The miracle drug Trikafta is a step closer for New Zealanders living with cystic fibrosis (CF), after its US manufacturer announced it will fast-track an application to have it funded here.
It comes after 17-year-old Bella Powell and other sufferers made a desperate plea for the drug to be made accessible.
"There are more than 500 Kiwis with CF that this could benefit and then you've got to think about the babies being born with this - if they can have this [drug] they're not going to go through some of the struggles I have because my lung function started declining when I was 12," Powell told The AM Show on Monday.
Not subsidised by state drug-buyer Pharmac, Trikafta is owned by US company Vertex and has set its price tag at $469,000 per year.
Pharmac had said it could not consider funding it unless there was an application but on Monday evening, a Vertex spokeswoman said it was committed to exploring access to the drug for New Zealand patients.
"Our mission is to secure the broadest sustainable access supported by the scientific evidence available, to ensure the greatest value and benefit can be delivered to patients and [the] healthcare system," the spokeswoman said. "We plan to apply to the appropriate Government agencies, using their available pathways, to achieve Pharmac-funded access in the fastest possible way."
The move now puts the ball in Pharmac's court.
Sir Bob Elliot, who's spent a lifetime treating and caring for people with cystic fibrosis and bought Powell a three-month supply of Trikafra, told The Project on Monday the price of the drug was "unethical".
"I know they've done a fabulous job in coming up with this formulation, it's been a long haul for them and an expensive one. But as a practising doctor, it just upsets me that this marvellous treatment is being dangled in front of people, [saying] 'if you took this you'd be okay, but you can't afford it'," he said.
"My life has been built around trying to help people with the illness, but I'd do it for nothing.
"The money is very secondary to me. But obviously, it's not to a drug company who wants to not only recover the money they've spent developing the drug, but also wants to make a massive profit."
Powell's mum Alley said she doesn't have to worry so much since she started taking the drug.
"Every night I'd used to go to bed and I'd worry - that I'd wake up in the morning and we'd be worse," she told The AM Show on Monday.
"I just hope that by making this public that people understand how important it is and if you have a drug like this - you're taking people out of that hospital system," she told host Duncan Garner.
