Cystic fibrosis: Bay of Plenty families disappointed as Trikafta unlikely to be funded

Trish Daniels and her son OJ Daniels who has cystic fibrosis. Photo / Andrew Warner

The mother of a teen with cystic fibrosis says it is "absolutely gut wrenching" that "miracle" drug Trikafta is unlikely to be publicly funded.

Another family is considering moving overseas to where the drug is funded.

Yesterday, Cystic Fibrosis New Zealand said Pharmac was not in a position to progress funding on the life-extending drug Trikafta.

In a statement, it said it had been told by Pharmac that Trikafta had not moved up its 'options for investment' list. This meant it could not move forward with funding the drug without a change in factors influencing the decision.

Pharmac has said it still wants to fund Trikafta, subject to its budget. The ranking of treatments on its list was confidential so it could negotiate the best prices for New Zealanders.

Cystic fibrosis is a genetically inherited condition that produces a thick and sticky mucus that mainly affects a person's lungs and digestive system. Specialists believe the average life expectancy of a person with cystic fibrosis is mid to late 30s.

Trikafta treats its underlying cause but is not publicly funded in New Zealand and costs about $330,000 per year.

Last month, Pharmac said it had received a clinical recommendation that Trikafta should be funded for people aged 6 and older and had already re-ranked Trikafta on its list following updated advice from its Pharmacology and Therapeutics Advisory Committee.

Yesterday, Cystic Fibrosis New Zealand said Pharmac told it Trikafta was re-ranked again in September, but it had not moved high enough up the options for investment list for it to progress to funding at this time.

For it to be re-ranked, change would be needed to factors such as an alteration in the price offered by manufacturer Vertex or in Pharmac's budget.

Cystic Fibrosis New Zealand chief executive Lisa Burns said it was "shocked and outraged" at Pharmac's decision not to prioritise funding Trikafta.

"We have been left confused and don't have the words to convey our deep disappointment for our cystic fibrosis community."

Tauranga father Glenn Ford pictured with his son Charlie. Photo / Andrew Warner

Tauranga father Glenn Ford, whose son Charlie has cystic fibrosis, said he and his family were considering moving overseas where Trikafta was publicly funded.

"As soon as Charlie gets sick ... we'll probably pack up and leave because [in his view] there's no help here.

"I'm just so disappointed - I've just about given up."

Charlie had taken a lot of time off school and Glenn was not working so he could look after him.

"That little magic pill could stop all of that and get life back to a little bit of normal."

Cystic fibrosis sufferer Troy Watson who lives in Whakatāne. Photo / Andrew Warner

Whakatāne cystic fibrosis sufferer Troy Watson said the news was "disappointing".

"I didn't have much hope for it to come through because we'd had hope in the past and it hadn't happened."

Rotorua mother Trish Daniels' son OJ Daniels has been on Trikafta since June through a compassionate managed access programme run by Vertex.

Trish lost her daughter, Santana Daniels, to cystic fibrosis in 2017 and has previously told the Rotorua Daily Post she feared losing her son too. The moment he was approved for Trikafta was "life-changing".

Trish Daniels and her son OJ Daniels who has cystic fibrosis. Photo / Andrew Warner

Trish said yesterday's news was "absolutely gut wrenching" and "a huge disappointment" for the cystic fibrosis community.

"We're absolutely gobsmacked - I can't believe after all the progress that they did in August where everything was so positive ... I thought we [were] moving forward with this."

Trish said OJ had a "little slip-up" where there was a delay in getting Trikafta when he was renewing his prescription.

"He was off his Trikafta for 24 hours and he started coughing pretty much by the end of the afternoon.

"As soon as the Trikafta arrived ... within the 12-hour period he was back to normal."

This showed what a "huge difference" Trikafta made for OJ.

"To see him from literally not going to make it to the end of the year or to his birthday, to how he is now, it's extreme."

Trish said OJ had put on weight and his energy levels were "amazing".

"If he didn't have access to Trikafta ... we'd be in some pretty difficult stages in his life."

Trish Daniels and Glenn Ford have previously described Trikafta as a "miracle" drug.

In a statement, Vertex said it was "extremely disappointed" Pharmac decided not to prioritise funding of Trikafta.

"We understand that Pharmac must make choices within a fixed budget, about which patient groups will have access to new, innovative medicines," the statement said.

"We are surprised by this decision, particularly given their recent public acknowledgment of the clinical impact that Trikafta would have on cystic fibrosis patients in New Zealand."

Vertex would keep working with Pharmac to find a way forward so those who could benefit from Trikafta would have the same access as patients in more than 30 other countries.

Pharmac director of operations Lisa Williams said it understood the substantial health need of people living with cystic fibrosis and Trikafta's benefits.

"Trikafta remains a medicine Pharmac would like to fund, subject to available budget."

Williams said it kept the ranking of treatments on its options for investment list confidential for commercial reasons. This ensured it could negotiate the best prices so more New Zealanders could access the treatments they needed.

"We take our decision making responsibility seriously ... all Pharmac staff are motivated to fund as many products as we can to achieve the best health outcomes for New Zealanders."

Williams said there were 79 treatments on its list. After the budget increase in May, Pharmac was working its way through them.

So far this financial year, Pharmac had made 18 new investments - four new medicines and wider access to 14.

"And there are more to come."

Williams said it had met with Vertex to reiterate its commitment to making progress that would secure the public funding of Trikafta.