety of medicines in New Zealand.

Vertex then needs to lodge an application with Pharmac and negotiate a deal for them to bulk-buy the drug.

Vertex said in a statement to Re: they “have had some early discussions with Pharmac and remain ready and willing to continue to engage with them.”

Pharmac’s director of operations Lisa Williams says they are “aware of the keen interest in this treatment and we’ve invited the supplier to apply for funding, however we can’t start our assessment of the evidence without detailed information from the supplier (including a price offer).”

“We’d like to assure you that Pharmac can, and will, move quickly to fund new medicines that show significant health benefits.”

Vertex did not release any details of what that price offer might be, however said their pricing would reflect “benefits to patients, healthcare systems and society, and our continued investment in developing new medicines for people suffering significant unmet health needs.”