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Mother-daughter duo meet with health minister about lifesaving cystic fibrosis drugs

Local mother-daughter duo Beth and Madi Vanstone met with the Minister of Health last week to share their concerns on behalf of the Cystic Fibrosis community over drug agency's restrictive criteria recommendations for life-saving drug Trikafta
Mother-daughter duo Beth and Madi Vanstone continue to fight for access to lifesaving medication for all Canadians. Submitted

Last week, Cystic Fibrosis (CF) patient Madi Vanstone and her mother Beth Vanstone met with Minister of Health, the Hon. Christine Elliott, to discuss the recent recommendations put forward by CADTH (Canadian Agency for Drugs and Technologies in Health) for the approval of the life-saving drug Trikafta. 

The meeting was arranged by the Vanstones and rare disease patient advocate MP Jim Wilson in hopes of convincing the Minister to consider the recommendations carefully, as they could exclude up to 25 per cent of CF patients in Canada. 

In June, the Cystic Fibrosis (CF) community was elated when Health Canada announced its approval of the life-saving drug Trikafta for those aged 12 and up with CF and at least one F508del mutation.

Their joy was short-lived after reading CADTH's restrictive criteria recommendations for the drug, which includes making funding available only to those with below 90 percent lung function. 

To remain on the drug and have it funded, CADTH is also recommending patients must gain five percent lung function in the first six months of use in order for the prescription to be renewed and to maintain the five percent indefinitely. Beth says this is problematic, as most healthy adults' lung function naturally declines with aging, like hearing and eyesight. 

The recommendations received negative feedback from the CF community, including Dr. John Wallenburg, chief scientific officer, Cystic Fibrosis Canada.

“If adopted by the provinces, CADTH’s draft recommendation will create an absurd situation in which patients will need to become demonstrably sicker to get this medicine, and those who receive the drug and experience important clinical benefits could see it pulled right out from under their feet," he said. 

Beth is hoping CADTH listened to the patients and doctors who had until July 22 to submit feedback on the recommendations. 

She was left hopeful after meeting with the Minister, when Elliott indicated that the province will not be adopting the CADTH recommendations. 

"It was very positive," said Beth about their meeting with Elliott. "She committed to adding the drug (Trikafta) to the formulary (a list of prescription drugs paid for by the government)  in the next few months without the criteria (from CADTH)." 

Nineteen-year-old Madi has been on CF drug Kalydeco for the past nine years, when she and her mother convinced the Ontario government to approve coverage, after a lengthy public battle. But her condition has worsened over the past year, and her doctors are now recommending she switch to the Trikafta medication. 

"I feel myself slipping back, and it’s awful," Madi wrote in a recent social media post.  "Recently my lung function dropped down to 53 per cent - the lowest it’s been since I was five years old. My doctors have recently expressed that it’s crucial that I switch to a new medication called Trikafta before my condition worsens. This is a medication that corrects both defective genes in my body (Kalydeco only corrects one), but isn’t yet available / funded for Canadian CF patients."

"Kalydeco has given her nine amazing years, but there is a better drug now and she needs to be on the better drug now," said Beth. 

Madi has been hospitalized three times in the past 10 months due to CF and is frustrated by the relentlessness of the disease.

She is headed to college in the fall and worries about getting sick while studying. 

"She's been struggling," said Beth. "It's hard."

With the federal election coming up later this month, Beth is wondering why none of the candidates have addressed Canadians' access to orphan drugs. In a recent op-ed article co-written with Nigel Rawson,  an independent researcher and an affiliate scholar with the Canadian Health Policy Institute, they raise concerns over the government's relationship with the biopharmaceutical industry. 

"If elected, the Liberals will no doubt continue with their intention to severely cut the prices of new medicines. The NDP will likely support them. The Conservatives propose a more collaborative relationship between the federal government and the biopharmaceutical industry. However, no party has presented a comprehensive strategy to get pioneering but costly medicines to Canadians who need them," read part of the op-ed.

When asked if the drug agency will be making changes to its draft recommendations based on the feedback received, CADTH confirmed the reimbursement recommendation for Trikafta  were reconsidered at its Expert Committee's Aug. 18 meeting.

"We received feedback from patient and clinician groups and requests for reconsideration from Vertex Pharmaceuticals (Canada) Inc. and the federal, provincial, and territorial public drug programs," said Janis Bogart, a communications representative from CADTH.  "The final reimbursement recommendation will be posted on and publicly available by Sept. 16, 2021."

According to CF Canada, it is estimated that one in every 3,600 children born in Canada has CF. More than 4,300 Canadian children, adolescents, and adults with cystic fibrosis attend specialized CF clinics.

Natasha Philpott

About the Author: Natasha Philpott

Natasha is the Editor for BradfordToday and InnisfilToday. She graduated from the Media Studies program at The University of Guelph-Humber. She lives in Bradford with her husband, two boys and two cats.
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