New federal drug pricing regulations mean disaster for Canadians with rare diseases

  • New regs unfairly and unnecessarily target rare disease drugs and all new medicines – including life-saving cancer and curative gene therapies
  • Proposed national pharmacare – including the Drugs for Rare Diseases Program – will be rendered meaningless – because there will be no new drugs
  • Canada has just become the worst country in the world to bring in new drugs and to conduct clinical trials for next-generation therapies

Ottawa, August 9, 2019 – Today, the federal government made it clear they either don’t understand – or they don’t care – about the devastating impact of the new drug pricing regulations on the 1 in 12 Canadians with rare disease – two-thirds of them children – or any patient waiting for a life-saving new therapy.

The federal government has finalized drug pricing regulations that would require companies to introduce new drugs at prices that are so low, they simply will not bring them to Canada.  This means that Canadian patients will have no access to clinical trials or new medicines until they have been approved in most other developed countries. These actions are most harmful to patients with severe, progressive and life-threatening conditions who have no other effective treatments.

All Canadians want to have access to medicines at affordable prices. However, the regulations changing how the federal Patented Medicine Prices Review Board (PMPRB) works are targeted at those medicines that inevitably have higher-than-average per-patient prices. These are medicines for small patient populations (like rare diseases), precision medicines for cancer patients who have failed traditional therapies, and curative therapies like advanced stem cell transplants and gene therapies.

The federal government says it will work in consultation with patients and other stakeholders over the next 11 months to develop the guidelines for implementing these changes.  Unfortunately, patients are entering into these discussions with very little faith that the government will truly try to understand and meet the needs of patients. After all, we just concluded nearly a year-long consultation where they absolutely refused to listen and made almost no changes to their draft regulations.

“Either the federal government can’t understand the disastrous impact on patients with high needs or they simply don’t care.” said Durhane Wong-Rieger, President and CEO of the Canadian Organization for Rare Disorders (CORD). “We are very concerned that the next 11 months of consultation will result in no better results than the past 11 months of discussions.”

“Beyond high-price medicines, these regulations will only encourage the United States to expand on their recently announced plans to ‘import cheaper medicines from Canada’ and compound our existing drug shortage problems,” Wong-Rieger notes. “Sadly, this federal government has also not offered any deterrent plan of action, other than to state that Canadian patients will not be harmed.”

In its determination to proceed no matter what feedback was given during the consultation period, the government even misrepresented the impact of the changes as presented in a report it commissioned by former Bank of Canada governor David Dodge and refused to make public, though it was by CORD and is available here.

The government is proceeding in the face of repeated warnings from CORD and hundreds of other organizations, including patients, researchers and developers. “At the end of the day, I have to ask: are they stupid or just uncaring?  I don’t think they are really that stupid,” noted Wong-Rieger, who was part of the Steering Committee for how the PMPRB would operationalize the regulations.

The federal government claims that these regulatory changes are important. They say it will reduce the drug budgets. We know there are many ways of lowering drug expenditures without harming patients. As an organization that represents patients with rare disorders, CORD fully supports the goal of making medicines affordable. But these proposed regulations are not the right way. Other countries have different ways of setting drug prices, and we should be adopting some of those approaches. No one is doing what Canada proposes because they are aware of the harm to patients.

The government is simply bulldozing the changes through in mid-summer before an election without taking into account the serious issues raised by this report and the input of hundreds of stakeholders, including CORD.

About CORD
CORD, the Canadian Organization for Rare Disorders, 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. www.raredisorders.ca

For further information:
Durhane Wong-Rieger
President & CEO, the Canadian Organization for Rare Disorders (CORD)
Tel: 416-994-0077
Email: durhane@sympatico.ca