Health Committee on Oct. 30th, 2018

[Raj Grewal]

That's a fair recommendation.

My last question is for both Ms. Vowles and Ms. Little.

What's the one recommendation you would like to see the government follow to make your lives easier?

[Mary Jane Vowles]

Our personal life or the life of all aHUS patients?

[Raj Grewal]

Either or both, to be honest.

[Mary Jane Vowles]

I would like to see the drug funded so that at this point in life I don't have to worry. Caryn can have a transplant. She could get the missing part of her life back in place, and not have to worry that the drug company or the Province of Ontario will cut it out after three months or whatever.

We have a history of every two weeks; that's all we can handle. That's all her body has ever been able to handle, no matter what, and I would like her to have the security to go on and be a productive member of the research world, where she seems to be headed, and be able to do great things for all of you.

[Erin Little]

For us in this case specifically, and there's a lot that I don't know, but I'm also trying to raise and home school two children and take care of a sick one. There's a lot that I wish I understood more about, but from what I do see and have felt the direct effect of, with drug companies coming into Ontario, something needs to change. We need to make sure we're two steps ahead of the pharmaceutical companies that want to come here, because they're always two steps ahead of us. That is something we need to be extremely cautious about. I'm so grateful to live in Ontario where we do have coverage. If we were forced to switch, we could switch to this high-priced drug and our daughter would still be on it.

The other piece is that when we welcome these companies to come to our country, they need to provide more to us, especially when their price tags are so high. They should be building business here. We shouldn't be paying for these high-priced drugs and then all of their business is going to other countries. They need to give back to the rare disease community as well. They should be advancing...again, we need pharmaceutical companies for advancements for our kids. But it's actually embarrassing that these companies come. They don't have to produce their drugs here. They don't have to employ people here, and even in our situation with Horizon pharmaceuticals, our potential patient support is in the United States. They don't even understand how each province is different.

There's that part. We need to have higher standards for the companies that come in, and we need to make the process more cohesive. The fact that a drug was approved in June 2017, and the potential treatment was taken away from us at that time, and there was lots of back-pedalling and trying to fix things, and then the price was just approved, and how we were going to cover it, over a year later.... It's embarrassing actually.

[The Chair, Liberal, Bill Casey]

Okay, thanks very much.

Now we have time only for one question, one question and one question.

We're going to start with Ms. Gladu, for five minutes.

[Marilyn Gladu, Conservative, Sarnia—Lambton, ON]

Thank you, Mr. Chair.

Thank you to the witnesses for sharing their stories. I'm going to summarize what I think I heard.

First of all, it's ridiculous that you have to apply to the special access program every three months for a drug that's prescribed by a doctor for a lifelong condition. That has to be fixed.

Second, it's ridiculous that CADTH is approving a drug that is 20 times the price and is the same exact chemical. Yes, you get something for technology of slow release, but being able to prevent you from continuing on a drug that was working I think is ridiculous as well. That's something that should be addressed in the recommendations.

Then, I think there is the question of funding and how we are going to be able to afford this. I agree that people need these drugs to live, but if I do a little quick calculation for the 27 life expectancy years for cystinosis and the more expensive drug, that would be $810 million. For the drug for aHUS, that would be $4.5 billion, if we think that people could live 30 years.

That is a huge amount of money, and as we see more and more solutions for rare diseases, we're going to have to come up with a way to fund these.

Would you agree that those are things we need to address?

[Mary Jane Vowles]

For sure.

[Marilyn Gladu, Conservative, Sarnia—Lambton, ON]

Very good.

Does the Horizon price have something to do with the recent renegotiation of NAFTA? Was this the company that was involved there and wanted to extend the coverage? I think they used to get 10 years of coverage on the patent and now they have lowered it to eight years. Is that Horizon, or is that a different company?

[Erin Little]

I can't say.

[Marilyn Gladu, Conservative, Sarnia—Lambton, ON]

You don't know. All right.

The last thing I want to ask is about the PMPRB process.

I think it was you, Mary Jane, who talked about the PMPRB when Soliris was being approved there was something they did that was not good. Can you tell me again what it was that they did that wasn't good and what we should do to fix that?

[Mary Jane Vowles]

What happened was that it was approved by the federal government, by Health Canada, and then when it went to the provinces, it was decided by CADTH that it wouldn't be approved because it was too costly and wasn't effective.

All the research said the drug worked. That was why Health Canada approved it. Why do we have two levels of government where one is undermining what the other said? If it's effective and Health Canada has approved it, how come a province can decide that no, it's not effective? Clearly all the research supports that it is.

[The Chair, Liberal, Bill Casey]

Mr. Ayoub.

[Ramez Ayoub, Liberal, Thérèse-De Blainville, QC]

Thank you, Mr. Chair.

Ms. Little, thank you for your testimony. Let me reassure you. Earlier, you said that you took time away from your family to come and testify. I imagine it's the same for you, Ms. Vowles. This is time well spent, because it is very important for the committee to hear from families and to know what they are going through on a daily basis.

My questions are mainly for Ms. Little because I have had less time to research the Vowles family.

Ms. Little, while consulting your foundation's website, I saw that some of your supporters are pharmaceutical companies. It seems to me that you are the one funding the research to find a drug or a cure; that's what you said. How do you see your relationship with these companies that are both supporters and suppliers? Isn't there some contradiction between these two aspects?

[Erin Little]

That's interesting. We don't take pharma money to run any part of our organization.

We host a golf tournament to fund research, and for our tournament in 2017, Horizon pharmaceuticals was a sponsor of ours. This was prior to the issue and what I have learned over the past year. This year they did reach out to us and wanted to sponsor our event, and I did not reply because I will not take their money.

I don't know where you read that we do take pharma's money because—

[Ramez Ayoub, Liberal, Thérèse-De Blainville, QC]

No, I haven't read that anywhere; I just read on your website that you have supporters. I saw the list of pharmaceutical companies. I was just wondering what your relationship was with them.

[Erin Little]

On the Liv-A-Little website?

[Ramez Ayoub, Liberal, Thérèse-De Blainville, QC]

Yes.

[Erin Little]

I'm not even aware that it must read that way. Thank you.

[Ramez Ayoub, Liberal, Thérèse-De Blainville, QC]

The fact is that this is about seeking funding for drug development. I may not have understood what you said, but I had the impression that your feeling was that you were responsible for part of this research on a certain drug or cure, in particular. Is that correct?

[Erin Little]

Yes. Horizon Pharma purchased what was Raptor Pharmaceutical. It was Raptor Pharmaceutical within the U.S. with the Cystinosis Research Foundation, which I'm a part of.

The family organization directly funded Raptor to bring Procysbi to the market, and Raptor then sold that piece to Horizon Pharma.

That whole research and development that was done was brought forward because of Raptor Pharmaceutical, which was then sold.

[Ramez Ayoub, Liberal, Thérèse-De Blainville, QC]

Do you want to make a comment, Ms. Vowles? No? Okay.

We are talking about rare diseases. There are few people affected in Canada, but a little more in the United States. How do you view research competition within the G7 countries, particularly with respect to large countries developing drugs?

There is competition between these countries, but the treatment and accessibility are not the same. In a country the size of Canada, there are several cases. As for you, Ms. Little, we're talking about 75 or 100 patients. How do we manage, on a global scale, to offer a service and do this research in such a way that it is profitable not for companies but for patients?

[Erin Little]

It's interesting because what we're finding, actually, when we do start to research rare diseases is that it is unfolding and is involved in helping other diseases. There's a drug library that is progressing.

Let's say that they take Cystagon and they see if it can help other drugs. So, within these rare diseases.... I understand that it would be really hard for these companies to invest a lot of money specifically into cystinosis, but when you can nail down a disease with the exact genetic mutation, it gives a whole new understanding to the body, to what's going on and to how we can actually apply it to other diseases.

That's another world that needs to stop being exclusive to themselves, too, and that needs to share more so that we can grow together and heal multiple diseases. It is possible, and I know that within our own disease community, with regard to cystinosis, it's helping with Huntington's disease. There's another disease that I can't think of. Within just one small community of 2,000 worldwide, you can potentially help, so if that other disease group is another 15,000, it can slowly spread and multiply.

[The Chair, Liberal, Bill Casey]

Thanks very much.

Now we're going to Mr. Davies for three minutes.

[Mary Jane Vowles]

May I answer that question? Is that possible? I can make it very short.