OJ Daniels, a 19-year-old living with cystic fibrosis, gets a one-month supply of Trikafta anonymously gifted to him.
News that public funding for the cystic fibrosis "miracle" drug Trikafta could be one step closer has been met with hope and excitement.
One Whakatāne cystic fibrosis sufferer says it would be a "totally different world" if the $330,000-a-year drug was publicly funded.
Cystic fibrosis is a condition affecting around500 New Zealanders and causes the body to create abnormally thick mucus. It blocks the tubes of the lungs, pancreas, liver, intestine and reproductive system, a Pharmac assessment of Trikafta summary said.
The estimated life expectancy of someone with cystic fibrosis is 37. There is no cure for cystic fibrosis, the summary said.
Trikafta is a treatment for cystic fibrosis and helps make the mucus thinner, stopping it from blocking the body's organs. It has been described by several members of the cystic fibrosis community as a "miracle" drug.
Yesterday, a Pharmac media release said it had received a clinical recommendation that Trikafta should be funded for people aged 6 and older.
Pharmac had already re-ranked Trikafta on its Options for Investment list following updated advice from its Pharmacology and Therapeutics Advisory Committee.
Cystic fibrosis sufferer Troy Watson said it was "wonderful news" and "a step forward".
Trish said OJ would spend anywhere between two and five weeks in hospital at a time.
"The cost alone is huge ... he's also got all the x-rays and the blood tests ... and all the care that has to go into treating him. It just reaches an extraordinary amount of money."
In her view, it was more cost-effective to fund Trikafta than to pay for a cystic fibrosis patient to go in and out of hospital.
Trish said if Trikafta ended up being publicly funded, it would be "about time".
"It's been a long journey for all the [people with cystic fibrosis] and for this to actually happen ... is basically saving lives and that's all we've wanted.
"I'm very happy. Hopefully, we certainly see Pharmac come to an agreement with Vertex and have this funded."
Glenn Ford pictured with his 12-year-old son Charlie Ford. Photo / Andrew Warner
Tauranga father Glenn Ford said it was "good to hear" but he had recently stepped back from advocating for Trikafta to be publicly funded. His 12-year-old son Charlie Ford has cystic fibrosis.
"I've just been concentrating on keeping my boy well."
Cystic Fibrosis New Zealand chief executive Lisa Burns said: "We are pleased to see that Pharmac has again made clear that it wants to fund Trikafta, in the light of all the evidence it has received."
In a Pharmac media statement, Pharmac director of operations Lisa Williams said Trikafta was a medicine it would like to fund and it had two proposals on its Options for Investment list - one for those aged 6 and over and one for those aged 12 and over.
"We now have recommendations from both our Respiratory Advisory Committee and from Pharmacology and Therapeutics Advisory Committee that the medicine should be funded for people 6 years and over."
Trikafta was last considered by the Pharmacology and Therapeutics Advisory Committee in November 2021.
Since then, Pharmac had received more evidence from Vertex and information from consumer groups and healthcare practitioners.
In April 2022, the Respiratory Advisory Committee reiterated its recommendation to fund Trikafta for people 6 years and over with "high priority".
In May 2022, the Pharmacology and Therapeutics Advisory Committee, which had previously deferred making a recommendation for people 6 and older, had now recommended funding for them with medium priority following consideration of the new evidence and information.
Pharmac also released its Technology Assessment Report for Trikafta - a health economic analysis used to determine the cost-effectiveness of a medicine. If Trikafta was funded, there would be some savings to the health system through the reduction of the need for hospitalisations, lung transplants and supportive care.
Pharmac had re-ranked Trikafta on its Options for Investment List having considered the updated advice from its clinical experts. It was continuing commercial discussions with Vertex to try to reach an agreement that would enable the funding of Trikafta.
