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Upcoming changes to PMPRB guidelines have rare disease community all fired up

'It's a balance between affordability and accessibility,' says policy researcher
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Rare disease advocate Beth Vanstone with daughter and Cystic Fibrosis Warrior, Madi. Natasha Philpott/BradfordToday

Patients with rare diseases across Canada are anxious about the upcoming changes to the PMPRB (Patented Medicine Prices Review Board), expected to take effect on Jan. 1, 2022. 

According to CORD (Canadian Organization for Rare Disease), in Canada, 1 in 12 people, (approximately 3 million) is affected by a rare disease. 

The PMPRB was created in 1987 as a way to prevent price gouging by pharmaceutical companies, by comparing prices in Canada to those in other countries. The PMPRB guidelines have added stricter rules for pharmaceutical companies to follow, to have their drugs accepted into Canada. The changes were initially set to take effect in January 2021, but were pushed back to July 1, 2021 - and then once again to January 2022. 

The guidelines received a lot of backlash from patient groups, who have been attempting to raise awareness about the changes over the past two years, hoping to have their voices heard. They are concerned the new rules will mean a lack of access for Canadians to "orphan" drugs.

Orphan drugs are those drugs developed, often after lengthy and costly research, to treat rare diseases that impact only a tiny percentage of the population. As a result, they tend to have extremely high price tags - beyond reach, without government assistance. 

"The PMPRB has consulted extensively with stakeholders on multiple drafts of its new Guidelines. The version of the Guidelines that is slated to come into force with the regulatory amendments incorporates many significant changes that were made in response to concerns voiced by the pharmaceutical industry and patient groups," said Jeff Wright, Manager, Communications for PMPRB.

The new federal government regulations will reduce the prices of medication drastically, in some cases by 60 per cent or more, which Nigel Rawlson, a pharmacoepidemiologists and pharmaceutical policy researcher in Saskatoon, says is unsustainable. 

"You can make drugs affordable, but if they don't come, is that beneficial to patients? It's a balance between affordability and accessibility," said Rawlson. 

Some patients are able to cover the costs of the medications with the help of private insurance, provincial health plans, or pharmaceutical patient programs, and some get access to drugs that are still in development or not yet approved by Health Canada through special government programs or pharmaceutical trials. Even then, there are no guarantees they will have access or that the drugs will work. 

CORD is working with the federal government on a $1Billion, two-year rare disease strategy that will allow for easy access to lifesaving drugs for the rare disease community, and set up infrastructure for rare-disease research and diagnosis. It is planned to take effect sometime in April 2022. 

The strategy will be an incremental process, with a few programs in place to start. 

"My goal is to say let's look at the drugs that are coming in, decide what the best program will be to make them available, " said CORD's Chief Executive Officer and President Durhane Wong-Rieger. 

She indicated it is not just the medications that need Canada's attention, but the research and development side, that can help detect, treat and prevent rare diseases and conditions.

"I think that's the challenge: where do we rank in terms of being able to attract investments in research, investments in clinical trials and early access to therapy?" said Wong-Rieger. 

Down the road, these innovations will end up costing the health care system less, she said.

"If you can cure a disease, or prevent a disease or keep a disease from getting to the point where people are disabled, isn't that the best use of health care dollars?" she said. "We don't want this new drug program built-in yet, because we have to be able to build it so we can anticipate the future drugs."

Wong-Rieger noted the government's recent backtracking on changes that would have cut back the prices of existing medications in Canada, which have now been "grandfathered" under the new guidelines.

"Because they (the Guidelines) were unreasonable, they were not grounded in science, they were seen as nothing more than punitive in trying to drive down drug prices without any basis and good reason for how it would be done," she said. 

The guidelines set to take effect on January 1, 2022, will apply to new drugs coming down the pipeline only. 

Rare disease patient advocate and mother of local Cystic Fibrosis (CF) warrior Madi Vanstone, is helping to work on the rare disease strategy with CORD. 

Eight years ago, the Vanstones made their first trip to Queen's Park to fight for OHIP health insurance coverage of lifesaving CF drug Kalydeco, and won. Without the coverage by OHIP, her family could have spent almost $350,000 a year for the drug, although insurance and the fact that she was part of a study reduced the cost to the Vanstones to about $60,000 per year.  

Because Canada has less than two per cent of the global share in the Big Pharma industry, rare disease patients believe most drug manufacturers will simply avoid the hassle of trying to market their drugs here.

"It is really discouraging to see that the Canadian government would rather mandate pricing, knowingly discouraging manufacturers of marketing treatments here, than to work at solutions to pricing issues by collaborating with drug manufacturers and policymakers," Vanstone said. "It's already tough enough navigating a chronic, fatal illness without spending a lifetime fighting for meds."

The PMPRB says the purpose of the reforms to their pricing rules is to prevent prices in Canada from being excessive by bringing them more into line with what most other OECD (Organisation for Economic Co-operation and Development) countries are paying and with their actual therapeutic value to patients.

"The expectation is that by no longer paying among the highest prices in the world, Canada will be able to afford to pay for more drugs for its population and patient access will improve," said Wright. 

While Health Canada and the PMPRB maintain that the new changes won't deter drug manufacturers from bringing new drugs to Canada, Rawlson argues that Canada continues to compare its drug prices with Europe, which is unfair, noting Europe is a much larger market than Canada and with all countries having at least one multinational manufacturer's headquarters or research production facility, and are able to recognize the importance of the biopharmaceutical industry.

"We (Canada) don't value innovation; we are clearly reliant on getting product from elsewhere," said Rawlson. 

Rawlson and Vanstone also point out that the nation's relationship with Big Pharma has been rocky for years, as highlighted during the pandemic, with delays in vaccine distribution. 

"It almost seems like you (the government) are fighting against the rare disease community when you're willing to pay higher prices for vaccines but want to get these rare disease drugs for free," said Vanstone. 

Vanstone is trying to arrange a meeting with the health minister of Canada,  Jean-Yves Duclos to discuss the impact of the new regulations and how they will affect Canadians. 

"These drugs need a separate pathway. They can't come through the same pathway (as common medications)," said Vanstone. "To do this and keep out treatments for people with rare diseases is unheard of. We are Canada, we are not a third-world country - they are mandating these prices to be so low....we're just not going to have access, and we're going to be sitting on the sidelines."

She used the example of CF drug Trikafta, which was only approved this year, two years after other countries like the U.S. 

"That shouldn't happen. Too many patients die waiting (for the drugs)," said Vanstone. 

Vanstone is hoping that the guidelines will be scrapped altogether. "None of it (the guidelines) works. If it worked they would have implemented them when it first came out," she said. 

But Wong-Reiger isn't too concerned about the anticipated implementation date.

"January 1st is just a date," said Wong-Rieger. "Yes, the guidelines will come into effect - but they are only guidelines."

She said although difficult to pull guidelines back once in effect, they are not the law, and they can be changed. 

"Guidelines are written to be able to help implement legislation and to be flexible," she said. "We will continue to fight this. If the government doesn't come to their senses by January 1st, we are not giving up the fight."