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Volunteer Advocate
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Questions and Answers Related to our Advocacy Work

We recently invited our online community to ask us questions about our advocacy work. We responded in the comments sections. We made a round-up of these questions and answers to make sure that others have the chance to see them too.

Q: What is the best thing someone can do right now to ensure Trikafta becomes available to Canadians who need it now, without delay?

A: We need for Canadians to continue to put pressure on the drug manufacturer, Vertex, and insist that Vertex submit Trikafta to Health Canada for review immediately. Vertex needs to understand that our advocates in Canada will be highly vocal and unwavering in the fight to gain access to life-saving treatments for Canadians living with CF.

To learn more on what you can do to help our organization advocate for access for those who need it, please visit cysticfibrosis.ca/our-programs/advocacy 

Q: Understanding that we need multi-party support to push ahead, and that MPs have the power to support our advocacy efforts, how can we both engage and inform MPs about how the current system is broken and why PMPRB changes are terrible for rare diseases?

A: We developed a package for MPs to educate them on the issue and how they can help us enact change on behalf of Canadians living with CF.

Q: I have seen international examples of letters written by CF Physicians advocating their government for urgent access to Trikafta on behalf of their patients, who cannot wait any longer. Is this something that we can encourage Canadian Physicians to take part in as well?

A: This is an approach that we have done in the past with clinicians, and we are working on something similar now for Trikafta.

Q: There has been a great deal of focus on access to Trikafta, but what is being done to get access to Orkambi – which is the only available modulator that is approved for use in children 2+? Since Trikafta would only be available for ages 12+, we will have half of our CF population under the age of 12 with no access to anything.

A: Our goal is that Canadians living with CF will have access to the treatments they need. We have been advocating for the provinces to cover Orkambi for many months now, and we have worked with clinicians to try and find prescribing conditions that will satisfy the Government so that we can provide access to these life-changing drugs to those who need it. Clinical trials for Trikafta are currently underway for children aged 6 to 11 years in Canada.

Q: Do any funds raised by Cystic Fibrosis Canada go to Vertex Pharmaceuticals for research? What is the role of CF Canada in the current negotiations between Vertex and the Canadian government regarding access?

A: Cystic Fibrosis Canada does not provide any funding to Vertex Pharmaceuticals. Together, with others in the Canadian CF Community, we are currently working with Vertex to encourage them to apply for Health Canada approval now. We’re applying pressure to the PMPRB to have a fast and fair process for these drugs. If you’re interested in helping with these efforts, please learn more on our website.

Q: In 2019 CF Canada launched a petition calling for the Canadian government to implement a rare disease strategy, and the petition is now closed. Why are you no longer accepting petition signatures, and what are the next steps for the data collected?

A: We were greatly encouraged and pleased by the volume of responses received to our petition, and by the thousands of Canadians who are being vocal about their support for a rare disease strategy in Canada. The petition was always intended as a part in our broader advocacy plan, and we are now in the process of utilizing the data collected to demonstrate the support that exists across Canada for a rare disease strategy. We are currently working on additional ways to activate our community that will continue to support our objective for better access to medicines now, and for a rare disease strategy.

Q: Is it possible to plan a peaceful rally or public demonstration intended to apply pressure related to access to Trikafta timed with an upcoming PMPRB meeting to review the submissions post deadline?

A: The PMPRB is a Canadian Federal independent semi-judicial agency. While they have had some stakeholder consultation sessions about the proposed guidelines, to our knowledge there are no public meetings that are scheduled to take place post the February 14 deadline. As at 6 February 2020, the PMPRB have not been very responsive to feedback, which makes us question if any direct action such as a rally would be an effective use of our community’s time. We are currently working on a strategy for the most effective next steps that can be taken.

Q: What amount of funds raised for CF Canada are going towards advocacy efforts? Are mediators, lawyers, or any other professional negotiators being funded or engaged to assist in our fight to secure access to life saving medications??

A: Over the last three years, CF Canada has made substantial investments in advocacy, including growing our dedicated internal advocacy staff and developing a network of more than 200 trained volunteer advocates across the country. (Find out about becoming an advocate.) We are currently utilizing all means at our disposal, including working with a highly skilled government relations expert, working with the professional members of our board of directors, and with our government relations advisory panel. We are also working closely with legal counsel to guide our actions related to advocacy.

Q: I have friends/family/loved ones who desperately need access to Trikafta and life-saving treatments – they simply cannot wait a day longer. What is CF Canada doing to help Canadians with CF get access now, and what can I do to help?

A: We understand the gravity of the situations faced by the thousands of Canadians living with CF and their loved ones. For the past 60 years we have been, and will continue to, work to improve the lives and outcomes of people living with CF – which includes getting access to the vital CF medications for all Canadians who need it. Find more information about what you can do to help us or write us directly at advocacy@cysticfibrosis.ca

Q: I’ve been reluctant to reach out to my MP/MPP about our need for help getting Trikafta to Health Canada for approval because I feel like I don’t fully understand the process and barriers that exist. What can I do to educate myself on what the issues are so that I can be a more effective and knowledgeable advocate when I reach out to my MP/MPP for help?

A: Know that you are not alone in feeling confused by all of this information, it’s very complex. Our National Advocacy Network offers online training for people who are interested in becoming a trained volunteer advocate, and our advocacy team is willing to help provide further clarification. For more information please email advocacy@cysticfibrosis.ca.

Q: What is CF Canada currently doing to help Canadians living with CF get access to Trikafta? 

A: Together with the broader CF community, CF Canada continues to put pressure on Vertex through emails, letters and meetings. We made key messages and contact information available to our community so that you can use it to write to the PMPRB (Patented Medicine Prices Review Board) about regulatory changes that will affect drug access in Canada.

Q: What information does CF Canada need to collect from the community (and countries providing access) in order to build a solid economic business case to prove that treating CF patients with access to CFTR modulator medications is more cost effective for our healthcare systems, economy, and citizens health than treating CF without access to CFTR modulators? 

A: CF Canada is currently conducting a study about the impact that CF has on individuals, families and society to inform a strong business case for coverage of these medicines. This study will include not only the direct cost of treating CF, but will also include the economic impact of time away from work, time away from school and the loss of economic contribution from people living with CF. For more information about this study, please contact advocacy@cysticfibrosis.ca.

Q: What exactly is the delay in Vertex not submitting Trikafta to Health Canada for review?

A: In short, the PMPRB’s regulatory changes are currently creating delays in applications to Health Canada. These changes are causing concerns throughout the pharmaceutical industry, including with Vertex Pharmaceuticals.

Q: Can you clarify if Canada is the only G7 country, or any country, with a similar PMPRB regulation that is currently coming into effect?

A: All countries that have a publicly supported pharmacare model do have some form of health technology assessments (HTA) to evaluate cost effectiveness of therapies.

Q: What types of things has CF Canada accomplished in the past five years with regards to national advocacy efforts, and what are the specifics of your organization’s plan moving forward?

A: We understand our community’s interest in better understanding our organization’s advocacy efforts, especially in light of recent changes with regards to new treatments and therapies available. As we work to grow our advocacy efforts across the country, one thing we have been working on is communicating better with our community. To stay up to date with our advocacy efforts, we would encourage you to follow our organization on Facebook and to keep an eye on our website for updates. You can also email us at advocacy@cysticfibrosis.ca for more detailed information.

Q: The Petition circulated by CF Canada in 2019, what are the next steps for this information? Will it be presented to the Government?

A: As an organization, we were really encouraged that more than 47,000 Canadians showed their support for a nationwide rare disease strategy by taking action on our online petition – this was an exceptional reach, beyond our initial target of 3,000 signatures! We are using these results to support our advocacy efforts as we seek to schedule meetings and have conversations with elected officials at both provincial and federal levels. Additionally, it has been an important national awareness building part of our efforts to improve access to CF drugs in Canada. 

We are considering the role that a formal, hard-copy government petition might play in future, however these are resource intensive endeavors. We did conduct a government petition campaign for access to Orkambi in 2018, and those petitions helped gather more than 10,000 signatures.

Q: Can you explain the difference between the Compassionate Access Program and the Special Access Program for drugs (SAP)? Are they two different avenues for accessing drugs that haven’t been approved yet? Does the patient pay for them if approved, or does the drug company? Does access end once the drug has been approved by Health Canada?

A: The Special Access Program (SAP) is a program that is offered by Health Canada for drugs that are not currently approved for sale in Canada and for individuals with demonstrated medical needs. The Compassionate Access Program is offered by the drug manufacturer who provides the approved drugs in Canada for individuals with demonstrated medical needs who cannot afford them. Someone who has been granted compassionate access under either program will continue on the drug indefinitely. However, if circumstances change, either program can be terminated. For example, if the drug is approved for sale in Canada but not reimbursed by the public healthcare system, the manufacturer may choose to terminate the program meaning no new individuals will be granted compassionate access.

Q: Can CF Canada provide a forum for patients and caregivers to communicate with one in order to strategize the best way to get access to these life changing drugs?
A:  CF Canada regularly hosts open calls, Facebook Live sessions and webinars to provide updates about our national advocacy efforts. Volunteers in our National Advocacy Network work collaboratively as a team to strategize and execute government relations initiatives to help improve public policy that directly impacts people with CF. Lead Advocates in the National Advocacy Network do host conference calls and chats regularly. For more information on how to get involved with the National Advocacy Network please contact advocacy@cysticfibrosis.ca.

Q: What can we do that you can’t?
A:  Right now we need to continue pressuring Vertex to submit Trikafta to Health Canada asap. Let them know that we are aware of their concerns and will be unwavering in the fight to gain access here. In addition we need to let the Patented Medicine Prices Review Board (PMPRB) know about our concerns over how the recent regulatory changes will affect access in Canada. We will be providing key messages that you can send to the PMPRB. We need to continue to meet with both provincial and federal elected officials. To stay up to date with our advocacy efforts, follow us on Facebook or keep an eye on our website for updates. You can also email us at advocacy@cysticfibrosis.ca for more detailed information.  

Q: Do we have an understanding of when clinical trials for Trikafta would happen for the two to 11 years group age?

A: Clinical trials always proceed in sequence; first adults, then 12 yrs and up, then 6-11 yrs, then 2-5 yrs, then, if desirable, children under 2, etc. In young subjects the emphasis is on safety. A phase III trial for Trikafta in children aged 6-11 was first posted in October 2018, and is currently recruiting.

Q: What is the best way for me to help put pressure on Vertex with regards to access to medications in Canada?

A: Letters can be sent directly to Vertex Canada encouraging them to apply to Health Canada for a priority review (180 day review).

Q: Can you elaborate on CF Canada’s strategic plan for advocacy in 2020?

A: As an organization, it’s important that our advocacy efforts are flexible, so we can respond in a timely fashion to things like the early approval of Trikafta in the USA, as well as the devolvement of the PMPRB consultation process. We are also conducting a number of studies to help support our advocacy efforts with evidence to have the greatest impact. One will study the burden that CF places on individuals, families, and society and will include not only the direct cost of treating CF but also the economic impact of time away from work, time away from school and the loss of economic contribution resulting from a loss of independence and productivity in general. In addition, we’re looking at the long-term benefits from CFTR modulators over time using Registry data as well as modeling the difference to our CF population of having, or not, the CFTR modulators. We’re currently recruiting specialized external advisors, engaging partners with aligned interests, will be hosting a day on the hill, and ensuring ongoing support for our advocates across Canada.

Q: Will a public rally and media relations be a part of CF Canada’s strategy for raising awareness and creating pressure on the entities that currently hold power to move things along regarding access, government and otherwise?

A: Cystic Fibrosis Canada and the volunteer program, The National Advocacy Network, have executed rallies in the past. Rallies can be an effective tactic, especially when the government is unresponsive to our ask or not willing to meet or not acting diplomatically. CF Canada has also conducted receptions, awareness days, and lobbying days at provincial legislatures. We encourage our volunteers and the community as a whole to reach out to their local elected officials and build relationships. We need elected officials at the Provincial and Federal level to understand that these new changes to the PMPRB and the lack of a separate drug review process for drugs for rare diseases are significant barriers to new medications, such as Trikafta, coming to Canada.

The Government Relations and Advocacy Team as well as the Communications Team at CF Canada work closely together on media relations efforts and raising public awareness. Our strategy moving forward will include both media relations and meetings with elected representatives and civil servants.

Q: Has CF Canada been able to meet with Provincial Premiers to make the case for access to Trikafta and other modulators for Canadians living with CF?
A: CF Canada has met with Premier Ford, along with other provincial premiers and ministers of health, to discuss access to CF medicines and the importance of a rare disease strategy. We will continue to apply pressure to the provincial and federal government to improve access to CF medicines and encourage all levels of government to develop and implement a separate, fast and fair review system for drugs for rare diseases.

Got unanswered questions? Send them to advocacy@cysticfibrosis.ca

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