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:
To get Trikafta submitted, fast-tracked and approved by Health Canada.
To get public funding for all modulator drugs that are currently approved in Canada.
To have a regulatory and reimbursement environment in Canada that does not unnecessarily limit our access to new life-saving therapies.This includes, but not limit to, stopping the proposed changes to Patented Medicines Prices Review Board (PMPRB) and creating a fast and fair review and reimbursement process for drugs for rare diseases and precision medicines in Canada.
Our work towards this includes:
Increased pressure on the federal and provincial governments (including the PMPRB) to do the right thing, through media, meetings, letters, formal submissions, consultations and much more.
Increased pressure on the drug manufacturer to work with the government, through meetings and ongoing discussions.
Continued activation and management of our National Advocacy Network of more than 250 trained volunteers across the country, to support the above in all provinces.
Increased resourcing towards these goals, including dedicated government relations consultants and communications professionals.
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.
Symdekois 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 systemis 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.
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).
Join us for a fun night of laughs on Thursday, June 17th at 8:00 p.m., just in time for Father's Day! Put the children to bed and pull up a chair for some good laughs about adulting. This show is virtual but NOT pre-recorded - it's live. We promise there will be NO bad Dad jokes!