For Sharon Stepaniuk, cystic fibrosis is not a rare disease. Two of her three children have it.
“They had a later diagnosis. My daughter wasn't part of the newborn screen. We didn't know that she had cystic fibrosis. We discovered that they both had CF once we had all three of our children,” she began, remembering the many challenges early on.
“She was just sick quite often and my youngest was failing to thrive. They got tested for CF and that’s when we found out.”
Even though Cystic Fibrosis Canada estimates one in every 3,600 children born in Canada has the disease, CF is still considered rare. Living with it usually means fighting to breathe and digest food with the help of a lot of medications and other treatments.
Kim LaFramboise’s son Carter was diagnosed with CF when he was one. Now 14, he’s managing his disease with a variety of prescriptions and other therapies, but they are holding out hope for something newer and better to come along.
“He's doing well but we're anxiously waiting for drugs that can give us the assurance that he's going to continue doing well,” she said.
A new drug called Trikafta was approved as a breakthrough therapy by the U.S. Food and Drug Administration last October and has the potential to help 90 per cent of people with CF, as it works on the most common genetic mutation causing the disease.
“We've been hearing really good things about it. We hear that lung function is going up quite significantly and that the people that are taking it gain weight and are just overall generally healthier,” LaFramboise continued.
The waiting is not easy and there’s no end in sight. Trikafta’s manufacturer, Vertex Pharmaceuticals, hasn’t yet applied to Health Canada for its approval for use in this country.
At the heart of this inaction is the price tag. According to Cystic Fibrosis Canada’s website, the list price of Trikafta is USD $311,000 per year. The process of determining the Canadian price starts with Health Canada’s approval before a drug review and reimbursement processes kick in.
A quasi-judicial body called the Patented Medicine Prices Review Board (PMPRB) operating at arm’s length from the Minister of Health then determines the maximum price the company can charge, and insurance companies negotiate directly with the company on what they will cover.
“What the mandate of the PMPRB is is to protect Canadian consumers from excessive prices with respect to patented medicines sold in Canada,” explained St. Albert-Edmonton MP Michael Cooper. “What they can do is pursuant to their mandate, review prices that patentees charge and if the price is found to be excessive, the board can order price reductions.”
Last week, Cooper and Stepaniuk along with other CF advocates called on Health Minister Patty Hajdu to delay the implementation of changes to the PMPRB regulations.
The changes, they say, will significantly reduce access to new, lifesaving therapies, especially in the case of rare disorders like CF. Efforts to impose price reductions of new drug therapies, Cooper continued, would also change how the pharmaceutical industry perceives Canada as a viable place for new drugs to come to market.
Drug pricing
Currently, Canada bases drug prices with respect to other comparator countries. The new changes remove two countries from that list, including the U.S., and add six others.
“What the effect of that would mean is a reduction in terms of the median prices of drugs in Canada by about 20 per cent,” Cooper explained, continuing that this would also change the value of how each new drug will be assessed.
This means the implementation of a cost-effectiveness analysis by the Canadian Agency for Drugs and Technologies in Health, whose mandate is to make reimbursement recommendations for public drug plans across Canada. It is the body that makes non-binding recommendations to provincial governments’ drug plans.
Sometimes those recommendations are dramatic.
Cooper explained in a statement the imposition of price reductions, which could be as high as 90 per cent, would make Canada an outlier among countries in the Organization for Economic Co-operation and Development, and a significantly less attractive market to launch new therapies, and Canadians with rare diseases including CF would be the most negatively affected.
“These changes have contributed to delaying and potentially making the game-changing drug Trikafta, which can treat up to 90 percent of Cystic Fibrosis patients, unavailable in Canada. The Minister has been repeatedly warned by the rare disease community about the devastating impact these changes will have on access to life-saving therapies. It is time that the Minister listened and put a halt to these ill-considered changes,” Cooper wrote.
The regulatory changes effective January 1, 2021 are purportedly aimed at reducing drug costs.
