Cystic Fibrosis Canada (CF Canada) launched a national campaign in May, which is Cystic Fibrosis Awareness Month, to call on the government to make life-changing medications accessible to those living with (CF).
Orkambi (ivacaftor/lumacaftor), developed by Vertex Pharmaceuticals, was approved in the United States in 2015 for CF patients 12 and older who have two copies of the F508del mutation in the CFTR gene. In the two studies that cleared the safety and effectiveness of Orkambi for approval, the medicine was found to effectively improve lung function and reduce the number of pulmonary exacerbations.
Orkambi potentially could benefit 2,100 of the 4,200 Canadians who live with CF today. Unfortunately, without public funding, many people cannot afford this therapy, despite its approval by Health Canada in January 2016.
In fact, the approval of Orkambi in Canada was not the smoothest. In August 2016, a few months after approval, the Canadian Drug Expert Committee (CDEC) of the Canadian Agency for Drugs and Technologies in Health (CADTH) announced a meeting to reconsider Vertex’s submission for Orkambi after the CDEC shared an embargoed recommendation for the drug in June that year.
National agencies were concerned about the drug’s optimal use and its sustainability in public drug plans, after concluding there was not enough evidence to prove its effectiveness.
British Columbia’s public drug program, PharmaCare, currently lists 15 high-cost drugs for rare diseases; the addition of Orkambi to coverage would add an estimated $29 million per year in costs to the program.
However, members of CF Canada were disappointed that patients still had to wait longer than predicted to have the drug available through public funding.
In 2017, Vertex and CADTH were still negotiating Orkambi’s public plan.
“Cystic fibrosis is a progressive, fatal disease. People with CF don’t have time to wait,” John Wallenburg, chief scientific officer at Cystic Fibrosis Canada, said in a press release. “Our governments need to step up and help these people now. Those with cystic fibrosis need to rely on more than their hope, they need to be able to rely on their governments to make decisions that will help them live longer, healthier lives.”
To that end, CF Canada is holding a national campaign, titled “Let’s Hope” to call on Canadians to give more than hope and support the mission of ending CF. The campaign features Canadians with CF sharing their hopes and fears, and is designed to show the government that Canadians support access to medication for people with CF.
Canadians also can show their support at the local Walk to Make Cystic Fibrosis History, taking place on Sunday, May 27.