Cystic Fibrosis Drug Rejected in Canada

According to a story from the Toronto Sun, the Canadian Agency for Drug and Technologies in Health (CADTH) recently announced that it would not be recommending the funding of the cystic fibrosis drug Orkambi for public use. This is discouraging news for cystic fibrosis patients. A total of 42 doctors that specialize in treating cystic fibrosis wrote to the agency to encourage the drug’s approval, but to no avail.

About Cystic Fibrosis

Cystic fibrosis is a type of genetic disorder which can have impacts throughout the body, but it is most characterized by the build up of abnormally thick, sticky mucus in the lungs. This mucus becomes a fertile breeding ground and habitat for potentially infectious bacteria. Many patients must take antibiotics for much of their lives. This disorder is caused by mutations of the CFTR gene. Symptoms of cystic fibrosis include progressive decline in lung function, lung and sinus infections, coughing up mucus, fatty stool, poor growth, infertility in males, clubbed digits, and digestive problems. Treatment includes antibiotics and medications or procedures intended to maintain lung function. Lung transplant is an option when lung function declines severely. Life expectancy ranges into the 40s and 50s with good care. To learn more about cystic fibrosis, click here.

A Disappointing Decision

The announcement also means that Orkambi will not proceed to the halls of the Pan Canadian Pharmaceutical Alliance (pCPA), where the negotiation of the drug’s price would have begun. The exact reasoning for CADTH’s decision is not entirely clear, although Orkambi has attracted some criticism in the past for its extreme price and, in the eyes of some regulatory bodies, only marginal effectiveness. The drug is only effective for patients with a specific mutation.

Last Ditch Effort

However, they is still a sliver of hope that Orkambi could eventually get approved. The chair of the Canadian Cystic Fibrosis Treatment Society, Chris MacLeod plans to reach out to Health Minister Christine Elliott. Chris has cystic fibrosis himself and understands the urgency for many patients, and Christine has a history of sticking up for cystic fibrosis patients in the past.

Firstly, CADTH has basically confessed that it lacks the data and capacity to make clear decisions about rare disease medications, so Chris is calling for a new panel of experts to issue a report to the Minister in 100 days with their own findings about Orkambi. In addition, he says that the Minister should begin negotiations with the drug company regardless of CADTH’s decision.


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