Beth Vanstone, mother of Cystic Fibrosis Warrior Madi Vanstone and director of the CF Get Loud campaign is desperately hoping to get the word out about the changes coming for Canadians when the new Patented Medicine Prices Review Board (PMPRB) rules come into effect on July 1.
Vanstone has experience in working with both government and drug companies on rare-disease drug approvals. Seven years ago, her daughter Madi made headlines when she approached then-premier Kathleen Wynne and persuaded the Ontario government to approve coverage for her life-saving drug, Kalydeco.
Kalydeco is a drug that "fixes" a genetic deficiency in some CF patients. Madi was among the three to four per cent of sufferers who could benefit from the drug.
But without the coverage by OHIP, her family would have struggled with the $350,000 yearly cost of Kalydeco. Even with private insurance and participation in a drug study, the Vanstones were looking at a cost of about $60,000 per year.
The Vanstones are now advocates for the CF community and continue to fight for more life-saving medications that have yet to be approved in Canada for CF patients and others living with rare diseases.
Just last week, Health Canada did approve the CF drug TRIKAFTA for those aged 12+.
Although good news for CF patients, Vanstone notes there is still "a lot of work to do" when it comes to approving 'orphan' drugs in Canada - drugs intended for rare diseases or conditions, that have a limited prospect of profitability.
The PMPRB was created in 1987 as a way to prevent price gouging by pharmaceutical companies by comparing prices in Canada to other countries. Canada has always compared their prices against wealthy countries, such as the U.S. and Switzerland, but recently switched to lower-cost countries such as Australia, to bring pricing down for the universal pharmacare system.
The PMPRB added stricter rules for pharmaceutical companies to have their drugs accepted into Canada. The changes were set to take effect in January but were pushed back to July 1, 2021.
"This is nothing that Canada should be celebrating," said Vanstone, noting Canada is "far behind" other countries when it comes to access to new medicines.
Patient groups such as the Cystic Fibrosis Community and the Gastrointestinal Society have been raising awareness about the changes throughout the past year and a half, hoping to have their voices heard about the lack of access Canadians will have to orphan drugs.
In response, the PMPRB started a $56,000 publicly-funded campaign in February 2021, which some patient groups claim is intended to discredit their concerns and accused them of spreading disinformation on PMPRB reforms.
Member of Parliament for Calgary Shepard, Tom Kmiec, who has been advocating for patient groups after losing one of his own children to a rare disease, obtained the PMPRB Communications Plan on May 21 through an Access to Information and Privacy Request.
Kmiec also shared internal emails on social media between executives at the PMPRB. In an email sent by director Tanya Potashnik, she wrote the (pharmaceutical) "Industry has been sucking Canada for decades".
"This is not an honest and fair dealing to create the best rules to govern excessive pricing of patented medication in Canada," Kmiec wrote in a Facebook post. "The PMPRB is out of control and its July 1st rules must be delayed once again to get to the bottom of what the regulator is doing."
"The most troubling aspect of PMPRB's Communications Plan is that it accuses us of 'disseminating disinformation' and further falsely claims that those of us who are raising valid and critical questions about the reforms are spreading baseless claims that 'aim to strike fear in people'," read part of a letter sent to Prime Minister Justin Trudeau by Gail Attara, President and CEO of Gastrointestinal Society.
“I am so saddened that a regulator in Canada would request and budget public money to conduct a lobbying and advocacy campaign rather than engaging in constructive dialogue,” said Durhane Wong-Rieger, President and CEO of CORD (Canadian Organization for Rare Disorders) in a recent press release from The (CORD).
According to Wong-Rieger, “Nobody wants sustainable affordable drugs more than patients but the majority of experts have raised serious concerns that the PMPRB regulatory practices will lead to unsustainably low and unpredictable prices in Canada. This is not a threat, it’s just the reality of a market that’s less than two percent of the global share.”
When contacted for comment, the PMPRB said they have long advocated for reform to its regulatory framework and have a responsibility to raise public awareness of its mandate and the rationale for the recent changes to its Regulations and Guidelines.
"The purpose of the PMPRB’s communications strategy is to disseminate objective and impartial information in order to address misinformation about the PMPRB’s mandate and the impact of the reforms so that the public can come to a more balanced and informed understanding of the issues," read a response from their communications team.
The PMPRB did not comment about the internal emails being circulated online between the PMPRB executives.
Vanstone and other patient groups are encouraging all Canadians to write to their MP, requesting that the Office of the Auditor General of Canada, Treasury Board, and Public Sector Integrity Commissioner investigate alleged "misconduct" on the part of the PMPRB board.
"This attempt by a federal regulator to silence patient voices, some of our most vulnerable citizens, with a publicly-funded disinformation campaign is not only misconduct, it erodes democratic discourse and civic values," read a post on the Canadian Cystic FibrosisTreatment Society Facebook page.
Vanstone acknowledges the price of orphan drugs is extravagant, but said that it's up to the government to negotiate the prices down.
"For anyone to say we are 'For Pharma' - well quite frankly, I'm for Pharma because they saved my daughter's life. To say we agree with the prices, no, it's the government to negotiate those prices, not put a blockade up," said Vanstone.
