March 11th, 2020

// Patients in the ‘Fight for Our Lives’ call on Parliament to STOP the PMPRB!

Patients in the 'Fight for Our Lives' call on Parliament to STOP the PMPRB!

As the world comes together to fight coronavirus, Canadians with life-threatening rare and common diseases are marching on Parliament Hill to ask for the same support in the Fight for Our Lives.

OTTAWA, March 10, 2020 /CNW/ - The Canadian Organization for Rare Disorders (CORD) is leading a contingent of Canadian patients with rare and common diseases to Parliament Hill at noon today to deliver one common message to their Members of Parliament: "Stop the PMPRB!".  The Patented Medicine Prices Review Board (PMPRB) is the single biggest threat to Canadian access to lifesaving and life-altering new medicines.

These new medicines are not just incremental improvements but revolutionary therapies that can cure cancer, restore eyesight, grow new bones, prevent heart attacks and kidney failure, replace blood cells, and repair and replace faulty genes. Some are already funded and available in other countries. But availability to Canadian patients is seriously at risk as companies evaluate the impact of the proposed PMPRB pricing restrictions.

The historic roles of the PMPRB are to ensure drug prices are not "excessive" and to promote research investment by the pharmaceutical industry. The review board has abandoned its mandate on research investment and is extending its role on price control. It is proposing a single standard pricing index for all new medicines, regardless of the severity of the condition or the unmet need. This arbitrary pricing index, which is not supported by any scientific evidence, expert economist, or any other country, proposes to drive Canadian drug prices from 40% to 90% below list price. According to many companies, this level of pricing would be far below the costs of research, development and production. One Canadian executive characterized the dilemma this way: "It would be impossible for us to make the case to launch new medicines in Canada at the same price that we offer to low income countries."

The threat of draconian price restrictions has already stopped some new treatments from coming to Canada. In the past year, companies have delayed or withdrawn submissions for more than a half-dozen innovative therapies and an equal number of submissions for extended or pediatric indications have been quietly cancelled. Even worse, companies have also withdrawn clinical trials from Canada, explaining that if drugs will not be launched here, they cannot ethically start patients through clinical trials. In 2019, the number of clinical trials started in Canada declined by 40% compared to 2018. Similarly, Canadian patients may be denied enrollment in clinical trials in the USA or elsewhere if continued access in Canada is unlikely.

"The inability to access clinical trials is literally a death sentence for patients with life-threatening or progressive diseases with no other effective treatments. Clinical trials have also helped to prepare clinicians and facilities to administer these therapies as soon as they are approved, saving many more lives," said Durhane Wong-Rieger, President & CEO of CORD.  "The PMPRB dismisses our concerns as "fear mongering", claiming that companies will not withdraw from Canada. But the exodus already started when the PMPRB issued its draft proposal for new guidelines a year-and-a-half ago."

Example of Cystic fibrosis

The first treatment (Kalydeco) for cystic fibrosis (CF), a genetic progressive lung disease that is usually fatal to children by their teens and early adulthood, was approved in Canada in 2012.  But it is effective for only a very small percentage of CF patients with a specific genetic mutation.

Improved therapies have been developed and two of these have been approved in Canada (with limited public funding for one therapy). However, the company that makes the most important new therapy (Trikafta) that could be effective for up to 80% of CF patients has concluded that it cannot afford to submit the drug for approval in Canada in the immediate future. 

A 23-year-old Halifax woman with CF, Chantelle Lindsay, died on February 19, 2020, after not being able to access Trikafta, which her medical team said was her final hope.1

"The federal drug price controls are a death sentence for many patients," said Chris McLeod, Chair of the Canadian Cystic Fibrosis Treatment Society and a CF patient who credits Kalydeco for his being alive and fully employed today. "The tragic recent passing of Chantelle Lindsay should be a wake-up call for politicians. Canadians are dying because new life-saving medicines are not coming." 

Example of Sickle cell disease

Sickle cell disease (SCD) is an inherited illness in which rigid, crescent-shaped and sticky red blood cells slow or block the flow of blood and oxygen throughout the body, resulting in extreme anemia and debilitating pain crises that can require medication and hospitalization. Recently, the Food and Drug Administration approved the first ever therapies for SCD, Adakveo and Oxbryta, which, respectively, prevent pain crises and red blood cell deformation. Neither treatment has been submitted to Health Canada and there are no immediate plans to file for approval.

"For decades, there have been no effective treatments for sickle cell disease. We now have a revolutionary new drug that can prevent the root cause of the disease, but it won't be made available to Canadians any time soon," said Biba Tinga, President and Executive Director of the Sickle Cell Disease Association of Canada. 

"Canadians cannot live with only cheaper old drugs. We deserve access to the truly amazing life-saving and life-altering treatments," said Durhane Wong-Rieger, President & CEO of CORD. "We support fair drug prices in Canada, but not arbitrary restrictive prices that make it impossible to get new treatments here.  We call on Members of Parliament to "Stop the PMPRB and stop the harm to patients."

About the Canadian Organization for Rare Disorders (CORD)

CORD is Canada's national network for organizations representing all those with rare disorders. CORD provides a strong common voice to advocate for health policy and a healthcare system that works for those with rare disorders. CORD works with governments, researchers, clinicians and industry to promote research, diagnosis, treatment and services for all rare disorders in Canada. For more information on CORD, visit:

1 Petracek H, Woman denied CF drug dies in Halifax hospital, CTV News, Feb. 19, 2020, accessed at



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