Residents fight for access to promising cystic fibrosis drug

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This article was published 05/09/2020 (1795 days ago), so information in it may no longer be current.

These days, some assume Tyler Funk has COVID when he goes to the store and coughs.

It’s not COVID, he said. He has cystic fibrosis.

“You never want to be in line at Dairy Queen waiting for an ice cream cone and you start coughing up blood,” Funk said.

Funk and his sister Janelle Dawson both have the disease. Cystic fibrosis mainly affects the lungs, but also the digestive system, Dawson said. Dawson is an advocate with Cystic Fibrosis Canada.

Humans produce quantities of thick sticky mucus, and people with cystic fibrosis can’t effectively clear it out of their lungs. Lung infections result and bacteria can accumulate.

COVID for people with cystic fibrosis would be horrible, Funk said, adding chances of death would be high since the virus attacks peoples’ respiratory systems. Lung function could decrease even more than it already would for people with cystic fibrosis. Dawson said they would likely need to be on oxygen 24/7, and might be left in a wheelchair. The disease could also warrant a listing for a lung transplant.

“Lung function declines until we no longer can breathe,” Dawson said describing cystic fibrosis, adding her lung function ranges from between 35 to 40 percent.

People with cystic fibrosis take a number of pills and antibiotics. Dawson administers an IV herself.

“It’s like a part-time job to stay alive and healthy,” Dawson said.

Cystic fibrosis has other side effects, such as CF-related diabetes.

Lung function lower than 30 percent usually results in a lung transplant, Dawson said. But being too sick disqualifies someone from getting a lung transplant, and complications can result as well. A lung transplant however, Dawson said, only buys time.

According to Kim Steele of Cystic Fibrosis Canada and data from the organization, the median age of survival for people with cystic fibrosis is 53.

“But of the people who died in 2018 of cystic fibrosis, half were under the age of 33,” Steele said.

There’s hope in the form of the drug Trikafta. Funk considers it “the ultimate drug.” Drugs that people with cystic fibrosis commonly take usually treat symptoms, Dawson said.

Trikafta is a modulator and changes the way cells function. The drug corrects cells, Dawson said, such that the lung function of people with cystic fibrosis dramatically improves.

“I personally know people who’ve come off of lung transplant lists after being on this drug,” Dawson said.

Some get rid of all the medications they take, Steele said.

However, getting the drug to people who need it in Canada has been a challenge, Dawson said, and is subject to complex pharmaceutical politics.

Trikafta, manufactured by American company Vertex Pharmaceuticals, is already available in the U.S. and is being fast-tracked for access in the UK and in Europe. A year’s supply of Trikafta costs about $300,000.

Trikafta isn’t available in Canada, and Vertex hasn’t even applied to have Trikafta approved.

The Patented Medicine Prices Review Board (PMPRB) is a Health Canada body that regulates prices of patented medicines sold in Canada.

Drug prices in Canada are high, and according to a recent decision, the PMPRB required drug prices be cut by 45 to 75 percent. The board has made efforts to reduce drug costs to Canadians to the tune of billions of dollars in the long term, according to a CBC story.

Both Canada and individual provinces have negotiating power in determining drug prices. But one researcher has argued Canada is reducing prices too quickly, creating the danger that pharmaceutical companies won’t apply to have their drugs in the country at all. This could mean those who need drugs in Canada won’t get them.

“Severe price reductions will drastically reduce Canada’s attractiveness for drug developers as a country in which to do business,” wrote Nigel Rawson and John Adams in the Financial Post.

Dawson said another regulation acts as a barrier for a company like Vertex to apply for Health Canada approval.

Right now, if a drug company submits to Canada, Dawson said, the drug price the government pays is confidential. The PMPRB wants to remove confidentiality.

There is further regulatory uncertainty regarding economic factors, thus companies such as Vertex aren’t exactly sure what they’re applying for, Steele said.

Steele said Cystic Fibrosis Canada wants Vertex and the government to step up. One measure Cystic Fibrosis Canada supports, Steele said, is Canada changing comparable drug prices to exclude the U.S. and Switzerland. The U.S. has significantly higher drug prices than do other comparable countries.

Doing so, Steele said could reduce drug prices by about 20 percent in Canada.

Some say the government shouldn’t reduce drug prices dramatically since expenses could be reduced elsewhere for Canadian taxpayers if Health Canada approves Trikafta, in the form of reduced hospital visits and increased work and income tax contributions.

Steele said Trikafta significantly improves the lives of those who need it, and urges the government to approve it.

According to one study, the estimated median age of survival for a child born with cystic fibrosis would increase by 9.2 years.

Steele is also interested to know of future political decisions related to national pharmacare. An original intent of the drug price reductions was to use the saved money to put toward a national pharmacare program. Steele is awaiting the minority Liberal government’s September throne speech.

An added problem for people with cystic fibrosis in Canada is even if Vertex applies to Health Canada and Trikafta is in the country, it could be another two to five years before provinces have any coverage. Manitoba, Dawson said, doesn’t have a cystic fibrosis drug plan.

According to CBC, Vertex has a compassionate coverage program, although not everyone will be approved. The application of 23-year-old Chantelle Lindsay was denied, and Lindsay died in February.

“I don’t have five years to wait, so we need this to be fast-tracked through the system,” Dawson said.