Cystic fibrosis: Tauranga mother hopes Trikafta will be funded for pre-schoolers

Tauranga boy Riaan Hamilton has cystic fibrosis – the 3-year-old often gets sick, has been hospitalised several times, and is at risk of permanent lung damage.Children aged 6 and older have had access to a drug that can help manage the condition, Trikafta, for almost two years. Now younger children – like Riaan – could get it.

Khali Hamilton thought of the “worst-case scenario” when she found out her son Riaan had cystic fibrosis at 3 weeks old.

She recalled her mother babysitting two people with cystic fibrosis decades ago, both of whom died young.

But when she found out about the drug Trikafta, she started thinking, “we can manage this”.

Cystic fibrosis is a condition that produces thick and sticky mucus and mainly affects the lungs and digestive system. It is ultimately terminal.

Trikafta treats the underlying cause of the condition and makes it more manageable. It has been described as a “miracle drug” by cystic fibrosis sufferers and their families.

Tauranga boy Riaan Hamilton has cystic fibrosis. His mother Khali Hamilton hopes Trikafta will be funded after Pharmac's Respiratory Advisory Committee recommended funding Trikafta with high priority for children aged 2 to 5 years with responsive genetic mutations. Photo / Supplied

Pharmac started funding the drug for people aged 6 and up in mid-2023 and recipients have said it has helped them walk without struggling to breathe and embark on new careers.

Pharmac’s Respiratory Advisory Committee has now recommended funding Trikafta with high priority for children aged 2 to 5 years with responsive genetic mutations.

On antibiotics every month

Hamilton told the Bay of Plenty Times Riaan was “relatively healthy” but regularly got sick.

“At least once a month, he’ll be on two-week antibiotics.”

Riaan had been admitted to Tauranga Hospital a few times – most recently in September – for a “tune-up”.

“Usually, it’s when he’s had a bout of oral antibiotics, and he’s just got a cough that just will not budge. So, they’ll put him on IV [intravenous] antibiotics for two weeks and then intensive physiotherapy while in hospital as well.”

Tauranga boy Riaan Hamilton has cystic fibrosis and regularly gets sick. Photo / Supplied

Hamilton said his day-to-day life was normal except for regular testing and X-rays.

He had to go to hospital every time he had a cough and have a suction tube put down his throat.

“Those sorts of interventions and procedures ... really do affect him.”

Riaan’s daycare had been a “huge support” and involved his peers “to make him feel like he’s not left out or different”.

Trikafta would make things ‘a lot easier’

Hamilton said she could not afford to pay out-of-pocket for Trikafta, which the Bay of Plenty Times has previously reported costs individuals about $330,000 per year.

She said it would be “a relief” if the medicine was funded for children aged 2 to 5. She understood Riaan would be eligible.

Having fewer interventions and treatments, and replacing medications with Trikafta would be “a lot easier”.

“Any new cough or illness he gets, there’s that chance of his lungs scarring and causing permanent damage.

Khali Hamilton understands Riaan would be eligible for Trikafta if it was funded for children aged 2 to 5. Photo / Supplied

“The earlier intervention … the better, just so he has those healthier lungs as he gets older.”

Hamilton has two children aged 15 and 14 and was pregnant with her fourth child, who she said could possibly have cystic fibrosis.

A ‘vital step’ for people with cystic fibrosis

A Cystic Fibrosis New Zealand press release said the charity welcomed the latest funding recommendation for Trikafta.

Since being publicly funded in April 2023, Trikafta has been shown to significantly improve lung function, reduce hospitalisations, and enhance quality of life, it said.

Chief executive Lisa Burns said the recommendation was a “vital step” towards ensuring all New Zealanders with cystic fibrosis had fair and equitable access to a “life-changing” medicine.

She said Trikafta had been available for this age group for more than 18 months in over 20 countries.

The charity urged Pharmac quickly implement the recommendation, allowing young children with cystic fibrosis to start treatment without unnecessary delays.

“The earlier children receive Trikafta, the better their long-term health outcomes,” Burns said.

What happens next?

Pharmac published the record of its Respiratory Advisory Committee August meeting on January 23, which said Trikafta was recommended with high priority for the treatment of cystic fibrosis in children aged 2 to 5 with a “Trikafta-responsive” mutation.

The agency’s chief medical officer, Dr David Hughes, has previously said the next step after a recommendation was a review by Pharmac’s assessment team.

This covered factors for consideration including the health needs of people with the condition, the health benefit of the new medicine compared with current treatment, suitability of the new medicine, the health need for Māori and Pacific peoples and other priority populations, and cost.

Next, Pharmac staff discussed a summary of the application’s assessment against the factors for consideration and where on its Options for Investment list the medicine should be ranked.

The Options for Investment list is the list of treatments Pharmac would like to fund if it has enough budget.

When a medicine is high on this list, “we aim to fund it when we have available 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.