Healthy Living

New, Promising Cystic Fibrosis Drug Needs Funding

New, Promising Cystic Fibrosis Drug Needs Funding

Canada has the highest rate of cystic fibrosis in the entire world, with approximately 1 in 134 citizens suffering from the disease. However, a recent drug that has been found to be "life changing" for some cystic fibrosis patients is not being funded.

Official response

Health Minister Adrian Dix officially stated that because of Orkambi's cost and efficacy, the Common Drug Review has recommended that it not be eligible for funding. This news comes as a devastating blow for many cystic fibrosis patients who have found Orkambi to be life altering, including Melissa Verleg from Vernon.

The manufacturers of Orkambi, Vertex Pharmaceuticals, have stated that they certainly have sufficient evidence for proof that the medicine works. Vertex's spokesperson Megan Goulart has stated that the drug indubitably performs well for patients who possess a specific type of cystic fibrosis. They claim that they have more than enough research to back up their statements, but that hasn't changed the mind of Canada's drug review agency.

Fighting back

Megan Goulart recently spoke out for the company, saying, "we share (Verleg's) and the whole cystic fibrosis community's frustration with the failure of the Canadian government to provide access to Orkambi." Verleg is just one of the many patients who are devastated by the news that they will no longer have access to the drug that has been helping them so tremendously.

Goulart also mentioned that Health Canada had approved the drug for patients in January of 2016. After this, Orkambi was sent to the Common Drug Review in order to hopefully be approved for eligibility for public reimbursement. However, as we now know, this big was not successful.

The evidence they have to prove that the drug was indeed successful, allegedly, is abundant. Goulart has stated that at the beginning of the review, Vertex put forth the results they had accumulated from two sets of six-month studies. In these studies, half of the patients involved were administered a placebo while the others received Orkambi. At the conclusion of the test, all of the patients involved in the study received Orkambi, where the effects going forward were also recorded. Goulart goes on to say that Vertex would like to resubmit more data that has been taken since, but the Common Drug Review is refusing to accept it, due to the face that the long-term study involves each and every participant taking Orkambi, whereas the review board will only take into consideration data that was acquired from placebo-based studies.

However, Goulart does not believe that placebo-based studies are the acceptable route at this moment, as Vertex has already established the drug's effectiveness. Due to the fact that patients are seeing benefits, they want all patients to be able to access the drug instead of submitting half to placebos where they cannot receive the advantages the drug offers. She explains, "once you know a medicine works, you can't not give it. It's not ethical to withhold it from patients."

Efficacy of Orkambi

Goulart explains that Orkambi is best suited for people who have a specific type of cystic fibrosis. Although cystic fibrosis patients do all fall under the same disease, the types vary dramatically. That's why it is important for each patient to have a personal approach to their medication, as certain drugs will work for some cystic fibrosis patients but not others. For example, Vertex also manufacturers a different product for cystic fibrosis, but it is focused on patients who have a different type of the disease. Because the two drugs are different, between both of them, approximately a third of patients are able to notice marked improvement, whereas the number would be fewer with one drug or the other. When a patient finds a drug that works for their specific type of cystic fibrosis, the results tend to be very intense.

Melisa Verleg is one such example of patients who experience dramatic differences in their disease once they find the correct drug, as Goulart explains when she states, "for (people like Melissa) it's had a significant impact on her life." That's why the recent decision from the review board is so heartbreaking.

Impact of the ruling

Goulart explains that it's a tragedy that public funding for the drug is being held up by procedural technicalities because in the meantime patients that could be benefitted from the drug are suffering. She explains, "we are at a road block. We are really trying hard to work with the provinces. The national recommendation is not binding- the provincial governments, whether together or individually, can decide if they want to negotiate with us directly."

High price

Some wonder why Orkambi doesn't simply make the drug more affordable, so the public funding would not be entirely necessary. Goulart stated that the high price is necessary, as billions were spent on its creation and further billions will be put into further research to produce more cystic fibrosis medications so more patients can be helped. Specifically, about 80 percent of the entire company revenue is invested into new research. Goulart defends this by saying, "it's about reinvesting so we can continue our progress to help more people with the disease."

How this ruling impacts Melissa Verleg, and hundreds like her

Unfortunately, due to the recent decision, Melissa Verleg will only be able to use the nine days she has left of her medication. After she finishes it, her doctor stated that she will have approximately a week before she will have to deal with the symptoms she used to have, once again.

Verleg has said that she is absolutely terrified. Life has just recently become normal to her again because of starting Orkambi, she explains, "I am able to be a mother to my children again, and a wife to my husband. I can cook, I can clean my house for the first time in four years. I can go and play with my children. I used to be a spectator in life watching it happen. Now I am involved in life, and not on the sidelines."

This would be a positive story, had she been allowed continued access to the drug. Unfortunately, her health insurance changed its benefits program that resulted in a $20,000 cap on annual spending. Unfortunately, Orkambi costs more than that- per month.

She has tried to make her case to her insurance company, but they still refused to grant her access to the drug. Now, she is appealing to the government, saying, "We pay into our private insurance, we pay our MSP premiums. We pay our taxes. We have a universal health care system and it needs to be universal. Price should be a non-issue."

For many like Melissa Verleg, starting Orkambi felt like a miracle when she could finally have the life she was used to back. Now, this miracle is being cruelly ripped away from her. If insurance will not cover life-changing and saving medications, then the government must be pushed to enable the health of its citizens. Verleg hopes that Health Minister Adrian Dix will reopen negotiations with Vertex, which would impact the lives of so many in Canada suffering as Verleg is.