The Fight for a Life-Changing Drug: Getting Trikafta Approved in Alberta

EDMONTON – Zack Bushell, 20, was diagnosed with a fatal disease just hours after birth.

“This is where our journey with cystic fibrosis began,” said Chris Bushell, Zack’s father.

For Zack, being diagnosed with cystic fibrosis meant two decades of respiratory infections, hospital visits and, at one point, a lung capacity of 40%. That was until the family found out about Trikafta.

“Zach was born in the United States and this new wonder drug has been available in the United States for almost two years,” Chris told CTV News Edmonton. “We were able to get it for him, and within three weeks (of use) his lung function improved by 70%.”

Cystic fibrosis is a genetic disease that affects the development of proteins in human cells. It can affect the respiratory, digestive and reproductive systems. Ultimately, this can impact life expectancy and overall quality of life.

Trikafta is being described as one of the greatest innovations in the field of cystic fibrosis that could improve the lives of more than 4,000 Canadians.

The only problem is its price of around $ 390,000 per year. Although Health Canada approved the wonder drug, Alberta did not agree to fund it.

In the Bushell family, Zach isn’t the only one with cystic fibrosis. Her 16-year-old brother, Brett, was also born with the disease.

“But he was born in Canada and doesn’t have access to the new drug yet,” Chris said.

“It’s frustrating,” added Brett. “You can drive 12 hours and get Trikafta.”

Last week, the Canadian Agency for Drugs and Technologies in Health released a draft recommendation saying that Trikafta should be approved, but only for patients with reduced lung capacity.

“I think it’s a little silly,” Brett said. “It’s like saying, ‘You don’t have a morbid disease, so we’re not going to put you in the hospital. “”

Dr Martha McKinney, a pediatric cystic fibrosis physician at Stollery Children’s Hospital in Edmonton, said the longer you wait to use a drug like Trikafta, the more lung damage can occur – damage that could be prevented with Trikafta.

“The longer we wait, the more lung damage occurs and has an effect on growth and the other organ systems involved,” McKinney said.

According to McKinney, the median age of death for people with cystic fibrosis in Canada is in their mid-30s.

The doctor described Trikafta as a combination of three drugs in one pill that helps bind to proteins in poorly functioning human cells to improve their functionality.

“It doesn’t work perfectly. But it works better enough in most people, “McKinney described,” in order to really decrease the symptoms because it just increases the function of this protein enough.

“It’s really revolutionary for a lot of people with cystic fibrosis,” she added.

In other countries, McKinney says that many people who have used the drug have seen fewer hospitalizations, improved organ functionality and an overall better quality of life. She added that some who were on organ transplant lists have been able to opt out, simply because the drug is able to help.

“They are able to do more things and get back a part of their life that they lost to illness,” McKinney said. “Until we find a cure (for cystic fibrosis), it really has the potential to increase quality of life, increase lifespan, and reduce disease in people with cystic fibrosis.”

Without the drug, Chris said his son would not be able to fully achieve his dreams.

“The only endpoint is constant hospitalization, multiple organ transplants and death,” Chris said.

Alberta Health spokesperson Karin Campbell said in a statement to CTV News Edmonton that they are awaiting a final decision on Trikafta.

“It is important for Albertans with cystic fibrosis to have access to this drug and we look forward to providing this drug to all eligible patients,” said Alberta Health.

Failure to find funding for the life-changing drug in Alberta would mean a move for the Bushell family.

“It’s either staying here and watching my son get sicker and sicker, or moving to one of the 35 countries where this drug is available.”

With files from CTV News Edmonton’s Chelan Skulski