'It's huge': Life-changing cystic fibrosis drug a step closer to being funded by Pharmac

Monday, 5 April 2021

Alana Taylor has cystic fibrosis. In 2015, she was near death until she was saved by a double lung transplant. (Video first published in August 2017)

Straight after waking up, Claire Scofield begins each day with a series of treatments – nebulizers, physio and oral medication.

When she gets home from work, she’ll do some exercise, using breathing techniques to cough to “get as much gunk out a possible”.

This is the reality of living with the genetic disorder cystic fibrosis. The condition causes sufferers to produce thick, sticky mucus that clogs the respiratory and digestive systems. More than 530 New Zealanders have the condition. Their life expectancy is late 30s.

Claire Scofield (pictured with her husband Dan) was diagnosed with cystic fibrosis when she was 8 years old.

But with national medicine-buying agency Pharmac now planning to fast-track the drug Trikafta, the lives of many people like Scofield could be prolonged.

* Supplier of expensive cystic fibrosis medication yet to apply for Pharmac approval

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* New drug gives hope to 14-year-old boy with cystic fibrosis

For Claire Scofield, taking Trikafta could improve her lung function, making tasks like exercising easier.

* Breakthrough drug would give child with cystic fibrosis a future

* Relief, excitement as Pharmac funds cystic fibrosis medicine

Six-year-old Mia James has cystic fibrosis. Her mother Kayla Hawkins is hopeful that breakthrough drug Trikafta, for treating the condition, will be funded.

Vertex, the company that manufacturers the breakthrough medication, last week met with Pharmac and New Zealand’s medicine regulator Medsafe and is making applications for registration and potential funding.

Mia James, 6, using her nebuliser. If she can take Trikafta, she may no longer have to use the nebuliser daily.

Pharmac director of operations Lisa Williams said the applications to both agencies would be considered at the same time, speeding up the approval process if it is successful.

Alana Taylor’s life was saved by a double lung transplant in 2015. But she still has cystic fibrosis and could need Trikafta in the future.

Vertex has said Trikafta works for 90 per cent of cystic fibrosis patients.

Pharmac funds another cystic fibrosis drug called kalydeco. But, costing about $383,000 a year, it is a drug that can only benefit about 35 people in New Zealand.

For Scofield, whose health had declined “quite rapidly” over the past few years, Trikafta could be an alternative to having a lung transplant – a last resort for people with cystic fibrosis.

While the 32-year-old from Alexandra still lived a “fairly active life”, including working full time, her lung function was about 35 per cent – making everyday tasks a little harder. She needed oxygen therapy at night and was hooked up to a machine while exercising.

Scofield said she'd done a lot of research about Trikafta and had spoken to friends in Britain and the United States who had taken it and seen noticeable differences in their condition.

“We’re seeing people on transplant lists who no longer need one and are getting 20 per cent increases in their lung function. If I was able to go from 35 per cent lung function to 50 (at a dream), I could go back to managing what I was in the past.

“Transplant talks wouldn't be on the cards any more, which would mean potentially thinking about a future with my husband which may involve kids,” she said.

For 6-year-old Mia James from Manawatū, the medication would not be a cure, but it would turn a life-threatening condition into a manageable one, freeing her from the daily imperative of nebulisers and physiotherapy in order to breathe.

After too many hospital stays to mention, stints on life support and the daily grind of clearing the little girl’s airways, the prospect of access to a life-changing drug is giving her mum Kayla Hawkins hope for her future.

Trikafta could also help Christchurch woman Alana Taylor in the future.

The 34-year-old teacher aide, who has had cystic fibrosis her whole life, had a lung transplant in July 2015 after being given just months to live.

The transplant transformed her life, but she still has cystic fibrosis in other parts of her body.

A lung transplant was the last resort for sufferers and if people were able to take Trikafta instead and prolong their life then it was totally worth it, she said.

“It's huge. Give us the funding, we need it.”