Young children suffering permanent damage from cystic fibrosis (CF) may soon be able to access a life-changing medication after Pharmac prioritised Trikafta for funding.
Some Kiwi families have left New Zealand in order to afford the drug for their preschool-aged children, which is not currently funded for people under the age of 6.
Trikafta, described 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, per year.
Pharmac released its Pūahoaho Report earlier this week, revealing Trikafta has been placed on the Options for Investment (OFI) list - though its ranking on the list remains a secret.
“This is tremendous news,” said chief executive of Cystic Fibrosis New Zealand, Lisa Burns.
“It is such an incredible thing to see Pharmac prioritise Trikafta for 2-5-year-olds. Obviously, a big part of that was helping them understand the impact that the medicine could have for this group of people.”
Pharmac receives applications to fund certain medicines, then assesses each application and places them on various lists of priority. Those on the OFI list are ones that Pharmac wants to fund if it can be done within the budget.
“It’s kind of the last stage of the process before any kind of funding gets announced or negotiated. It’s the best possible outcome that we could have hoped for,” Burns said.
“It’s another positive step in the right direction for the application, so yeah, it’s moving at a really good pace, which is what we wanted to see.”
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.
For mum Kayla Delaney, the news that her newborn baby had CF was “devastating”.
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 get 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 eats to help her body digest food properly. Before going on Trikafta, she went on 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 Adalyn was “doing amazing” and the family was happy they had made the move to get her the medication.
Pharmac began funding Trikafta for people over 6 in April 2023, and did not fund it for younger people because the medicine was only approved for that age group then. Trikafta received Medsafe approval for younger children earlier this year.
As of last week, there are 115 applications on the OFI. Trikafta is one of 10 new proposals on the list. There is no definite timeframe for when or if the medication will receive funding.
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.