Leah Rumney’s Christmas wish was to be free of cystic fibrosis.
But it’s a wish which broke her mother’s heart because she knew she couldn’t make it happen for her 11-year-old.
Not without Trikafta – a breakthrough treatment Cystic Fibrosis NZ says is widely heralded as having the potential to turn cystic fibrosis from a life-threatening condition to a manageable condition.
But it is not currently registered and funded in New Zealand.
And at more than $470,000 a year the drug is simply not attainable to many with cystic fibrosis, including the Rotorua girl.
So she has to keep on wishing.
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.
Diagnosed with a heel prick test as an infant Leah goes to hospital twice a year for a course of IV antibiotics usually for three weeks at a time, her mum Janelle Rumney tells the Rotorua Daily Post.
“The last time she was there she was on school camp and she just got really unwell. She could hardly walk to the toilet without coughing, she was hardly breathing and couldn’t talk because she was coughing so much.
“Sometimes we know its gonna come because she has been getting unwell and other times its really sudden.”
Chest physio three times a day has been a part of Leah’s daily routine since she was 4 months old.
“It’s horrible and trying to explain to her that we are doing these things to help her, but she is still sick.
“It’s really hard knowing there is this medication out there that would dramatically improve her life, but we can’t get it.”
It’s not that Rumney “can’t” get it as such. She could foot the annual cost of almost half a million dollars for her daughter – but that is simply unachievable.
It’s left Rumney holding on to hope that one day soon, she will be able to supply her daughter with a means to change her life.
“Hopefully she wouldn’t be in hospital as regularly, she wouldn’t be off school all the time unwell, tired or sick.
“She is 11 and she still falls asleep on the drive home from school because her body is so exhausted.”
Trikafta works to clear all the mucus that is filling Leah’s lungs and pancreas. Without that stuff in there, Rumney hopes her daughter would be able to absorb food and vitamins better.
It would mean fewer chest infections as well. Essentially, “her whole quality of life would improve”.
“Leah knows the possible outcome, that only 50 per cent make it to 31. She is aware of that so we try to make the good times really good times and we just get through the hard times because that’s what you’ve got to do.”
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, they say 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 around 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.”
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