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Explained: the PMPRB’s impact on access to CF medicines and what you can do to help

The Patented Medicine Prices Review Board (PMPRB) and its proposed draft guidelines have become a major concern in the Canadian cystic fibrosis (CF) community. This is because they are impacting access to the break-through cystic fibrosis medication, Trikafta, and will likely impact access to other CF medications into the future.

Cystic Fibrosis Canada wants to help you better understand the PMPRB, their guidelines, how they’re impacting access to medicines and what you can do to help, particularly as we prepare to share our response to these draft guidelines and the actions we can take together. Read on for an overview, including background info, current events and next steps.

What is the PMPRB?

The Patented Medicine Prices Review Board (PMPRB) is a quasi-judicial independent federal agency that regulates the maximum amount that companies can charge for their medicines in Canada. It forms part of Canada’s drug regulatory and review system, along with other bodies such as: the Canadian Agency for Drugs and Technologies in Health (CADTH), Institut national d’excellence en santé et services sociaux (INESSS) and the pan-Canadian Pharmaceutical Alliance (PCPA).  

When a drug comes to Canada it must go through Canada’s drug regulatory, review and reimbursement system. The drug must go through various aforementioned review bodies before it is eligible for public reimbursement. The first step in the review process is approval by Health Canada for safety, efficacy, and quality. The drug then goes to the PMPRB for a pricing review.

This means that before any drug can be sold in Canada, it must be reviewed by the PMPRB. It is their job to regulate the prices of patented medicines sold in Canada to ensure that they are not excessive. 

A ‘patented medicine or drug’ is one that is covered under patent protection. For the most part, this means that only the pharmaceutical company that owns the patent is able to manufacture, market and profit from the drug during the life of the patent, which means there are no generic options available to consumers while the patent is valid.

How are the PMPRB guidelines negatively impacting access to CF medications?

The PMPRB has a set of guidelines that inform the regulation of patented drug pricing in Canada. The contents of the guidelines are very important to drug manufacturers, as they influence how their drugs will be assessed for pricing and ultimately the price at which their drugs may sell within Canada.

These critical guidelines are currently in the process of being updated by the PMPRB and the new proposed guidelines include changes that are creating uncertainty for drug manufacturers. This uncertainty is seeing Canada left behind as other countries across the world, like the USA, UK, as well as the European Medicines Agency, negotiate access to the life-changing cystic fibrosis medicine, Trikafta.

The proposed guidelines are making Canada a less desirable place for drug manufacturers to bring their drugs, and Canadians, including those living with cystic fibrosis, are missing out on critical medicines as a result.

How does this affect people living with CF in Canada?

Cystic fibrosis is a genetic disease and while there have been effective therapies and treatments developed to treat the symptoms of the disease, the best health outcomes to date have been from gene modulating drugs.

Gene modulating drugs treat the genetic defect that causes cystic fibrosis. They are innovative therapies that can change the lives of the people who need them.

Currently, there are four modulators of the cystic fibrosis CFTR gene that exist. All of them are made by manufacturer Vertex Pharmaceuticals. These modulators are Kalydeco, Symdeko, Orkambi and Trikafta.

There are more than 1,700 genetic mutations of the CFTR gene that can cause cystic fibrosis. This means that a modulator that treats one patient may not be effective for another.

To date, three of the CF modulators have been submitted to the Canadian drug regulatory system. All received approval at the first step in the system, a review by Health Canada to ensure the drugs are safe and effective for human use. However, the manufacturer put only two of these drugs –Kalydeco and Orkambi  – through the public review and reimbursement system. Cystic Fibrosis Canada continues to pressure Vertex Pharmaceuticals to submit Symdeko to Health Canada. Presently it is only available through some private insurance programs.

The Canadian drug review and reimbursement system’s overly restrictive drug eligibility criteria means that these life-changing CF modulator drugs have had a difficult time finding their way to public reimbursement after Health Canada approval.

In the eight years since modulators were first brought to Canada, we’ve seen little progress and access is incredibly limited. 

Kalydeco has become publicly funded for those with the G511D mutation, and is still under negotiation to cover the remaining mutations indicated by Health Canada. Kalydeco treats 4% of the CF population in Canada.

Orkambi, which treats up to 50% of the cystic fibrosis population has extremely limited access for children in Alberta, Saskatchewan and Ontario. It is also available on a case-by-case basis in Quebec.

More alarmingly still, Trikafta, considered a breakthrough drug that can treat up to 90% of people living with cystic fibrosis in Canada, has not even been submitted to Health Canada yet. This is, according to the manufacturer, is due to the PMPRB’s proposed new guidelines that make it nearly impossible for them to bring such therapies to Canada.

Canadians living with cystic fibrosis have limited to no access to the life-changing drugs they need and this is unacceptable. We need access to all CF modulator drugs now.

What can the CF community do to help?

Since January 2020, Cystic Fibrosis Canada, together with the cystic fibrosis community, has been calling on the PMPRB to stop the implementation of their revised guidelines and make changes that will remove the barriers for innovative CF medications to come to Canada.

The CF community rallied with us and we were pleased when the PMPRB announced on June 1 2020 that they were delaying the implementation of their proposed guidelines. They subsequently released new draft revised guidelines and invited stakeholders to submit written responses by Tuesday, August 4 2020.

Cystic Fibrosis Canada is preparing a response to the draft guidelines, working together with independent third party experts, community groups (e.g. CF Get Loud), healthcare coalitions, (e.g. Best Medicines Coalition, Health Charities Coalition of Canada) and other organizations (e.g. the CF Treatment Society and the Canadian Organization for Rare Disorders) to make it as strong and thoughtful as possible.

When our response is final, we will be sharing it publicly with our community and with Members of Parliament throughout the country. We will also provide an advocacy toolkit that members of our community can use to meet with their Members of Parliament about the changes and access to Trikafta. These tools will also include a template letter to the PMPRB to show your endorsement for our response to their draft guidelines and advice for how to engage your local Members of Parliament on these issues.

How to take action

Here are a few of the upcoming ways you can help us go further in the fight for better access to life-changing cystic fibrosis medicines:

  1. Read our written response to the PMPRB revised draft guidelines – Tuesday, July 28 2020
  2. Send a letter to the PMPRB, using our pre-prepared template, to show your support for our response and recommendations – Tuesday, July 28 2020
  3. Learn more about these issues by joining our webinar discussing the PMPRB guidelines and our submission – Thursday, July 30 2020
  4. Engage with your local MP using a letter template we will provide