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Volunteer Advocate


Provinces deal another blow to Canadians living with Cystic Fibrosis
November 14, 2017

Toronto, ON (November 14, 2017) – Cystic Fibrosis Canada is extremely disappointed that provincial governments have decided not to negotiate the price of Kalydeco, an effective cystic fibrosis (CF) medication, for Canadians with CF who have specific mutations and could potentially benefit from this drug.  

Currently, only those with one specific genetic mutation can access Kalydeco under provincial health insurance plans. In the United States, the government covers the cost of Kalydeco for people with 38 variations of the genetic mutation, meaning that those who need it are not forced to pay for this costly drug on their own.   

The news that the provinces will not negotiate the price of Kalydeco for the remaining mutations suggests that certain individuals are being discriminated against because of their genetics, excluding them from benefitting from this medication for something that is completely out of their control, and for reasons that are unknown.      

Recently, Health Canada removed all references to the orphan (rare disease) drug regulatory framework from its website. The framework, which was announced in 2012 and was expected to be implemented in 2017 to 2019, would have set out standards for how to look at rare disease drugs separately from common drugs. To CF Canada’s knowledge, the framework has been placed under a new Drug Regulatory Plan known as the Regulatory Review of Drugs and Devices initiative. CF Canada is hopeful the new framework will address the same underlying issues we are facing in Canada, and will be implemented as soon as possible.  

Without a designated rare disease framework, CF Canada fears pharmaceutical companies that produce rare disease drugs will not offer their medicines in Canada. In addition, Canada may not be able to support and attract research into treatments for rare disease research and clinical trials, leaving people with rare disease such as cystic fibrosis at a disadvantage.

All of this comes at a time when Canadians living with CF are facing the biggest battle of their lives as it relates to accessing life-changing medications. CF Canada has been actively advocating provincial governments to reopen negotiations on the price of Orkambi, a life-changing CF medication that has been approved by Health Canada and is being prescribed by doctors, but is being refused coverage by the provinces.

On November 1, Vertex Canada, the makers of Kalydeco and Orkambi, presented the provinces with a proposal on Orkambi at a what the company describes as a significantly reduced price. CF Canada is urging the provinces to negotiate with Vertex and to reach an agreement on price, so that over a thousand Canadians living with cystic fibrosis who could potentially benefit from this drug will finally be able to access it. That is over a third of the CF population in Canada.

The time is now for the provinces to make good on their promise to protect the health and wellbeing of their residents, particularly those in such a dire situation. Recent breakthroughs in developing drugs could substantially improve the lives of thousands, but without the provinces’ support, the majority of those who need these drugs will never get them.  


Cystic Fibrosis

Cystic fibrosis is the most common fatal genetic disease affecting Canadian children and young adults. It is a multi-system disease that affects mainly the lungs and the digestive system. In the lungs, where the effects are most devastating, a build-up of thick mucus causes severe respiratory problems. Mucus also builds up in the digestive tract, making it difficult to digest and absorb nutrients from food. As improved therapies have helped to address the malnutrition issues, ultimately most deaths related to cystic fibrosis are due to lung disease. There is no cure.


Cystic Fibrosis Canada

Cystic Fibrosis Canada is one of the world’s top three charitable organizations committed to finding a cure for cystic fibrosis and is an internationally-recognized leader in funding CF research, innovation, and clinical care. We invest more funding in life-saving CF research and care than any other non-governmental agency in Canada. Since 1960, Cystic Fibrosis Canada has invested more than $244 million in leading research, care and advocacy, resulting in one of the world’s highest survival rates for Canadians living with cystic fibrosis. For more information, visit


For more information, please contact:

Phil Norris, Senior Manager, Corporate Communications
Cystic Fibrosis Canada

Tel: 416-485-9149 ext. 291
Tel: 1-800-378-2233 ext. 291

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