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Volunteer Advocate

Access to Medicines

Access to medicines is a huge priority for everyone in the cystic fibrosis (CF) community, and it’s a huge priority for Cystic Fibrosis Canada.

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

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. In June of 2021 Trikafta was approved by Health Canada. Learn more about Trikafta. 

Unfortunately, a lack of access to CF modulators means that to date, very few people living with CF in Canada currently benefit from these advancements. But we are gaining ground. We are going further to gain access to modulators for all who need them. 

In addition to the work we do to gain broad access to modulators, we 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.

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) was submitted for approval in Canada in December 2020 and approved by Health Canada in June 2021. 

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.

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). 

One year later, the pCPA announced that they have agreed on a negotiated price for both drugs, for all Health Canada approved mutations. Although Orkambi and Kalydeco were held up in negotiations for quite some time, this is a step in the right direction for the CF community. The next step is for all the provinces in Canada to immediately fund Orkambi and Kalydeco, in their provincial drug plans. For more information see our Q&A. 

What is the pcpa?

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.

For more information please see the Q&A.