Proposed drug policy changes put sick Canadians at risk, patient advocate warns

February 22, 2018

Category: Critical Illness Insurance, Long Term Care

Heidi Westfield – Postmedia Network

The Canadian Organization for Rare Disorders represents an estimated one in 12 Canadians living with rare diseases.


One of Canada’s leading health policy advisors warns that vulnerable patients will be put at risk if proposed changes to drug pricing go into effect in this country. Dr. Durhane Wong-Rieger, head of the Canadian Organization for Rare Disorders (CORD), says patients should be “very concerned” about a patented drug pricing policy review now underway. The new rules, she says, will make it harder for patients to access new and potentially life-saving therapies.

“It will cost people their lives, we know that,” Dr. Wong-Rieger says. “There are already patients where drug access is delayed, who actually do die before the drugs actually make it to Canada. The new proposals will only make the situation worse. It will happen in greater numbers, at a faster rate.”

The Canadian Organization for Rare Disorders represents an estimated one in 12 Canadians living with rare diseases. These patients are diagnosed with a range of conditions, such as cystic fibrosis, hemophilia, muscular dystrophy and Gaucher’s disease. For many, their medical condition is so unusual it can take years to pinpoint a diagnosis. The vast majority live with diseases where no known treatment is yet available.

Dr. Wong-Rieger notes that Canada already lags behind countries such as France, Sweden, Germany, and Scotland when it comes to accessing medications for rare diseases. Proposed changes to the Patented Medicine Prices Review Board (PMPRB), she says, will make it even tougher to make innovative treatments available.

Last May, Canada’s Health Minister at the time announced a review of the PMPRB, designed to ease pressure on public healthcare budgets by changing the pricing model for patented medicines. Consultations are continuing this year, with the new regulations poised to take effect January 2019.

The PMPRB is an independent federal watchdog created in 1987 that regulates and monitors the prices of brand name medicines. The Board regulates the maximum price that drug makers can charge wholesalers, hospitals, and pharmacies for patented medicines. It does not have jurisdiction over mark-ups by pharmacies and other retailers or wholesalers, nor does it regulate the price that drug plans or insurance companies agree to reimburse.

Dr. Wong-Rieger sees the value of challenging drug makers to find cost reductions and reduce waste. She stresses, though, that it is vital to look beyond a price point equation when it comes to the health and quality of life of Canadians. There are other factors to consider, and patients should not be left waiting for treatment during what can be years of price negotiations.

When it comes to rare disease research, she notes the risks of changing drug policy are substantial. Finding a new treatment for these disorders is a complex undertaking. It often involves a sizable investment in research, and years of clinical trials. To offset those costs, the price tag for new medicines, especially for rare diseases, is often significantly higher than those of established therapies for more common ailments.

If the federal government drops the “ceiling price” for new medicines too low, Dr. Wong-Rieger warns it will lead to a chain reaction of negative consequences for patients living with rare conditions. A lower price point will act as a disincentive for drug makers to invest in research and clinical trials in Canada. Instead, she cautions, industry leaders will turn to other countries offering more favourable prices to recover the cost of developing a new medicine.

The proposed new PMPRB regulations, she predicts, will also mean fewer options for Canadians hoping to join clinical trials, and less access to innovative medicines for the patients who are pinning their hopes on the next medical breakthrough.

“Why are we beating up on these patients? No other major developed country is taking this type of approach to rare disease drugs,” Wong-Rieger says. “We are going to put ourselves at a further disadvantage in terms of getting companies who want to come to Canada early.”

The better way forward, in her view, is to follow the example of Europe and many other jurisdictions, to create a special pathway for access to rare disease drugs into Canada. Dr. Wong-Rieger is also calling for a special process to make new and potentially life-saving medicines available immediately – not after a market price is finalized. Sick and vulnerable Canadians, she stresses, should not be left on the sidelines when it comes to drug pricing policy.

This story was created by Content Works, Postmedia’s commercial content division and Patient Diaries, on behalf of Innovative Medicines Canada (IMC) and an IMC member company.