Radio-Canada published an intriguing digital feature on Trikafta, a drug that has profoundly improved lung health for many cystic fibrosis patients, transforming both their present lives and future prospects. Dr. Elias Matouk, Director of the Cystic Fibrosis Clinic at the MUHC and a researcher in the RESP Program, highlighted the potential psychological challenges patients may face when adjusting to a future that now appears far brighter than they had anticipated. Dr. Larry Lands, Director of Respiratory Medicine at the Montreal Children’s Hospital and Senior Scientist in the RESP program, also shared insights on the advancements achieved with this treatment. You can explore the first and second parts of the report and tune in to Ça nous regarde, featuring an interview with reporter Ariane Labrèche on Radio-Canada.
In 2019, Trikafta, a breakthrough medication, transformed the treatment of cystic fibrosis by improving lung function and reducing severe symptoms for many patients. Many patients found themselves laughing without coughing, pursuing dreams, and, for the first time, breathing freely. While it is not a cure, its impact has been life-changing, significantly reducing the need for lung transplants. For example, at the Montreal Chest Institute, lung transplants for cystic fibrosis patients have dropped from 20 a year to just one.
However, Trikafta is extremely expensive, costing over $300,000 per patient annually in Quebec. Beyond the financial burden, some patients experience side effects like liver issues, and many face new psychological challenges as they adapt to a dramatically different outlook on life. Furthermore, the drug only works for patients with a specific genetic mutation, F508del, which accounts for about 90% of cystic fibrosis cases. This leaves some patients with other, rarer, mutations unable to access Trikafta’s life-changing effects.
Even for those eligible, access varies widely. In Canada, some provinces cover the full cost of the expensive treatment, while others place financial burdens on patients. Globally, the situation is even more challenging, with the drug often out of reach for people in many low- and middle-income countries. These disparities highlight the urgent need for fair and equitable access to this life-saving medication and the development of new therapies for patients with rare variants not targeted by these new medications.
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La fibrose kystique les condamnait à une mort précoce. Plus maintenant. Radio-Canada. Signé par Ariane Labrèche. September 30, 2024.
Un miracle médical s’est produit. Mais pas pour eux. Radio-Canada. Signé par Ariane Labrèche. October 1, 2024.