Kiwis living with cystic fibrosis are celebrating a major milestone in the fight for access to life-saving medicine after pharmaceutical company Vertex submitted its application to have the drug Trikafta funded in New Zealand.
Cystic fibrosis is a genetic disorder which clogs up the lungs with mucus, making it hard to breathe. More than 500 New Zealanders are living with it and until now, it was effectively a death sentence - those with it usually don't live past 40, and often die as teenagers.
Ed Lee has been campaigning hard to have the drug made accessible in New Zealand.
"We have been battling for this application for a year now, and with this news it brings a sense of hope and joy that our community will finally be able to live long and healthy lives."
Lee has been taking 'miracle drug' Trikafta for more than a year. He's been funding it himself, at an extraordinary cost of more than $400,000 per year.
But this entire time, he's been fighting to have the drug for all Kiwis who need it and says tonight he will be celebrating with a bottle of champagne.
Pharmac says it is beginning to assess the medicine, starting today.
"Before we can fund any medicine, we need advice from our clinical experts, a deal with the medicine supplier and enough money in the budget to fund it now and in the future," says Pharmac's director of operations Lisa Williams.
Pharmac has also acknowledged it is one of the most expensive applications the organisation has ever received.
"If it makes it to our options for investment list, we'll aim to fund it when we have money available in our budget."
And if there is no money in the budget, the agency has previously committed to Newshub it would be willing to go to the Government for more money.
Drug-maker Vertex said in a statement, it's willing to work collaboratively and flexibly with Pharmac as the application moves forward.
"Vertex has made significant progress in New Zealand over the last few months and is committed to ensuring all eligible patients have sustainable access to our triple combination treatment as soon as possible".
Cystic Fibrosis New Zealand CEO Jane Bollard is very pleased with the announcement and says she looks forward to Kiwis with CF having access to Trikafta as soon as possible.
"This is a major step forward in securing access to Trikafta for Kiwis with cystic fibrosis."
Medsafe is currently reviewing Trikafta for use in New Zealand.
The drug has been given priority review status, a process reserved for medicines that offer "a significant clinical advantage or significant potential cost savings for the taxpayer."
The application will also be processed faster than usual at Pharmac too.
Standard practice is to wait for approval from Medsafe before the Pharmac process can begin. But rather than waiting, both applications will be considered in parallel.
Pharmac says the scientific evidence will be assessed in the "next few months" before a recommendation is made on whether Trikafta should be funded.
"If they recommend that it should be funded, we will undertake our economic assessment of the medicine and compare and rank it against all other medicines awaiting funding."
