Cystic fibrosis: Trikafta means Rotorua mother goes from preparing for her son’s final days to planning his 21st

Rotorua cystic fibrosis sufferer OJ Daniels will be celebrating his 21st birthday in February - a milestone his mother, Trish Daniels, did not think he would make. Photo / Andrew Warner

OJ Daniels can now walk without struggling to breathe, Jessie McKay can now plan for a future, and Isaac Tait now has “more time”. These Bay of Plenty residents have cystic fibrosis - a debilitating genetic condition that threatened to cut their lives short were it not for a drug called Trikafta. Eight months after Trikafta was made publicly available in New Zealand, journalist Megan Wilson speaks to people whose lives have been changed by the “miracle” drug.

Within two years, Rotorua mother Trish Daniels went from preparing for her 19-year-old son’s “last days” to planning his 21st birthday.

Less than two years ago, Trish said her son, OJ Daniels, wouldn’t reach the age of 21 if he didn’t start taking a “miracle” drug called Trikafta.

Now, they are planning a “huge celebration” for the milestone birthday in February.

OJ has cystic fibrosis - an ultimately terminal condition that produces thick and sticky mucus and mainly affects a person’s lungs and digestive system. It affects about 540 people in New Zealand.

Trikafta treats its underlying cause but at $330,000 per year, the drug had been out of reach for many families as it had previously not been publicly funded in New Zealand.

Several Bay of Plenty families called Trikafta a “miracle” drug.

In June 2022, OJ was approved for free access to Trikafta through a compassionate managed access programme run by the drug’s supplier and manufacturer, Vertex, and has been taking it since.

Good news followed for other cystic fibrosis sufferers in New Zealand when Pharmac announced in December 2022 that funding for Trikafta was proposed to start on April 1, 2023.

The news was met with elation, happy tears and celebratory cake and champagne by cystic fibrosis sufferers in the Bay of Plenty.

In March 2023, Pharmac confirmed the funding of Trikafta for people with cystic fibrosis, aged 6 and over , who met certain eligibility criteria.

From “preparing for his last days” to planning his 21st birthday

OJ Daniels pictured in April 2022 before being approved for access to Trikafta. Photo / Andrew Warner

Trish Daniels told the Rotorua Daily Post that Trikafta had been “absolutely life-changing” for OJ.

“We thought we had lost the battle for a moment there - it was quite a difficult time just before finding out he was getting on Trikafta. We were preparing for his last days.

“But now ... he’s going to the gym, he’s gone out on dates ... "

She said Trikafta had helped OJ put on weight, going from 48kg before Trikafta to 60kg now.

OJ Daniels said he could now walk "without struggling to breathe". Photo / Andrew Warner

Trish said OJ hoped to get his first job next year.

“His whole outlook on life now has changed ... It’s just the best thing that could have ever happened to him.”

OJ said some of the highlights over the past 18 months included “going out more often” such as walking and going to the beach, “without struggling to breathe”.

“Before I couldn’t even walk around the corner, now I walk basically anywhere and everywhere.

“I went nightclubbing for the first time ... that was very fun.”

A new job, saving for a home and “starting to think about the future”

Tauranga woman Jessie McKay, who also has cystic fibrosis, graduated from her flight attendant training in May last year and has been working on transtasman routes since.

“It all happened because of the drug [Trikafta]. I got the job because of it and then everything was falling into place. Everything is so normal ... I don’t even have to think really about my health as much,” the 22-year-old said.

“Since being on the drug, I really haven’t got sick that much”.

Tauranga woman Jessie McKay, pictured in New York, is now working as a flight attendant.

McKay, who now lives in Auckland, said she had visited family in Brisbane and friends in Sydney through work.

“It’s so nice to go over and see them and it [being] a part of my job almost.”

McKay said she had always lived “in the now” and hadn’t planned for the future.

“I’m starting to save to buy a home when I’m ready. I’m starting to think about the future a bit more which I kind of stopped myself from doing for a long time.”

McKay said she and her family had “peace of mind” by her having Trikafta.

“We all can just live a little more stress-free.”

A burden has been “lifted”

Isaac Tait's parents were paying almost $400,000 per year for Trikafta before it was publicly funded in New Zealand. Photo / Andrew Warner

Whakatāne father Isaac Tait, who has cystic fibrosis, said Trikafta being funded in New Zealand was a burden being “lifted” for him and his family.

Tait’s parents, Jenny and Peter, had been paying $396,000 per year for Trikafta before it being publicly funded in New Zealand.

The 42-year-old said it meant he could do “a lot more planning”.

“I’ve also been really focused on my family as well. I got married just over a year ago, I’ve got a 2-year-old so he takes up a lot of time.”

Tait said he and his wife, Britney, were hoping to have another child soon and wanted to travel.

“It’s just given us more time.”

What’s next?

Cystic Fibrosis New Zealand chief executive Lisa Burns said “some significant and historical progress” had been made with the funding of Trikafta.

She said people who had started taking Trikafta were adjusting to thinking about their futures.

“Many have not had to consider there was a future due to missed education, employment, and relationship opportunities.”

Others required further support for their mental health and wellbeing, she said.

Burns said the charity was supporting and advocating for those who did not currently have access to a therapy that treated the cause of their cystic fibrosis.

“For some, it’s because they cannot tolerate Trikafta while for others it’s because modulator therapies simply don’t work for the genetic mutations they carry.

“Our immediate focus is working with Pharmac to widen access to Kalydeco to those who currently fall outside the eligibility criteria.”

Burns said about 360 people with cystic fibrosis could access Trikafta and about 30 people were taking Kalydeco - a modulator therapy funded by Pharmac in 2020.

Pharmac’s director of advice and assessment and chief medical officer, Dr David Hughes, said Ivacaftor - branded as Kalydeco - was a treatment for cystic fibrosis that Pharmac funded for people who met certain criteria, including the patient being 6 years or older with specific gene mutations.

Hughes said an application for widened access was received by clinical advisors on the Respiratory Advisory Committee. It was recommended with high priority for funding.

The next step was to assess the application and rank it on the options for investment list.

“We understand more people would like to access this treatment … We work hard to fund as many treatments as we can for people in New Zealand from within our fixed budget.”

Hughes said Pharmac was in regular contact with Cystic Fibrosis New Zealand regarding the status of the application.

The agency could not give a definitive timeframe for if, or when, funding decisions would be made as the priority of funding applications change over time.

Hughes said 328 people had accessed Trikafta as of the end of September.

“We understand how much of a life changer this treatment has been to the New Zealanders living with cystic fibrosis and their families, and we’re very grateful to have been able to fund this medicine from our fixed budget.”

Megan Wilson is a health and general news reporter for the Bay of Plenty Times and Rotorua Daily Post. She has been a journalist since 2021.