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Volunteer Advocate

Access to Medicines

Access to medicines is a huge priority for everyone in the cystic fibrosis (CF) community. 

Highly effective CFTR modulator therapies now exist, improving health outcomes and helping people with cystic fibrosis live longer, healthier lives. 

Unfortunately, a lack of access to these medications means that very few people living with CF in Canada currently benefit from these advances. 

Cystic Fibrosis Canada is calling on government to implement a fast and fair process for disease-modifying therapies like Trikafta. We also apply pressure on provincial governments to cover symptom management drugs for CF.  We have had successes, but there are still far too many gaps in access to CF medications and disparities among provinces in how they are funded. 

In the fall of 2019, the newest CFTR-modulating medication, Trikafta, was designated a breakthrough medication in the U.S. and fast-tracked for approval. CF Canada and the Canadian CF community sprang into action, working hard to overcome the barriers to access for Trikafta in Canada. Learn more about Trikafta

For the latest news on CF Canada's advocacy work, please visit our blog.


Important DRUG ACCESS priorities 

Our CEO, Chief Scientific Officer,  program team, volunteers and Board are pushing for three things:

Our work towards this includes: 

Kalydeco (ivacaftor) is a pill for people ages 6 and older who have the G551D mutation of CF which helps the defective CFTR protein work at the surface of the cell.

Orkambi (lumacaftor/ivacaftor) is indicated for the treatment of cystic fibrosis (CF) in patients 2 years of age and older who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Symdeko is a precision medicine that is used to treat cystic fibrosis in people who have a particular set of genetic mutations. A disease-modifying drug, it is a combination of ivacaftor and tezacaftor. 

Trikafta (elexacaftor/ivacaftor/tezacaftor) has not been submitted for approval in Canada. CF Canada is pushing hard for Canadians to have access to this life-saving medication. 


Understanding the Canadian Drug System

Canada's drug review and reimbursement system is already onerous, duplicative, and inadequate. It can take between 2-5 years for some drugs to get to patients, and even longer for precision medicines and drugs for rare diseases, if these medicines get to patients at all. These regulatory changes will make it even more difficult to get life-changing and life-saving medicines to patients.


PMPRB changes

On Thursday, July 30, 2020 at 1 p.m. EST, Cystic Fibrosis Canada will be hosting a one hour Q&A webinar session hosted by Dr. John Wallenburg, Chief Scientific Officer, and Kim Steele, Director, Government and Community Relations, for Cystic Fibrosis Canada. The webinar will focus on the PMPRB, our response to the revised draft guidelines and what they might mean for access to life-changing CF medications, and how you can take action to support the policy amendments we will be calling for. If you would like to submit a question on any of these topics, please send an email to advocacy@cysticfibrosis.ca before July 30.

Please join the webinar by clicking this link: https://us02web.zoom.us/j/82881482662

On June 19, 2020, the Government of Canada released draft guidelines to the regulations of the Patented Medicines Prices Review Board (PMPRB). We are carefully reviewing the revised draft and based on the content, we will determine how we continue to push forward in our relentless fight to get life-changing CF drugs into the hands of those who need them.

Here are our next steps:

We will share more information shortly. 

In May of 2017, Health Canada proposed changes to the Patented Medicines Prices Review Board (PMPRB) regulations.  These changes incorporate new factors in determining whether a medicine is being or has been sold at an “excessive” price. 

Since the changes to the Patented Medicines Regulations were first proposed, the patient community has consistently raised concerns that these changes will negatively impact access to new medicines for Canadian patients.

The new PMPRB regulations require patented drug manufacturers to significantly reduce their prices. By some estimates, these changes will require reductions in price as high as 75%, but the actual reductions depend on a number of assumptions that themselves are creating uncertainty.  Upon implementation, Canada will become an outlier among OECD countries and a much less attractive market in which to launch innovative therapies, including precision medicines that can alter the course of devastating diseases like cystic fibrosis.

The impact these regulatory changes have had is chilling. From November 1, 2019 to February 29, 2020 Health Canada registration of new clinical trials decreased by 60% compared with the average of four years prior. Moreover, “between 2015 and 2018, 49 per cent of new drugs were approved in Canada either prior to or within a year of their approval in the United States. In 2019, the rate fell to just 15.6 per cent. That’s a drop of more than two-thirds.” - Nigel Rawson, & John Adams, Price controls imposed by shortsighted politicians keep new life-saving drugs out Financial Post. Retrieved from https://business.financialpost.com/opinion/price-controls-imposed-by-clueless-politicians-keep-new-life-saving-drugs-out 

The PMPRB, and the federal government, have repeatedly assured patient groups that access to new medicines will not be impacted by the proposed regulatory changes. Unfortunately, we now know that access to new drugs for Canadians with cystic fibrosis has not only been delayed, but may be denied altogether.


pCPA Negotiations with the Drug Manufacturer

On June 12, 2020, in a promising development for Canadians living with cystic fibrosis, the pan-Canadian Pharmaceutical Alliance (pCPA) announced their agreement to begin negotiations with CF drug manufacturer, Vertex Pharmaceuticals.

This pCPA is made up of drug program leaders from all provincial, territorial and federal governments who work collaboratively to negotiate drug prices for their repsective programs. Its pricing decisions are a precondition of having a medication covered on provincial drug formularies.

The two medications subject to negotiations, Orkambi and Kalydeco, are known as CFTR-modulators. Rather than just treating symptoms, these drugs target the basic defect from specific mutations that cause cystic fibrosis (CF).

This is a positive step in a complex process.

For more information please see the Q&A.