As reported in the Global News; what if you had a draining rare disease, and a life-changing treatment was out there but wasn’t available in your country?
At Dalhousie University in Canada, researchers have found that a new drug Trikafta, used to treat cystic fibrosis (CF), could dramatically change the quality of life for 90% of patients with CF. This is incredibly exciting for the CF community, as any patient group would feel when a medication comes that could dramatically improve their healthcare needs. The head researcher on the project, Santa Stanojevic, said how amazing it is to have a therapy that treats the underlying cause, the first therapy in history to do so for CF.
Cystic Fibrosis
Cystic fibrosis is a rare inherited disorder that causes a build up of a thick mucus. This is because the mucus disrupts the normal flow of the respiratory and digestive systems, causing damage. This progressive disease has an array of effects- as the clogged airways trap bacteria, causing infections, damage to the lungs and respiratory system, difficulty breathing, and absorbing nutrients. This then leads to patients experiencing coughing and wheezing, congestion, constipation, the inability to gain weight, difficulty exercising, difficulty breathing, and salty-tasting skin. There are a variety of treatment options that seek to prevent further infection and symptoms which include antibiotics, therapies, and various surgeries.
Canadians Want Trikafta
New research explored the impact of Trikafta entering the Canadian market. They studied the variation in patient’s health outcomes if they received the drug in 2021, 2025, 2030, or it never became accessible to the Canadian patient’s at all.
They found an array of improvements. Patients receiving the treatment showed a 60% reduction in people who suffered from severe lung diseases, although 18% were more likely to experience mild lung disease symptoms. Patients were also much less likely to have severe chest infections, with a 19% decrease in relevant hospitalizations. Most notably, the study found that patients could have their estimated survival rate improve by 9.2 years over 10 years.
These improvements could dramatically help the quality of life for many people with CF who often have extremely intensive treatment regimes to deal with their symptoms. In a conversation between Jeremie Saunder, host of the podcast Sickboy, and Floval News, he explained, he not only consumes around 40 pills on a daily basis, he also invests two hours into other remedies daily.
In contrast, Trikafka sounds like a miracle drug. Saunders said to the Global News that just reading one page about the drug brought him to tears. He couldn’t comprehend how just one pull could have such a big impact.
Canadians with CF
Currently, the drug’s manufacturer Vertex has not submitted an application to be made available, but a company representative had mentioned that Canada’s federal drug pricing reforms may be a cause. About 120 of the 5,000 some Canadians with CF have access to treatment via a medical route known as the Special Access Program. However, the majority of the patient’s are left feeling severely disappointed. For these Canadian patients, access would be life changing, and restrictions feel suffocating. They hope that the community will rally behind the drug and find access.
Saunders has talked about the impact this could have on parents with children affected by cystic fibrosis. He said if they could have access to the drug it could greatly administer the child’s life and give the parents some peace of mind.
