'We can't wait': Life-saving cystic fibrosis drugs hard to access, advocates say

No parent wants to outlive their children, but an Edmonton womanfears she might because her kids can't access the drug that would help treat their rare medical condition.

Two of Sharon Stepaniuk's three children have cystic fibrosis (CF), agenetic disease that causes severe damage to the respiratory and digestive systems.

"I really want my children to have the same potential that any other child has," Stepaniuk told CBC News.

There are drugs available that can slow or stop the progress of cystic fibrosis,but getting coverage for them in Canada is rare.

Stepaniuk and 400 other Albertans sent letters to Health Minister Tyler Shandro earlier this month, calling on the province to make cystic fibrosis drugs more accessible, and to support the development of a federal strategy on rare diseases.

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"If we had a rare-disease strategy which allowed these drugs to be approved, and our government went to the table and negotiated prices on these drugs and they were part of our publicly funded system, it would be such a weight off of me," Stepaniuk said.

In the U.S., the Food and Drug Administration recently approved the drug Trikafta, which is said to slow or stop the progression of CF in about 90 per cent of patients.

"This is a drug that people are literally dying to get," Stepaniuk said.

It was approved in Ireland last week, according to CF Ireland, but getting access to Trikafta in Canada likely won't be so easy.

"We're fearing that it might not come to Canada if our governments don't start covering these medicines," said Kim Steele, director of government and community relations for Cystic Fibrosis Canada.

'Blockbuster drug' not submitted for approval

Health Canada has the power to approve new medicines, but provincial governments can decide which medicines get public funding, meaning drug coverage varies across the country.

Getting coverage for CF medicines that have been approved in Canada, namely Orkambi and Kalydeco, is highly uncommon due toeligibility restrictions.

Steele said to her knowledge, only one person has qualified for coverage of Orkambi out of the three provincesthat offer reimbursement Alberta, Saskatchewan and Ontario. Without coverage, she said it would cost about $250,000 for a year's supply of either medicine.

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Vertex Pharmaceuticals, the company that manufactures Trikafta, hasn't submitted the drug for approval in Canada.

"Here is this blockbuster drug that could treat many people and many mutations, and the manufacturer does not see Canada as a good market because the reimbursement environment is so dismal," Steele said.

"There's just not a fair assessment of these drugs and therefore, Canadians can't get them."

The small number of people affected by cystic fibrosis and other rare diseases limits the number of clinical trials that can be conducted. Steele said it's unfair to hold drugs for rare diseases to the same evidential standard for drugs where more robust trials are possible.

"All we're asking for is for our federal and provincial governments to develop a separate process for these drugs so that they can get to the patients in a fair and equitable and timely fashion," she said.

Moreaccessible treatments focus on the symptoms, and include medicine for excessive chest mucus and infections and physiotherapy to keep theairway clear.

'We can't wait'

The federalgovernment hopes to implement a nationalpharmacareprogram. Meanwhile,Budget 2019 proposed investing up to $1 billion into a strategy for high-cost drugs for rare diseases, starting in 2022.

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"We can't wait until 2022 or 2023. People are already losing opportunities to live healthy and well," Steele said.

"It's probably going to take a decade or more to get to national pharmacare, and this is something that is more easily done ... We can set up and start a new process for these drugs while national pharmacare is being built."

Stepaniuk said she hopes to meet with Health Minister Shandro to encourage him to advocate for the 604 Albertans who have CF.

In an emailed statement to CBC News, Shandro said Alberta co-leads a provincial and territorial working group on costlydrugs for rare diseases.

"This group is developing a new national process for reviewing drugs, as a basis for a longer-term project to develop a national rare disease strategy," the statement reads, noting the province hasn't gotten any updates on the federal governments proposal for a new strategy.

He said the province isn't considering broad coverage for Orkambi, but is working with the manufacturer of Kalydeco to expand coverage within a year.

Stepaniuk said the provincial and federal governments need to act fast.

"If [my children] don't get access to these drugs sooner rather than later, their lung function is going to decrease enough that by the time they do finally get access to them, it might be too late."