Pharmac has reached a provisional agreement with medicine supplier Vertex to fund Trikafta, a life-extending drug for those with cystic fibrosis.
Cystic fibrosis is an ultimately terminal condition that affects about 540 people in New Zealand. Specialists believe the average life expectancy of a person with cystic fibrosis is mid-to-late 30s.
But at $330,000 per year, the drug was out of reach for many families as it had not been publicly funded in New Zealand.
That changes today.
Te Pātaka Whaioranga Pharmac’s director of operations Lisa Williams announced, following its budget increase in May and commercial negotiations with the supplier, Vertex, that it was initiating consultation on the funding of Trikafta for people aged 6 and above with cystic fibrosis who meet certain eligibility criteria.
“Trikafta has the potential to make a substantial difference to the lives of those people in New Zealand living with cystic fibrosis and for their whānau and communities too,” she said.
“We estimate that, if funded, Trikafta could give people with cystic fibrosis up to 27 more years at full health, when compared with supportive care. This would significantly reduce the impact of the condition on people with cystic fibrosis, their whānau and communities.”
Williams said Pharmac had been actively working towards this step in the funding process since receiving recommendations from its Respiratory Advisory Committee and its Pharmacology and Therapeutics Advisory Committee that the medicine should be funded.
“Our expert advisors have told us that treatment with Trikafta significantly improves lung function and other symptoms of cystic fibrosis. The evidence is clear that Trikafta can benefit people with cystic fibrosis, so we are pleased to start consultation on a proposal to fund it,” she said.
“I want to acknowledge the time that many people have put into advocating for those living with cystic fibrosis and to those who have shared their own very personal stories with us. We have heard them all, and we know there will be a lot of people thrilled to hear that we are proposing to fund Trikafta.”
The consultation was available on the Pharmac website for anyone wanting to have their say.
Williams said the consultation was “a very important step” in the process.
“It’s how we check that the people who will get the most benefit from the medicines will be able to access them. If approved, we will also be working closely with our colleagues across the health sector to plan for the implementation of Trikafta.
“We have been saying that we wanted to fund Trikafta - and now we are a step closer.”
Funding of Trikafta was proposed to start on April 1, 2023.
Cystic Fibrosis New Zealand chief executive Lisa Burns said “there just aren’t the words to convey what this means for our community.”
“Funding Trikafta will bring life-changing benefits to people with cystic fibrosis, their families, whānau, the wider community, and our health system.”
Health Minister Andrew Little said he was pleased Trikafta could soon be available for New Zealanders in need.
“I know how important this treatment is for people with Cystic Fibrosis, I know what a radical change it means for their lives,” he said.
Little revealed the Government had stepped in financially to help Pharmac secure the deal after assistance was requested a couple of months ago.
“[Pharmac] got to the point where they considered they were close to a deal, but it required a small amount of additional support we’ve now provided.”
He added it was his understanding Pharmac’s agreement also included other cancer treatments, which was another incentive for the Government to provide the necessary financial support to secure the deal.
Little wouldn’t give the exact number that was signed off by Finance Minister Grant Robertson, except to say it was in the millions and significantly lower than the $191 million the Government invested in Pharmac’s medicines budget for the next two years.
On Pharmac reaching a provisional agreement, Little said it indicated Pharmac had listened to calls to pay closer attention to rarer disorders.
The stories of those living with cystic fibrosis
In April, Tauranga father Glenn Ford spoke to the Bay of Plenty Times about his 11-year-old son Charlie who has cystic fibrosis.
Through tears, Glenn said it was “really cruel” Charlie had been dealt the card of cystic fibrosis. Without Trikafta, “his life will just be cut short”.
He worried about Charlie’s future without it.
“Will he get a job? Will he get married? Will he have his own family?”
Charlie said living with cystic fibrosis “sucks” because it meant constant hospital visits, spending hours taking medication and missing out on several school camps due to being sick.
Charlie used a nebuliser daily which contained a saline solution that loosened phlegm in his lungs so he could cough it up.
He just wanted to be “normal” and said Trikafta would allow him to “breathe better”.
“It will save more lives and most of us can just be normal.”
Natalie Wineti spoke to the Rotorua Daily Post in October about living with cystic fibrosis, as her doctor told her to consider getting a lung transplant because the organ’s function was declining.
The 38-year-old said the solution was Trikafta which would mean she would not need a transplant.
“I can only imagine what my future could look like on Trikafta,” she said at the time.
“My life is literally in Pharmac’s hands and it’s got the power to end the pain and suffering.”
Wineti, who is married with a 10-year-old son and two stepchildren, is a fulltime social worker, covering the Bay of Plenty, Taupō, Waikato, Hamilton and Te Aroha.
“I’m up at about 5am every day to start my treatments before I head off to work.”
Wineti takes more than 40 tablets per day and uses inhalers and nebulisers.
“It’s really time-consuming and exhausting but necessary for the sake of my health,” she said.
“After my treatments are done, I get to work, 9am to 5pm, and then return home in the evening to do those treatments again for an hour and a half.”
Wineti said life with cystic fibrosis was “exhausting” as she struggled to breathe, talk and walk.
“I spend hours and hours awake at night just coughing so hard to try to clear the secretions that are built up in my lungs.
“By the time I’ve cleared enough to be able to fall asleep, it’s time to get up for work. It’s never-ending.
“There is a miracle drug out there that can help people with cystic fibrosis maintain a quality of life - it’s so life-changing.
“It would make a world of difference for everybody with cystic fibrosis because we want to live and grow, not just survive. Because that’s all we’re doing, we’re surviving.”