It's $300,000—
Evidence of meeting #119 for Health in the 42nd Parliament, 1st Session. (The original version is on Parliament’s site, as are the minutes.) The winning word was procysbi.
A recording is available from Parliament.
Evidence of meeting #119 for Health in the 42nd Parliament, 1st Session. (The original version is on Parliament’s site, as are the minutes.) The winning word was procysbi.
A recording is available from Parliament.
9:35 a.m.
President, Liv-A-Little Foundation
Yes.
It's roughly calculated from the price that we had to search tirelessly for. That is what Olivia's dosage would come to.
9:35 a.m.
Conservative
Ben Lobb Conservative Huron—Bruce, ON
Okay.
Mary Jane, does the province pay for the drugs that Caryn takes now or do you have insurance?
9:35 a.m.
Board Member, Canada, atypical Hemolytic Uremic Syndrome Canada
Caryn is currently under Alexion.
She started in grade 8. When she started on the drug, she was part of a child project with two kids from SickKids and six children from other countries. When Alexion went back to giving her the drug compassionately, it was based on my fighting with them over a promise they had made that they would continue her on the drug. They have her on that now, but she could be dropped at any time, just as they dropped her the other times.
9:35 a.m.
Conservative
Ben Lobb Conservative Huron—Bruce, ON
Are you aware of any other people in the country taking this drug who are covered, or is it all under the compassionate label?
9:35 a.m.
Board Member, atypical Hemolytic Uremic Syndrome Canada
Some are covered by the Ontario government. They have a whole bunch of outlined rules, and if you don't meet certain criteria, they take you off the coverage. One of the main criteria problems is that after a transplant they can cut coverage after, I think, six months or something, and if someone's illness were to reoccur, they would lose the transplant.
9:40 a.m.
Board Member, Canada, atypical Hemolytic Uremic Syndrome Canada
The illness reoccurs, and whenever the illness occurs, it's death. You're always on death's doorstep as it reoccurs.
9:40 a.m.
Conservative
Ben Lobb Conservative Huron—Bruce, ON
Erin, for Olivia, how many pills per day does she have to take to maintain a quality of life?
9:40 a.m.
President, Liv-A-Little Foundation
She is probably at 50 pills a day to maintain her health.
I want to add, if I may backtrack—sorry—to what the issue is here as we talk about pharmaceutical companies coming here, and as Mr. McKinnon said. You asked about the cost. The pharmaceutical companies are two steps ahead of us. The other downfall is that when it comes to these rare disease markets and there are only 75 patients to sell something to, I wouldn't start a business to sell something to 75 people.
What they'll do is they'll come in under SAP as well. The pharma companies want to offer that compassionate usage of their products until they are approved so that we have something rather than nothing, but then, in all the fine print, they say that once a patient comes under their compassionate care, they remain there. Even when it's approved, the insurance companies won't pick up the cost because the company has already agreed. If a company comes in and offers our daughter a new product and, say, 35 people go on SAP, they don't have many patients to continue to build a business offer.
I think it's important to add to that, because it's another really big issue with pharma companies wanting to work with us as well as trying to build a business, make a profit and potentially hopefully continue to give back to rare disease communities.
9:40 a.m.
Board Member, atypical Hemolytic Uremic Syndrome Canada
I'm down a lot, but I do hemodialysis at home every day.
9:40 a.m.
Board Member, Canada, atypical Hemolytic Uremic Syndrome Canada
Caryn's pills are for high blood pressure. The actual eculizumab is every two weeks. There's an infusion of eculizumab every two weeks.
9:40 a.m.
Conservative
Ben Lobb Conservative Huron—Bruce, ON
I think it's important to highlight the fact that, as I'm learning as the process goes, if not for the parent advocacy for their child, the future is almost certain for the children as they are impacted by this. Maybe both parents could talk briefly about your own personal commitment—I'm not saying it's a sacrifice—to your children to make sure.... As for some of the sacrifice, maybe that's meant your own career that you had in mind.
9:40 a.m.
President, Liv-A-Little Foundation
I'm grateful that we're here today. I know that having patients come here and speak is relatively new to this kind of situation. I guess I really never knew what advocacy meant until I started in the role of doing it. It takes time away from my children, but I'm grateful to be here. I'm grateful that you guys are willing to listen to a different perspective that's not a medical or educational one. It's more about experience. At the end of the day for all this stuff, we're the ones who are impacted, yet nobody understands the full impact.
We came from Port Elgin. It's a trip to get here. We had to leave our kids and entrust them to somebody else, which I'm okay with, but at the end of the day.... I couldn't be more proud to do it, but we shouldn't have to do it. There's not enough listening to the patients.
I understand. If you took one patient from each rare disease, you would have thousands of people and you couldn't listen to all those people, but it's about listening to the people who are actually affected, not just the players. I have no skin in the game. I'm here away from my family. I am actually losing to be here today.
I think it's important to find that voice of the patient population and integrate that with what we're doing with Health Canada and policies and procedures in making a system that works for us. I know that it can't be perfect for everybody, but it has to work in a better way than it is today.
9:40 a.m.
Board Member, Canada, atypical Hemolytic Uremic Syndrome Canada
I've been a single mom since Caryn was three. I had to keep my job; it wasn't a choice. Caryn is one of three children.
All those months she spent in hospital, I was there with her every night. I left work. I drove to SickKids. I would get up in the morning at six o'clock and head back to work. I did that for months on end.
That affected my boys. I found out more about that this weekend than ever before. They talked about the take-home meals they used to endure. We were referring to Little Caesars. They had buckets of pasta. I haven't had buckets of pasta in years, since she's been on this. But they remember that. They remember the Toonie Tuesday, because that's what they got on Tuesdays. I love cooking, but I wasn't able to do that when my boys were young.
I know this illness gave me a lot of strength that I didn't know I had. Part of the illness over Caryn's years was that there was no clear-cut treatment, no clear-cut answer. There were points when there were things happening where....
For instance, there was one apheresis machine in Toronto, and it got wheeled from Toronto General to SickKids in the underground tunnel. It could do two treatments a day. They decided after they'd started Caryn on it, and she was six months.... Maybe it was the first one they'd ever done this way. It was very scary, although I didn't know enough to be afraid. They wheeled it back. They did it for five days in a row. Then they decided that another patient needed it, and she wasn't going to get the machine. I was going to have to talk to the media and show my blue-eyed, blonde-haired little girl. In the end, the machine came back to Caryn. I have no idea what happened to the other patients who were supposed to be on it.
Through that, we, together.... When I say we, the doctors and I worked together on a plan that was agreeable to both of us in stretching out the treatments on the apheresis machine and trying whatever together. Together, we created research through this illness.
I don't mean to sound smug or smart ass, but I was the one who linked the nephrologist at SickKids to Dr. Kaplan at the Children's Hospital of Philadelphia at the time, so they could find out more about the research. All there was about Caryn's illness back when she was six months old was one chapter in a textbook, which they gave me to read. It was way over my head, but I learned to understand it. We've come a long way since then.
Certainly, lots of treatments are working, and the doctors know a whole lot more than they did when progressing through her illness. When she was diagnosed back in the day—I remember we could go on the Internet—there was one other case, and it was of another blue-eyed, blonde-haired little girl, but she did not survive the illness, the aHUS, the way Caryn did. Most kids, as much as they survived, were left vegetables. We've moved beyond that.
9:45 a.m.
NDP
Don Davies NDP Vancouver Kingsway, BC
Thank you, Mr. Chair.
First, the voice you're bringing to this committee is every bit as valid as what we hear from the medical side, and I want to commend you on the information and the education you're bringing to this committee. I think you probably know more about the system and about the conditions you're dealing with than many people in the health care system. I want to thank you for being here and sharing your story. It takes a lot of courage and sacrifice, as you've said.
I want to start by trying to understand the finances here.
Ms. Little, how much does Cystagon cost per year?
9:45 a.m.
President, Liv-A-Little Foundation
For our daughter, we're just under $15,000 a year.
9:45 a.m.
President, Liv-A-Little Foundation
Her dose would switch to potentially $300,000, because it's a cost per pill.
9:45 a.m.
NDP
Don Davies NDP Vancouver Kingsway, BC
Yet if I understand your experience, Cystagon was working very well for your daughter, but the system wanted to switch her from Cystagon to Procysbi at many times the cost. Do I understand that correctly?
9:45 a.m.
NDP
Don Davies NDP Vancouver Kingsway, BC
Did anybody from Health Canada or the system explain to you why they wanted to move your daughter from a drug that was costing $15,000 a year and was working to one that you didn't know whether it would work and would cost $300,000 a year?
