Teenager OJ Daniels wants to breathe without difficulty. Charlie Ford just wants to be a "normal" kid and run around with his mates. But neither can do these things because they have cystic fibrosis - a life-threatening, debilitating genetic condition. There's a ''miracle drug'' that can transform their lives
Cystic fibrosis: Rotorua mother 'desperate' for Trikafta funding
OJ Daniels lost his sister Santana Daniels to cystic fibrosis in 2017. Photo / Andrew Warner
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Trish's daughter Santana Daniels died from cystic fibrosis in 2017 when she was 21.
She worries OJ is now facing the same fate.
Trish and OJ are "desperate" to get Trikafta - a "miracle drug" which is not publicly funded in New Zealand and costs about $330,000 per year.
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"I lost my daughter to cystic fibrosis and if we don't get Trikafta I'm likely to lose my son,'' she tells the Rotorua Daily Post Weekend.
"If we don't get him on to Trikafta and if we don't get him on to the list for a transplant, he won't make his 21st."
Tauranga father Glenn Ford also has a son with cystic fibrosis.
Through tears, Glenn says it is "really cruel" his 11-year-old son Charlie has been dealt the card of cystic fibrosis. Without Trikafta, "his life will just be cut short," Glenn says.
He worries about Charlie's future without it.
"Will he get a job? Will he get married? Will he have his own family?"
These two families, and others, are speaking up and fighting for Trikafta - which treats the underlying cause of cystic fibrosis - to be publicly funded.
The Rotorua Daily Post spoke to Whakatāne brothers Ashley and Troy Watson, who both have cystic fibrosis. Ashley, 28, has access to Trikafta free through a compassionate managed access programme funded by the drug's manufacturer and supplier. But Troy, 20, is "too well" to be eligible and without government funding, there is "no chance" for someone like him.
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Cystic fibrosis is a genetically inherited condition that produces thick and sticky mucus and mainly affects a person's lungs and digestive system.
It affects about 540 people in New Zealand. Specialists believe the average life expectancy of a person with cystic fibrosis is mid-to-late 30s.
A petition calling for public funding of Trikafta was presented to Parliament last week, saying clinical trials have shown "significant improvement" in health outcomes for people with the condition.
Through tears, Trish told the Rotorua Daily Post Weekend that having a child with cystic fibrosis is "probably the worst thing" a parent could go through.
"It's one thing to lose one child but knowing there's a possibility of losing ... your youngest as well to cystic fibrosis when there's a drug out there that could save his life.
"I don't understand why the Government hasn't already stepped in," she says.
"I don't understand why we are standing around watching our young ones die - these are kids. Kids who are not making their 16th birthday, their 18th birthday, their 21st."
Trish says Trikafta would change cystic fibrosis from being a life-threatening illness to "manageable".
"He's spent 19 years like a prisoner.
"It gives them a life, a second chance."
Trish says with the amount of money spent keeping OJ "as well as possible" through hospital visits, staff and medication, "we could've had Trikafta a million times over".
"[The Government] needs to step in and help Pharmac fund Trikafta otherwise my son might not be here next year."
OJ's battle with cystic fibrosis
OJ says life with cystic fibrosis was "very restricting" as his immuno-compromised state made him "very vulnerable".
He mainly stays at home and spends several hours taking medication and coughing up mucus. He is underweight and has "quite low" energy levels.
OJ was admitted to hospital last month due to breathing difficulties, coughing and "massive" weight loss.
"I was very stressed out during that admission - very scared. I didn't think I would make it out alive."
He says Trikafta would "majorly" change his life as it would allow him to get a job and "experience what breathing feels like".
"Right now it feels restricting, but I heard it feels nice."
Six months ago, OJ tried to access Trikafta through a compassionate access programme through its supplier Vertex but was unsuccessful because he "didn't meet the criteria", Trish says.
Following his recent hospital admission, OJ will try again.
Trish says he is now in a position where his health is "just going to deteriorate from here onwards".
They are planning to meet doctors in Auckland next week to start talking about Trikafta and the option of a double lung transplant.
But there is no guarantee OJ would make the waiting list for a transplant.
"It could be a lot worse for him by waiting for a transplant, which is why we're urging for the Government to step in to try and help Pharmac fund Trikafta."
In Tauranga, Glenn is aware Vertex has a compassionate access programme for Trikafta.
In his view: "They won't give it to Charlie or anyone else at the moment until they're basically on their death beds.
"When they're really sick and there's no hope, they'll get it then."
This makes him "bloody angry".
"It brings tears to my eyes. Why should it have to get to that?"
Glenn says the family have not tried to access Trikafta because of the cost, but he knows it is available in the UK and from April 1, in Australia, and the family have considered moving to one of those countries.
"For us to be so close and to be here ... it makes you want to pack your bags and go there just so you know you can make your son well.
"If it got to the point of having to, we would definitely ... but it shouldn't have to get there.
He thought there was "hope" when Pharmac told them in October that Trikafta was a "high priority" for funding.
But it has since been "back-benched" when Pharmac announced in February that its clinical advisory committee has recommended funding Trikafta with "medium priority" for people over 12.
"That really hurt when it got so close."
Charlie says living with cystic fibrosis "sucks" because it means constant hospital visits, spending hours taking medication and missing out on several school camps due to being sick.
Charlie uses a nebuliser daily - it contains a saline solution that loosens phlegm in his lungs so he can cough it up.
He just wants to be "normal" and says Trikafta would allow him to "breathe better".
"It will save more lives and most of us can just be normal."
Rotorua MP Todd McClay says it is "heartbreaking" to hear there are people in the community whose lives can be prolonged or saved with Trikafta.
The Government needs to "get on top of its spending" by making sure the "important things" are done, including buying drugs such as Trikafta.
Pharmac operations director Lisa Williams says Pharmac would like to fund Trikafta but it operates on a "fixed budget" that the Government sets.
"We can't avoid the fact that Trikafta is a very expensive medicine.
"There will always be more medicines we want to fund than we can afford. This means we have to make difficult choices about what medicines are funded."
The price, along with costs or savings to other parts of the health sector, is a considered factor during the assessment of funding applications, she says.
Pharmac acknowledges there is an "unmet health need" for another effective treatment for patients with cystic fibrosis and it has placed Trikafta on its options for investment list.
"While Pharmac can negotiate some of the best prices for medicines in the world, when faced with a medicine which has such a long patent life and no competition, such negotiations can be challenging."
Williams says Vertex has a monopoly on the drug because it is the only one that can make and sell the medicine.
"This means there is no competition, thus limiting our influence over the price Vertex decides to offer to sell Trikafta in New Zealand."
Pharmac continues to talk to Vertex about the cost, but cannot provide a timeframe for if or when a decision will be made to fund Trikafta.
Health Minister Andrew Little says the Government "broke the three-year funding freeze imposed on Pharmac by the previous Government" which took the budget to $1.1 billion this year.
"How that money is spent is a decision that is and should be made by medical experts, not politicians, and that's why it's important that Pharmac's decision-making remains independent of the Government."
He questions pharmaceutical companies about the price of medicines at "such an expensive level".
Vertex pharmaceuticals Australia and New Zealand senior country manager Sabrina Barbic says the company has a "well-established managed access programme" available in several countries, including New Zealand.
It provides compassionate access for "the most critically ill patients" who meet "certain objective criteria".
When asked what makes the drug so expensive, she says the price reflects the clinical value and benefits to patients, caregivers and health systems.
Beyond the value to the cystic fibrosis community, revenue allows Vertex to continue investing "significantly" in research and development.
"Each year, we reinvest over a billion dollars into developing new medicines for people with cystic fibrosis and others, who today have few or no treatment options."
More than 30 countries - including Australia, Canada and the UK - have funded the therapy for all eligible patients.
Vertex is committed to ensuring all eligible New Zealanders with cystic fibrosis have "sustainable" access to Trikafta "as soon as possible" and will continue to work with Pharmac.
How does cystic fibrosis start?
Charlie was 3 days old when his parents were told he had cystic fibrosis.
"It was the most horrific news when we found out," Glenn says.
He and wife Kim both had a gene that mutated when they had Charlie. Their first son Henry, 13, does not have cystic fibrosis.
Cystic Fibrosis New Zealand chief executive Lisa Burns says it is a "cruel twist of fate" which brings cystic fibrosis to life.
Two people "by ill-luck" carry a "faulty gene" that leads to a child being born with cystic fibrosis.
Burns says it takes two people carrying the gene, along with "a copy of the CFTR gene mutation," to have a child with cystic fibrosis.
If this occurred, there is a 25 per cent chance the child will have cystic fibrosis.
It is estimated one in 25 Caucasian people are cystic fibrosis carriers in New Zealand. The incidence is "much lower" in Asian, African and Polynesian populations.
