Provincial health authorities are determining whether to approve funding for a life-changing, state-of-the-art therapy for people with severe hemophilia A.
The alternative subcutaneous treatment is called Hemlibra.
“Right now, Hemlibra is a better therapy, hands down,” said Wendy Quinn, president of Hemophilia Saskatchewan. “It is a game-changer.
“We haven’t seen this type of change for bleeding disorders in 50 years. It is revolutionary.”
Hemophilia A is a rare inherited bleeding disorder caused by the deficiency of factor VIII.
For humans, the lack of factor VIII protein disrupts the coagulation cascade, resulting in blood clots forming slower, or not at all in hemophilia A patients.
Playwright and live scriber, Rick Waines, 54, is a severe hemophilia A patient and tainted-blood HIV survivor from Victoria, BC.
“This is the first time in my life where I haven’t had access to every treatment for my condition,” said Waines. “My whole life there hasn’t been much that is available, but whatever has been available I have had access to it.
“Now there is a treatment that exists in the world that isn’t an option for me.”
Hemlibra is for factor VIII deficiency, meaning that protein is missing from a hemophilia A patient.
“Hemlibra completely mimics factor VIII,” said Quinn. “It’s a puzzle piece that is like a bridge.”
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From birth until he was about seven, every time Waines would hemorrhage, which was about once or twice a week, he would have to go to emergency for a few hours and get infused with cryoprecipitate at the hospital.
By age eight he was able to administer his own intravenous (IV) treatments at home.
Advancements in hemophilia A treatments have improved over the years, but can be a burden on individuals as they are administered through an IV, or port for babies.
“I wouldn’t call it [intravenous therapy] something that is super comfortable or pleasant,” said Waines. “It’s finding veins and enduring that every other day.
“I would be relieved to not have to do it anymore. I also have some anxieties. While 54 isn’t ancient, as we get older our veins may not be as easy to find. Like what then?”
It’s rare for a developed country like Canada to not have Hemlibra as a treatment.
“It is absolutely asinine,” said Quinn. “We are supposedly the leader in bleeding disorders for the world. We founded the World Federation of Hemophilia.
“So, the fact we are one of the developed countries that don’t have Hemlibra is really embarrassing.”
Hemlibra was licensed by Health Canada back in 2018, under compassionate grounds for those with an inhibitor to factor VIII.
In 2019, Hemlibra was approved by Health Canada as safe and effective for all patients with hemophilia A, not just those with inhibitors.
“We don’t have a cost for Hemlibra,” said Quinn. “Factor VIII will always be lower than Hemlibra therefore nobody would get it.
“That’s the piece that we are fighting as we go forward. To change that cost model, and remove that criteria from their recommendation because then nobody would receive it.”
For Waines, Hemlibra would offer peace of mind as far as his old age.
“There are all kinds of things about that seem very attractive to me,” said Waines. “If I was infusing once a week subcutaneously I think that would be pretty cool to go three weeks without having that sort of psychological intrusion.
“My bleeding control would be better, my state of mind would be better and I would be assured when I am 78 and needing to control my bleeding that there would be a way to do that without being in a hospital.”
The Canadian Hemophilia Society created a letter-writing campaign to raise awareness of the importance of Hemlibra.
“Canadian Hemophilia society has asked we write letters to our MLA,” said Quinn. “We have had some good responses and are going to keep on doing it.
“We encourage anyone who wants to support the cause the write a letter by going to the Canadian Hemophilia Society website.”
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