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Federal Government Announces Progress on Nation-wide Strategy for Rare Disease Drugs

March 22, 2023

Cystic Fibrosis Canada calls on the provinces and territories to implement strategy and improve access today

TORONTO, March 22, 2023 – Today, the federal government announced its approach to a 2019 commitment to improve drug access for Canadians with a rare disease, a strategy that Cystic Fibrosis Canada with the Canadian cystic fibrosis (CF) community has long advocated for. At the announcement in Montreal today, the government committed up to $1.5 billion over three years in support of the first ever National Strategy for Drugs for Rare Diseases. Much of the funding will go to the provinces and territories to support improved access to emerging and existing rare disease drugs through bi-lateral, individualized agreements. It is unclear what this means specifically for the CF community.

“This is a good start. We are pleased to see this news today as we have long advocated for a nation-wide strategy for rare disease drugs alongside our community," said Kelly Grover, President and CEO, Cystic Fibrosis Canada. “But there is still much work to be done as we learn how the government's approach will be implemented and how quickly it will take for the provinces and territories to step up before we know the impact it will have on our community."

In its 2019 budget, the federal government committed $1 billion over two years, with up to $500 million per year ongoing, for a national rare disease strategy to help Canadians with rare diseases get the medicines they need. Until today, there had been little movement on the details of this promise, while other developed countries have had a strategy for many years, including the United States’ Orphan Drug Act, which has been in place for 40 years.

Today’s announcement included funding for research and improved data sharing for rare diseases, as well as the creation of centres of expertise and the establishment of a stakeholder Implementation Advisory Group which will provide advice and continued engagement as the plan is unrolled. Cystic Fibrosis Canada will be seeking to understand how rare diseases like ours with existing infrastructure already in place--such as our registry, CF clinics and clinical trials network—can leverage this funding to ensure we deliver the greatest impact for our community.

“We are glad to see decision makers have recognized the specific challenges that Canadians impacted by rare diseases experience when it comes to accessing the drugs they need,” said Kim Steele, Director of Government and Community Relations for Cystic Fibrosis Canada. “But we will be counting on the provinces and territories to make this a true success story for Canadians living with rare diseases. We need buy in from all levels, in particular the provinces and territories to act swiftly to help those in our community who are still struggling to access the medications they need or those paying deductibles that are too high.“

For years, Cystic Fibrosis Canada and the Canadian CF community have advocated for access to life-changing CF drugs. Because we did not have a nation-wide approach and commitment to funding drugs for rare diseases, the community had to advocate relentlessly for the single-greatest innovation in the history of CF every step of the way, from Health Canada approval, to the Patented Medicine Prices Review Board (PMPRB), the Canadian Agency for Drugs and Technologies in Health (CADTH) draft and final recommendations, to each and every jurisdiction in Canada. The drug is now funded across Canada for eligible patients aged six and up. Cystic Fibrosis Canada has done this difficult, but important work before, and will continue to work hard to ensure our community gets the fair and equitable access it needs to live healthy lives.

Cystic Fibrosis Canada is encouraged the proposed approach includes a patient input mechanism through the Stakeholder Implementation Advisory Group and we look forward to working with our community and the government to implement this roll out for rare disease drugs.

“I have been working with Cystic Fibrosis Canada as a patient advocate on the rare disease strategy for as long as I can remember," said Hailey Laxer, a law student at McGill who lives with CF and was present at the press conference today. “It is rewarding to see the government prioritize this today. I look forward to seeing how the government works with the rare disease community to move forward to improve the lives of people across the country.”

Since 2019, Cystic Fibrosis Canada has asked the federal government to honour its commitment to implement a strategy for drugs for rare disease. Cystic Fibrosis Canada has asked that this strategy improve access to drugs for rare diseases, be collaborative and sustainable, with defined roles for both public and private payers, while streamlining drug review processes and reducing duplication, to expedite access to drugs for rare diseases.

Cystic Fibrosis Canada is pleased to see this first step in a rare disease strategy while honouring and remember the people we have lost along the way to today’s announcement.


Members of the community and supporters are invited to join a Facebook Live today, Wednesday March 22 at 7:30 p.m. ET on the Cystic Fibrosis Canada Facebook page. Hosted in English, the event will bring the community together to discuss this step forward in improving access to rare disease medications.

About cystic fibrosis

Cystic fibrosis is the most common fatal genetic disease affecting more than 4,300 Canadian children and young adults. There is no cure. Of the Canadians with cystic fibrosis who died in the past five years, half were 37 years old. Cystic fibrosis is a progressive, degenerative multi-system disease that affects mainly the lungs and digestive system. In the lungs, where the effects are most devastating, a build-up of thick mucus causes severe respiratory problems. Mucus and protein also build up in the digestive tract, making it difficult to digest and absorb nutrients from food. In addition to the physical effects of the disease, mental health concerns are emerging; anxiety and depression are common among this population. Double lung transplants are the final option for patients with end-stage disease; most fatalities of people with CF are due to lung disease.

About Cystic Fibrosis Canada

Cystic Fibrosis Canada has dramatically changed the cystic fibrosis story. We have advanced research and care that has more than doubled life expectancy. Since being founded by parents in 1960, Cystic Fibrosis Canada has grown into a leading organization with a central role engaging people living with cystic fibrosis, parents and caregivers, volunteers, researchers and healthcare professionals, government and donors. We work together to change lives for the more than 4,300 Canadian children and adults living with cystic fibrosis through treatments, research, information and support. Despite our remarkable progress together, we are not yet done. Not when half of the Canadians with cystic fibrosis who died in the past five years were 37 years old. We will keep pushing, keep going further until all people with cystic fibrosis can and do experience everything life has to offer —and enjoy everything life has to offer. Learn more at


For more information:

Nicole Young, Director, Marketing and Communications

Tel: (905) 317-5529




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