Trikafta funding gap for young children with cystic fibrosis needs filling – Editorial

Adalyn Delaney, 4, and her family have moved to Australia so she can get funded access to Trikafta to treat her cystic fibrosis.

Editorial

THE FACTS

• Kayla Delaney and her family moved to Australia for her 4-year-old’s access to the cystic fibrosis drug Trikafta.
• Trikafta is funded in New Zealand only for those over 6, leaving younger children without access.
• Pharmac is assessing funding for younger children but faces budget constraints and prioritisation challenges.

There is something very wrong when New Zealand can’t properly look after some of its most vulnerable and sick children.

This is the scenario that has played out for Lower Hutt mother Kayla Delaney and her family, who have had to move across the Ditch so their 4-year-old girl, Adalyn, can get a funded, life-changing cystic fibrosis drug.

Cystic fibrosis causes the body to produce thick, sticky mucus, causing obstructions that occur mainly in the lungs and pancreas. People with the condition are more susceptible to lung damage, infections and digestive issues.

There are about 550 people with it in New Zealand. Some estimates put the average life expectancy at 31. Children under 6 can suffer irreversible damage.

But one drug can make a dramatic difference.

Trikafta has been touted as a miracle drug, and NZME has widely reported on it.

It is not a cure, but it treats the cause instead of the symptoms. Importantly, it can improve quality of life and is estimated to dramatically extend life expectancies.

Only people over the age of 6 can get funded access because Pharmac has not approved it. Cystic Fibrosis New Zealand estimates that about 55 children would benefit from Trikafta.

The drug’s unfunded cost per person in New Zealand is about $330,000 a year.

Pharmac, an independent Government-funded Crown entity, decides which medicines and related products are funded.

It approved funding for people over 6 in April 2023 and a respiratory advisory committee recommended a year ago that it fund it, with high priority, for children aged 2-6 who met certain criteria. Medsafe approved this age group in March.

So, what’s the hold-up?

Pharmac says its teams “are working hard to finish our assessment of this application” and once that is done, it will compare it against applications for other medicines.

“We can’t say if or when a decision about funding this medicine will be made.”

At the same time, in Queensland, Delaney says her daughter has responded well to Trikafta. The family may stay there long-term because there is more funding for medicines that may help Adalyn.

She says it is disappointing that it is “taking so long” for the drug to be funded for young children in New Zealand.

It is disappointing. But this is not Pharmac’s fault.

Pharmac manages about $1.7 billion in taxpayers’ money to pay for medicines, vaccines and other related products.

However, its budget will never be enough to pay for all the drugs needed to help desperate Kiwis battling horrendous diseases and conditions.

Pharmac Minister David Seymour describes cystic fibrosis as a “terrible illness” and hopes the drug-buying agency makes “the right decision” on this issue.

He will argue for more Pharmac money in the next Budget.

This is encouraging, but it still feels like New Zealand is coming up short.

Could the Government dramatically increase Pharmac’s budget?

Of course it can.

It just needs to cut spending in other areas.

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