Tauranga boy pleads for Trikafta drug to ease cystic fibrosis

Harry Oxenham has cystic fibrosis and hopes a treatment drug will change his life. Photo / George Novak

The humming of the machine pumping evaporating medicine into Harry's lungs is monotonous.

It's a little like the sound of a dishwasher or the

washing machine.

The sound is relentless - constantly filling the silence of thought as Harry's parents, Aly, Dan and I talk.

Ironically, relentless is how Aly describes the half-hour treatment regime that sees Harry using three different mouthpieces, to achieve three different results for the sticky mucus on his lungs, otherwise known as cystic fibrosis.

No doubt you get used to it.

Although he might not have to if Trikafta - a breakthrough treatment - was registered and funded in New Zealand.

Cystic Fibrosis NZ says the drug is widely heralded as having the potential to turn cystic fibrosis from a life-threatening condition to a manageable condition.

At more than $470,000 a year, the drug is simply not attainable for many with cystic fibrosis.

Harry Oxenham using a nebuliser to turn his liquid medicine into a mist. Photo / George Novak

Therefore, 6-year-old Harry Oxenham has had to get used to the regime; it's how he starts his day, and how he ends it.

But he tells me he doesn't mind because at least he can go on his tablet to play games at the same time.

It's a similar routine to Rotorua's Leah Rumney, who last week told the Rotorua Daily Post Weekend of her wish to one day live a "normal" life with the help of Trikafta.

As hypertonic saline works to pull liquid into Harry's lungs making the mucus thinner, including through his sinus where it can be thick like toothpaste - just another complication of his condition - Dan tells me the first year of Harry's life was "pretty rough".

The common heel-prick test didn't pick up his condition but months of constant sickness and the "failure to thrive" meant his parents were growing more concerned.

The pressure was put on GPs to determine why baby Harry was struggling and eventually a sweat test was completed to check the level of salt coming out of Harry's pores, something that is generally high in cystic fibrosis patients.

"Just to rule it out, that was their words, but it didn't rule it out," Dan said

"It ruled it in."

There are three different rounds of treatment Harry undertakes morning and night. Photo / George Novak

Cystic fibrosis is an inherited life-threatening disorder that damages the lungs and digestive system. It affects the cells that produce mucus, sweat and digestive juices, causing these fluids to become thick and sticky.

They then plug up tubes, ducts and passageways - making it difficult, sometimes impossible - to breathe.

The diagnosis came with some sense of relief because now Dan and Aly knew what was wrong with their baby boy.

But, it was life-changing too, Aly said.

"When you have a child you instantly, even before they are born, start dreaming of what they're going to be, the hope and aspirations.

"Then suddenly you're landed with this and it's like 'wow'. Then you learn of the life expectancy and it's the worst day ever."

In New Zealand, there is no accurate data on average life expectancy but respiratory specialists believe it to be currently in the mid- to late-30s.

In some cases, it is a lot lower.

Overseas, Trikafta is only given to patients over the age of 12.

Harry Oxenham, 6, thinks cystic fibrosis "sucks" although the constant smile on his face would make you think otherwise. Photo / George Novak

Aly hopes that in the six years before Harry to be able to take it, the drug will become accessible in New Zealand.

"If Trikafta was available and funded and accessible, the future would look bright, and he would live a long fulfilled life."

It means the dreams she had while carrying her baby have the potential to come true.

She points out the cost of medical equipment, syringes, medical consumables and hospital admissions that Harry alone brings with him.

Harry has had more than 20 general anaesthetics in his short life already, she said.

"All of that cost, and he is a mild case of cystic fibrosis, so the cost of that and the burden on the health system versus the investment of $400,000 plus per patient, the Government would be able to balance it out."

But it's not the only cost that comes with Harry's diagnosis.

"The cost to us as a couple, as a family, quality of life. There are so many things we have to be aware of so if you are out with a family you are dealing with a sibling that can and a sibling that can't.

"It adds another dimension to parenting and family life."

Harry is starting to take control of his treatment regime, aside from the sterilisation of everything which takes another half-hour. Photo / George Novak

Cystic Fibrosis New Zealand chief executive Jane Bollard says it simply: "Trikafta should be publicly accessible to all Kiwis who could benefit.

"We would like to see Trikafta funded in New Zealand without further delay. Kiwis with cystic fibrosis can't wait any longer."

While Pharmac is aware of the "keen interest" in the drug, it says there is nothing it can do until a funding application is made by the supplier of the drug, Vertex.

Bollard said she had secured the agreement of the key stakeholders to attend a facilitated meeting with Cystic Fibrosis New Zealand, and Trikafta For Kiwis, in March this year, after months of discussions.

Bollard hoped all the organisations could work together to identify a pathway to public funding of Trikafta in the shortest possible time.

An application for registration has not been submitted to Medsafe for Trifekta, nor has a funding application been received by Pharmac.

Pharmac director of operations Lisa Williams said she was aware of the keen interest in the treatment and had invited Vertex, on a number of occasions, to apply to Medsafe for registration and to Pharmac for funding.

That way it would be able to be marketed and sold in New Zealand.

"Pharmac can't start our assessment whether or not the medicine should be funded out of our fixed budget without detailed evidence and information from the supplier including a price offer.

"We understand from media reports, and from people in New Zealand that are self-funding the medicine, that Trikafta costs approximately $470,000 per year per person, and that there would be approximately 450 people in New Zealand who could benefit from this medicine."

This means pre-any discounts Pharmac might be able to negotiate this medicine would cost New Zealand $211.5 million a year, Williams said.

It would be about 20 per cent of Pharmac's current fixed annual budget.

"We understand and appreciate that cystic fibrosis has a significant and distressing impact on those who have it and their whānau.

"We know funded access to effective medicines is important to everyone – it's important to us too because we strive to get the best health outcomes for all New Zealanders from the medicines we fund in a fair and equitable way."

Vertex, in a supplied statement, confirmed it was looking forward to continuing discussions around the future of Trikafta in New Zealand.

"The process of registration and funding of medicines in New Zealand is complex with a number of pathways available to companies for registration, each with differing review timelines.

"The applications are always complicated and time-consuming, requiring extensive data and clinical trial information and it is important that our application is thorough," the statement read.

Vertex said it recognised the frustrations of those still waiting for their chance at a new life through the help of Trikafta.

"While there are set processes that must be followed, we are open to discussing creative and innovative ways for people living with cystic fibrosis to access our treatments in New Zealand."