Unfunded drug the last hope for 18-year-old with cystic fibrosis, autism, and two years to live – Stuff.co.nz

Rebekah Jessen is a full-time carer for her 18-year-old son, who has the double-whammy diagnosis of cysitic fibrosis (CF) and autism.

As Matthew Lamberts autism prevents him from performing his own care, Jessen has had to give up work to look after him.

And with his CF rapidly deteriorating, Matthews quality of life is very poor. His life expectancy is likely only another couple of years.

Rebekah Jessen

Matthew Lambert during a recent hospital stay wearing a vest that shakes his chest and helps to break up mucus.

The familys only hope is the drug trikafta, which is the first treatment to treat the underlying causes of CF, rather than the symptoms.

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But with trikafta not funded by Pharmac, and at a cost of about $430,000 a year, the outlook is grim.

For anyone, CF is a nasty illness. A genetic condition that causes the body to produce abnormally thick, sticky mucus, CF causes lung and chest infections, digestive problems, and issues with other internal organs.

Rebekah Jessen

Rebekah Jessen has given up her work as a midwife to care for Matthew fulltime.

Theres no cure, but symptom management requires daily physiotherapy to help break down the mucus on top of a drug regimen. As CF is degenerative, the regime becomes longer and more involved as the patient ages.

It takes several hours of physio and treatments just to keep [Matthew] well on the daily, Jessen explained. That includes physio three times a day, plus time on a nebuliser inhaling antibiotics and another mucus-thinning drug.

People without autism can start to become a bit more independent with their own treatment but Matthews never been able to gain any independence with his treatment, so anything that has to be done has to be done by me, Jessen said.

Matthews condition has worsened over the last year. Hes been hospitalised four or five times, for weeks at a time.

His quality of life is poor, said Jessen. Matthew used to be a vibrant, happy boy who went to school, danced with the charity StarJam, and participated in his community. Now hes almost completely housebound.

He knows that hes sick, and he will comment a lot: Im having trouble breathing, Im coughing a lot, I cant breathe. Hes in a lot of discomfort.

Rebekah Jessen

Matthew spends several hours undergoing physical therapy and on a nebuliser every day, but his condition is worsening.

For most CF sufferers in New Zealand the only hope of significantly prolonging their life is a lung transplant, but Matthew isnt a candidate because he has liver damage that would make the surgery too risky.

Trikafta is his last chance, and its a good one: Internationally, it has shown to work for around 90 per cent of CF patients.

Thereve been people that have gone from 20 per cent lung function to 60 per cent lung function, Jessen said. Gaining weight, able to be off oxygen, having better quality of life and less hospitalisations because of this drug.

REbekah Jessen

Matthew has been hospitalised a number of times in the last year. Trikafta campaigners claim the drug could radically improve his condition.

Trikafta has been the subject of a large campaign on behalf of a number of patient advocacy groups. It was one of the drugs named in the petition Patient Voice Aotearoa took to Parliament the week before the budget, when Jessens hopes were buoyed by the number of MPs who came out to acknowledge the demonstrators.

We all thought, Theyre hearing us, theyre seeing us. And then [when] that menial amount that was given to Pharmac, we thought: Nobodys hearing us at all.

The $200 million over four years given to Pharmac in the 2021 budget has been widely criticised, with Patient Voice Aotearoa spokesperson Malcolm Mulholland calling it nowhere near enough.

Rebekah Jessen

Rebekah Jessen says she will keep fighting for trikefta to be funded, as it is her only option left.

In order to consider funding a drug, Pharmac needs to receive an application from a supplier, in trifaktas case a company called Vertex. It has not received an application to date, but critics say with Pharmacs funding at the current level, it would be meaningless.

Patient Voice Aotearoa said New Zealands spending on medicine was about $2 billion a year behind other OECD countries. Trikafta is funded in countries including the UK, the US, Denmark, Germany, Switzerland and the Republic of Ireland.

Jessen said she had always been a Labour supporter, but she was frustrated at the current Governments lack of action on drug funding.

People with CF in New Zealand had some of the worst outcomes of any internationally, she said.

Young people will die. If they dont get access to these drugs, they will die. Give Pharmac the money they need.

One of those young people is Matthew, whose treatments are reaching the maximum point of what they are able to do for him.

Matthew was unlucky to be born in this country, Jessen said.

As a parent this is my only option, and my sons life is worth fighting for, and I will keep fighting for as long as required. What choice do I have? I shouldnt have to fight, but this is what it comes down to.

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Unfunded drug the last hope for 18-year-old with cystic fibrosis, autism, and two years to live - Stuff.co.nz