A family considering leaving New Zealand to access a life-changing medicine for their toddler can now stay home after Pharmac proposed funding the “miracle drug” for younger children.
The 20-month-old child’s father said he was “nearly in tears” at the news that Pharmac had made a provisional agreement to fund Trikafta for children aged 2-5 with cystic fibrosis (CF).
Trikafta, described by some as a “miracle drug” and by one user as “the most incredible medical breakthrough that we’ve had for cystic fibrosis”, is not a cure for the condition, but treats the cause instead of symptoms and is estimated to extend users’ life expectancies by decades.
The previously reported unfunded cost of Trikafta per person in New Zealand was about $330,000, excluding GST, a year.
CF is a genetic condition that causes the body to produce a thick, sticky mucus, causing obstructions primarily in the lungs and pancreas. Those with the condition are more susceptible to lung damage and infections, and issues with their digestive system.
Pharmac’s Pharmacology and Therapeutics Advisory Committee previously said Trikafta could add 27 years to someone’s life. While previous estimates have put the average life expectancy for people with CF in the 30s, updated figures in Australia, where more medications have been funded for longer, are closer to the mid-50s.
The drug has been available to people aged 6 and over since April 2023, but younger children will soon be able to access it from April this year, subject to public consultation.
“This proposal is game-changing for families,” said Simone Brown, executive lead for Cystic Fibrosis New Zealand (CFNZ). “It will give our young children living with CF the best possible start in life, and hope to parents who have been nervously awaiting this moment.
“We know of at least five families who have moved to Australia or further overseas to access Trikafta for their young children. This proposal will enable those with young children with CF to remain in New Zealand and provide the potential for those who have already left to reunite their families. We also welcome Pharmac’s proposal to fund Trikafta so it can be used as soon as clinically appropriate, regardless of age.
“Public consultation is the final hurdle to achieving access for our community, and we are calling on all New Zealanders to make a submission to Pharmac to ensure this proposal becomes a reality.”
Pharmac’s proposal also includes plans to widen access to the currently funded Kalydeco and provide a new modulator therapy, Alyftrek.
“CFNZ’s goal is that as many people with CF as possible have access to a medicine that treats the cause of this devastating and life-limiting condition. Pharmac’s proposal is a major step towards achieving that.”
Christchurch couple Edward and Emily Harrington-Ashton have a 20-month-old son, Noah, who has CF. They said access to the medication would be life-changing.
“I was almost in tears when I got the news. It was a relief more than anything, to be honest,” said Edward.
“We had considered moving to Australia and leaving behind our families and support networks to get access to Trikafta for Noah. It’s a massive relief that we can stay where our comforts are and give Noah the best chance possible for a long and happy life.
“We’re extremely happy and want to pass on a huge thanks to everyone involved in making this happen.”
Public consultation is underway and closes at 5pm on February 11.
CFNZ said it was seeking support “in getting Pharmac’s proposal across the line and Trikafta into the hands of our little ones”.
The Herald previously reported on one Kiwi family who moved to Australia to access the medication.
Adalyn Delaney was suffering the effects of CF before she was even born, with health staff discovering during a scan that she had a bowel obstruction. The moment she was born, she had to be rushed away to surgery.
Adalyn had to have two surgeries within her first five weeks of life, and another at the age of 1 to have scar tissue removed. She has been hospitalised multiple times, including for pneumonia, a collapsed lung and another bowel obstruction.
She had to go on a nebuliser (a mask-like device that turns medicine into a vapour she can inhale) three times a day when she was healthy, and had to take medication when she ate to help her body digest food properly. Before going on Trikafta, she needed antibiotics every time she got a cough.
The family moved to Queensland in June to access Trikafta, which is funded for small children in Australia.
Delaney recently told the Herald that Adalyn was “doing amazing”, and the family was happy they had made the move to get her the medication.
Meanwhile, Hutt Valley woman Hannah Meates went on Trikafta a couple of years ago.
“Everybody should be able to have access to something like that if it’s going to literally save their life ... like, they can avoid all the lung damage and all of that stuff that’s irreversible,” she said.
“I think something like Trikafta is the most incredible medical breakthrough that we’ve had for cystic fibrosis.”
Melissa Nightingale is a Wellington-based reporter who covers crime, justice and news in the capital. She joined the Herald in 2016 and has worked as a journalist for 10 years.