Health Committee on Oct. 30th, 2018

[Erin Little]

This is where it comes down to the bureaucracy of business and Health Canada. It was basically because the new drug was approved. SAP is there for special access when there are no drugs currently available in the country to take.

Procysbi was approved in July 2017, and we went to get our next dose filled in November. That's when we got our letter of cancellation, strictly based on rules around drugs coming into the country and having exclusivity, because they went through the process. That is the reason they wanted to switch us to Procysbi.

The other thing with Procysbi is that the ingredients of the drug are the same as Cystagon. It's the same drug. The only difference between Cystagon, which Olivia is currently on at $15,000 a year, versus Procysbi, is that it's enteric coated. They enteric coat it so that you only have to take it every 12 hours versus every six hours.

I know when you have children, the dream is to sleep. I gave that up when I had kids to begin with, but at the end of the day, they call it a breakthrough drug even though the ingredients are the same. This drug would not even be on the market if it weren't for families like ours and others across Canada and the U.S. who were fundraising to make this drug. We funded this drug to happen to begin with, which is a real kick if you're somebody like us who just wants the best for your child.

This drug, although it has the same ingredients, is slowly released over 12 hours, so you only have to take it twice a day versus six times a day. But in the fine print that nobody else reads is the fact that, with this drug, I have to limit my eight-year-old, who already has a hard enough time eating food, because the medication, the same ingredient, makes her nauseous. On top of that, we have to limit her food intake for eight hours in a day, an eight-year-old. I bet most of you couldn't sit around this table and limit eight hours of your waking time...to schedule your life around taking just one medication.... Olivia is on six other pills that have to be taken, too, which we also have to stagger.

She's on sodium bicarbonate. You can't take sodium bicarbonate with any kind of slow release because the bicarbonate would dissolve that drug immediately in her stomach and she would get a double dose, because with this new breakthrough drug, Procysbi.... I lost my train of thought.

[Don Davies, NDP, Vancouver Kingsway, BC]

Ms. Little, can I come back to that? I want to just follow up with the same kind of question for Ms. Vowles.

I'm trying to get a handle on the price of eculizumab versus Soliris.

[Caryn Vowles]

They're the same drug.

[Mary Jane Vowles]

They're the same drug. They just have a different name.

[Don Davies, NDP, Vancouver Kingsway, BC]

Okay, so that is the same drug.

A 2015 article in The Globe and Mail said this:

Canadians with atypical hemolytic uremic syndrome (aHUS), a life-threatening genetic disease that damages vital organs and affects fewer than 100 people in the country, often require treatment with the drug Soliris at a hefty price tag of more than $700,000 a year. The cost spurred the federal Patented Medicines Price Review Board to call a public hearing into why the manufacturer, Alexion Pharmaceuticals, charges more in Canada than in other developed countries

Do you have any information to share with this committee on the outcome of that PMPRB hearing? Did they determine that the price of Soliris is excessive?

[Mary Jane Vowles]

I do not know.

[Don Davies, NDP, Vancouver Kingsway, BC]

Does it cost $700,000 per year?

[Mary Jane Vowles]

Yes, my understanding is it costs $750,000.

[Don Davies, NDP, Vancouver Kingsway, BC]

Ms. Little, I'll go back to you, and whenever you get your train of thought just pipe in.

[Erin Little]

Okay, yes.

[Don Davies, NDP, Vancouver Kingsway, BC]

You mentioned that your family is forced to reapply every three months to continue to receive the life-saving drug. In the past, your family had to apply every six months, and it was all but guaranteed you'd receive that approval, which is no longer the case.

Can you outline why that changed the application process that was imposed by Health Canada?

[Erin Little]

Prior to all the changes, I didn't even know they had to apply every six months; honestly, I thought it was once a year. I would call the pharmacy when we were headed down to the clinic and I would just say that Olivia needs a refill on her prescription and there were no questions asked. It would be there, and every three months we would get it refilled. When everything came into place with Procysbi, they just changed the way we have to apply to every three months. Why, exactly, we were never given a reason. There are speculated reasons around the issue that they have to be cautious because there is a marketed drug in Canada so they have to be cautious on how and who they give access to Cystagon to.

We have clinic in two weeks, and I messaged down because I'm extremely proactive, because this drug Cystagon now comes from the U.K., so I have to worry about it being imported on time, getting stuck in customs and so on. These are all things that I choose to take on as a parent and worry, because I take such great care of my child. There are other parents who are really whimsical about it, and that's fine, but they're in situations where they have run out of drug and then they have to look within our community to help them until they get their supply because they have to go through this process. The disease isn't going away in three months. I wish it would. My understanding is that it's just a business; they have to watch.

[The Chair, Liberal, Bill Casey]

Now we go to Mr. Grewal.

[Raj Grewal, Liberal, Brampton East, ON]

Thank you, Mr. Chair.

Thank you, witnesses, for sharing your stories.

As my colleagues have already said, I think everybody around the table can agree, across party lines, that when we hear more of the patient experience, it definitely gives us an opportunity to try to go back to the table and fix some of the—I hate to say it—more common sense things that need to be fixed in government bureaucracy.

Ms. Little, your story is one we hear too often, not just in the medical scenario but in a lot of scenarios in which the government makes things more complicated than they need to be when people are just trying to help their children or help vulnerable people in our communities.

I wanted to ask, along Mr. Davies' line of questioning, about the cost of the two drugs and the fact that this new drug has this time release formula in it and, because of that, it's become so much more expensive. You may not have the data, but how many people have this type of rare disease in Canada? I know it impacts children, mostly.

[Erin Little]

In Canada, there are between 75 and 100 patients.

[Raj Grewal, Liberal, Brampton East, ON]

There are 75 to 100. What about in the U.S.?

[Erin Little]

In the U.S. there are roughly 500.

[Raj Grewal, Liberal, Brampton East, ON]

We're looking at a very small number for any pharmaceutical to be developing a drug, because it's not going to be profitable, no matter how many patients there are, when you're playing with such a small pool.

The provincial government is subsidizing the full cost of the medication today. Is that correct?

[Erin Little]

In Ontario, yes.

[Raj Grewal, Liberal, Brampton East, ON]

Is that not the case across the country, though?

[Erin Little]

No, each province is different on who covers it and and how it's going to be covered. It's relatively new. They announced this within the past four to six weeks that it's going to be covered this way.

[Raj Grewal, Liberal, Brampton East, ON]

Okay, do most of the 75 patients live in Ontario?

[Erin Little]

No, we're spread out. There's one patient in Saskatchewan and a few in the Maritimes, and Quebec has the highest population of cystinosis; how many exactly, I'm not sure, but we do know that they have a large population there. I know, Dr. Midgely, who was here in front of you as well, has 18 patients. Our nephrologist has four of us in her clinic.

[Raj Grewal, Liberal, Brampton East, ON]

I don't like making assumptions, but I want to ask you this. Are you in contact with families that are going through the same thing in the U.S.? Do you have any comments on their experiences? Are some of the states doing a better job at this than we are?

[Erin Little]

Yes, in the States, where the biggest issue lies right now is.... Yes, they get it if they want it, if their insurance will cover it, right? Some patients pay $92,000 a month to be on this drug in the U.S. Yes, that's what it costs a month to be on this drug, but the insurance company covers it. But they have the choice. They have the choice to stay on one. In Canada, people will say to me.... The company that now owns Cystagon—it was bought recently by a new company from the U.K.—but they haven't applied. I can't be punished for somebody else's business decision, and that's exactly what is happening in our country.

This drug Cystagon has been around for 35 years and it is trusted. It works. It's an awful drug to want to be on. Some days I can't believe I fight for it. I should be fighting for a new drug. So that's the biggest thing.

In the States they have a choice and there are families for whom the thought of every 12 hours is appealing and they switch to this drug because the other fear was, what if we did want to switch Olivia? What if we said she's ready and we want to switch her?

Our fear is what happens if there are really bad side effects. It has happened in the States. Their white blood cystine levels go all over the place and it just causes an upheaval in life.

There are families in Ontario who have considered switching and they should have the right to switch as well. I'm not against it being here, but they're wondering what happens if they put their two-year-old daughter on this new drug and the side effects are so bad and they can't switch back. What if they don't let them switch back? That should not be something we have to worry about. We just want to keep our kids alive and healthy, and now we're worried because we want to try this new drug but we can't.