Deal on cystic fibrosis drug nears
Trikafta to add decades to sufferers’ lives
Pharmac has reached a provisional agreement with medicine supplier Vertex to fund Trikafta, a lifeextending 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 midto-late 30s.
Trikafta treats its underlying cause and has previously been described as a “miracle drug” by cystic fibrosis sufferers and their families.
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 changed yesterday. Te Pa¯taka 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 wha¯nau 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 wha¯nau 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.
Williams said the consultation was “a very important step”.
“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 the funding “will bring life-changing benefits to people with cystic fibrosis, their families, wha¯nau, 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.
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 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 $191m 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 wanted to be “normal” and said Trikafta would allow him to “breathe better”.
Natalie Wineti spoke to the Rotorua Daily Post in October about living with cystic fibrosis. Her doctor told her to consider getting a lung transplant as the organ’s function was declining.
The 38-year-old said the solution was Trikafta which would mean she would not need a transplant.
Wineti, who is married with a 10-year-old son and two stepchildren, is a fulltime social worker, covering the Bay of Plenty, Taupo¯, Waikato, Hamilton and Te Aroha.
Wineti takes more than 40 tablets a day, and inhalers and nebulisers.
“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.”