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Kiwi teen with cystic fibrosis sees ‘miracle’ results with new drug Trikafta, but it costs $470k a year

A Kiwi teen has seen miraculous results with a new drug designed to help people living with cystic fibrosis.

Bella Powell, 17, has spent much of her life in hospital, and when she was just 15 she was told she only had two years left to live.

Cystic fibrosis affects the cells that produce mucus. They then plug up the tubes and passageways which puts massive amounts of pressure on the lungs, eventually wearing them out.

Powell started taking Trikafta, a drug that’s in limited use in New Zealand, and says her breathing had cleared in six hours after taking her first pill.

She takes three pills a day, two in the morning and one at night. Two weeks ago before she started taking them, she could barely walk. But now she says she feels great.

“[It’s] more than a miracle. If there’s a word that’s bigger than a miracle, it’s that.”

But Trikafta costs $469,000 a year. It’s owned by American company Vertex, which sets the price and it isn’t subsidised by Government drug-buyer Pharmac.

She also only has a three-month supply.

“You get something like this and everything is just better. And then every week you finish a box and you’re like, only a few more weeks of this.”

Since cystic fibrosis is destroying Powell’s lungs, she needs a lung transplant. But that might not work and it’s risky – she could die on the waiting list or on the operating table.

Living with her condition is like “having a pound of weights on your chest”, she says.

“Your ribs can’t move, you can’t get enough air in. Your body is always sore.

“You can’t explain it unless you have it. It would be like you breathing through one of those one teeny tiny straws all day.”

She adds she can’t do what every other 17-year-old does.

“That’s really hard. When you just want to walk with your friends to class. The really small things. And you can’t do that.”

Medical researcher and cystic fibrosis expert Sir Bob Elliott has tried to help Powell for much of her life.

“I felt I just couldn’t let Bella die knowing there was a drug out there that could help her.”

Knowing that Trikafta might be able to save her, he paid for her three-month supply which cost about $100,000.

“I’m not in life to make money. I’m here to help people with the disease.”

Elliott made a medical breakthrough in the 1970s with the ‘heel prick test’ that identifies cystic fibrosis at birth. He spent many years trying to find a cure, but couldn’t.

He says Trikafta is a “miracle drug”.

“I’ve been treating cystic fibrosis most of my professional life. This is what we’ve been waiting for and it works.”

He says supplier Vertex charges too much for the drug. A price that is, in his opinion, morally wrong. 

“I think it’s cruel actually, and unethical.”

While he believes the price needs to be dropped, he’s also calling on Pharmac to get involved and do a deal immediately.

“Get on with it, start negotiating with Vertex for supplies of this drug at a reduced cost.”

Elliott was knighted at his home a week ago because he’s dying of cancer and was too sick to travel.

“I’m not well. I’m 86. And I have a terminal illness. I won’t be around all that much longer.”

After dedicating his career to helping New Zealanders survive cystic fibrosis, he’s suddenly so close to seeing a cure available here.

“It would be nice. It would complete a life’s work, actually. To see this through to being no longer a fatal disease… That would please me no end.”

There are 503 people with cystic fibrosis in New Zealand and nearly all of them want Trikafta. But it would cost $236 million a year, which is why a deal needs to be done with Vertex to push that down.

Vertex said its goal is to continue working with stakeholders in New Zealand, and Pharmac said it hasn’t had an application from Vertex to fund Trikafta.

Health Minister Chris Hipkins says it isn’t for politicians to “second guess” those clinical decisions.

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