Local resident Jenna White is asking for help from the government in order to make a new, life-changing cystic fibrosis (CF) medication readily available across Canada.
White has been a fierce advocate for CF patients since meeting her partner, Aaron Hart, who has struggled with the disease his entire life.
It is estimated that one in every 3,600 children born in Canada has CF. The disease mainly affects the digestive system and lungs, causing chronic infections and inflammation that leads to lung damage, as well as malnutrition due to the inability to absorb nutrients.
There is no cure for this genetic disease.and although its severity differs from person to person, it will eventually lead to death in the majority of people with CF.
But a new medication, not yet widely available in Canada, is offering hope.
Pharmaceutical company Vertex has developed Trikafta, which is capable of helping more than 90 per cent of CF patients. Not only does it increase patients’ lung capacity by a minimum of 14 per cent but it can expand life expectancy by at least nine years.
While Hart’s lungs are significantly scarred and the damage is irreparable, the drug can prevent further damage. The overall goal of the medication is to have it accessible to children before their disease gets to the point where it can’t be managed well.
“To watch your partner in declining health and know that this suffering is preventable is the most excruciating experience,” said White. “Years of his life are being stolen from him as we wait for access to medication.”
Although nearly 4,500 Canadians have CF, Canada is one of the only developed countries that doesn’t yet have the medication approved for all citizens.
“The fact that Vertex Pharmaceuticals has struck deals with countries around the world to provide cystic fibrosis patients with access to Trikafta proves they are willing to work with governments to ensure that patients are taken care of. It’s curious as to why our government isn’t doing the same,” said White.
The medication is currently allowed through the federal government’s Special Access Program, but Hart’s application was denied and others with the disease have died waiting to be accepted.
Only 122 people have been prescribed Trikafta across the country. According to a study conducted by a research team from Dalhousie University, the Hospital for Sick Children (SickKids) and St Michael’s Hospital, by 2030 Trikafta could reduce the number of people living with severe lung disease by 60 per cent and reduce the number of deaths by 15 per cent.
“This medication is literally a miracle drug and Canadians with CF deserve to be given this innovative medication,” said White. “It’s the closest thing to a cure since the disease had been discovered.”
Hart’s life is significantly impacted by the disease on a physical and emotional level every single day.
His daily routine involves an hour of hands-on therapy including both exercises and medication. Every year he is admitted to hospital for lung infections, sometimes at the speciality CF clinic at St. Mike’s in Toronto for up to a month.
“Aaron literally has constant lung infections, he is sick all the time,” said White. “It’s excruciating for us to know that there is a medication that can significantly reduce the amount of lung infections he contracts and that it’s available in our neighbouring countries but not here.”
Because of CF, he now also lives with liver disease and diabetes.
The COVID-19 pandemic has added even more stress and daily challenges as Hart has hardly seen anyone or left his apartment in six months.
“The response from our local members of government has been disappointing and has left us feeling lost as to where to {turn] next,” she said.
White said that she has contacted MP Scott Aitchison, MPP Norm Miller, Premier Doug Ford, and provincial health minister Christine Elliott and has received either minimal or no response.
White invites anyone who feels passionate about this cause to contact their MP (Scott Aitchison’s email is [email protected]).
Hart and White are part of advocacy group CF Get Loud Canada which also has a support community on Facebook here.
To follow Hart and White’s journey, visit their blog The Lung and the Short of It.
To learn more about CF and Trikafta, Cystic Fibrosis Canada’s Trikafta Can’t Wait page here: cysticfibrosis.ca.
Don’t miss out on Doppler!
Sign up here to receive our email digest with links to our most recent stories.
Local news in your inbox three times per week!
This is a heart-breaking story: I’m extremely saddened for both of you. Unfortunately, every possible avenue is seemingly closed to you.
Firstly, Vertex has not even submitted the product to Health Canada for testing and evaluation. This would seem to be a case of the FDA being wary of our stringent approval mechanisms. That would make me concerned about possible side effects (to which the FDA tends to turn a blind eye).
Secondly, the fact that you were rejected by the SAP is unsurprising; as they tend to favour acute over chronic cases.
Furthermore, if/when it is approved in Canada, to extrapolate from what you say; young children and babies will have a decided advantage.
Finally, as the drug was FDA-approved only last year; no generic versions will be possible in the U.S. until 2039.
I hate to be all gloom and doom. My feeling is that even before it becomes available in Canada; you start campaigning to reduce the proprietary period from 20 years. I’m on slippery ground here, because I think that Vertex’s 20 years would still apply in Canada. All I know is that Canada allows exceptions to that period.
Another choice is to work with Cystic Fibrosis Canada, who of course, are also hard at work lobbying for this drug. Of course, Vertex is the source problem. If they would only submit it to Health Canada; and in a total contrarian stance for Big Pharma; consider lowering the 20-year period, that would be ideal.
Everyone is all in for children; EXCEPT when money is involved.
I wish both of you all the very best.