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KALYDECO® (ivacaftor) is a prescription medicine for the treatment of cystic fibrosis in patients ages six and older who have the specific G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or G970R mutation, or ages 18 and older who have the R117H mutation in the Cystic Fibrosis Transmembrane Regulator (CFTR) gene. In Canada, the number of patients with these mutations is about 180. Kalydeco, a pill taken twice a day with fat-containing food, helps the protein made by the CFTR gene function better and, as a result, improves lung function and other aspects of cystic fibrosis such as increasing weight.
On November 26, 2012, Health Canada approved Kalydeco for use in Canada for patients with the G551D mutation. Approval for the additional 9 mutations was received on June 6, 2014, and approval for R117H was received on March 13, 2015.
On March 26, 2013, it was announced that the Common Drug Review’s (CDR) Canadian Drug Expert Committee (CDEC) recommends that Kalydeco be listed on the formulary listing of publicly funded drug plans for the treatment of cystic fibrosis in patients age six and older who have the G551D mutation in the CFTR. Cystic Fibrosis Canada submitted patient input to CDEC for the additional mutations on July 22, 2014 and it received a positive recommendation on December 19, 2014.
Following the CDR recommendation for G551D, Kalydeco was subject to the Pan-Canadian Pharmaceutical Alliance (PCPA), where negotiations with Vertex were led by Alberta on behalf of the provinces (with the exception of Quebec). A letter of intent was entered into between the participating PCPA jurisdictions (Quebec excluded) on a pricing agreement for Kalydeco on June 16, 2014. A letter of intent typically sets out the standard terms for health ministries and Vertex when considering provincial coverage for Kalydeco. The additional 9 gating mutation indications are currently going through the PCPA process with Alberta again leading negotiations.
Each provincial health ministry considers how Kalydeco will be funded in accordance with its own drug reimbursement framework. Kalydeco is currently reimbursed in Ontario, Alberta, Saskatchewan, Nova Scotia, Manitoba, New Brunswick, Yukon and British Columbia for individuals with G551D above the age of 6. Cystic Fibrosis Canada continues to advocate the remaining provinces to make Kalydeco available to patients who need this medication, and will advocate for the addition of the nine gating mutations when the LOI is signed. We are following the R117H indication as it begins to make its way through the Health Technology Assessment (HTA) process and will submit patient evidence at the appropriate time.
Quebec has its own drug review process through the Institut national d’excellence en santé et en services sociaux (INESSS). We recommend that patients in Quebec speak to their CF clinics regarding access through the Patient D’Exception program.
For patients with private insurance who have been prescribed Kalydeco by their healthcare provider for one of the 10 mutations indicated, kalydeCARE™ is a Patient Assistance Program which can help navigate the reimbursement system. For more information please call: 1-855-CARE-571 (1-855-227-3571).
In recent news, Kalydeco is in clinical trials as part of a combination study with lumacafor for patients with the homozygous DF508 mutation. We encourage patients taking part in these trials to reach out to us at firstname.lastname@example.org and share their experiences which will help us to gather testimonials for a Patient Input Submission to the CDR process once the combination therapy enters the HTA process.
We want to thank all our advocates especially those patients and families personally impacted by Kalydeco for their tenacity and courage in the fight for Kalydeco.