Health Committee on Oct. 30th, 2018

[The Chair, Liberal, Bill Casey]

Go ahead. Make it very short.

[Mary Jane Vowles]

The expert is here in Canada. We've brought him here from Germany, and he is studying atypical HUS. The results are coming here, and Caryn is part of that research. It is related to some other illnesses as well, but he is here at SickKids working with a doctor at Toronto General and one at St. Mike's on a lower level.

Thank you.

[The Chair, Liberal, Bill Casey]

Thank you.

Mr. Davies.

[Don Davies, NDP, Vancouver Kingsway, BC]

Ms. Little and Ms. Vowles, do you happen to know if the research that was done behind the drugs that you're relying on was done in public universities or in Canada at all? Do you know where the research that formed the basis of these drugs was done?

[Mary Jane Vowles]

Do you mean the actual test sites, where they would have done the tests?

[Don Davies, NDP, Vancouver Kingsway, BC]

No, I mean the actual research.

It's my understanding that a lot of research on the chemistry and the molecules is publicly funded, often at universities, and the intellectual property is then often commercialized by the pharmaceutical industry, as opposed to the initial research being done by the pharmaceutical companies themselves.

Do you know where the basic research into the molecules that form the basis of the medication was done?

[Erin Little]

For us, for cystinosis, the research is being done in the States. We have doctors up here doing some research, new research, but everything has been funded directly by patients, because our rare disease population gets no public funding.

I can't think of it off the top of my head with Cystagon, but it was originally developed by a doctor, Jerry Schneider, in California. That's all I can answer to that.

[Don Davies, NDP, Vancouver Kingsway, BC]

Ms. Vowles, do you know?

[Caryn Vowles]

Initially, I think the research to do the actual drug was done by Alexion, but since then, research has been done at different hospitals. I don't think any has been done at any of our universities. Most is done outside of Canada, other than at the place my mom mentioned. Dr. Licht is the expert there.

[Don Davies, NDP, Vancouver Kingsway, BC]

Okay.

I have very little time left.

I want to end with you, Caryn.

I want to give you, as the patient and the person most affected by this policy, the last bit of time here to tell our committee what you think we should do. If you were the prime minister, and you could make a decision as to how we could change our system to help patients like you, what would be your recommendation and advice?

[Caryn Vowles]

I think you have to help people across Canada and not just in certain provinces. I know our board works at trying to help all the different provinces. Yes, we do it in Ontario and Quebec, and I think we're working on it elsewhere, but if a pharmacy comes in and works with only one province and offers one price to that province and a different price to another province.... If you try to do it across Canada, you will have more patients to go for, and you can drop the price that way, because you are now negotiating for more people.

[The Chair, Liberal, Bill Casey]

That's a good place to end.

I want to thank the witnesses for their testimony. It is very personal and very helpful.

I'm sitting here, Caryn, thinking that you must have not only good medication but also a good dose of persistence and determination by the sound of things. You just finished your biomedical engineering degree. You're working on your master's, and you plan to have a Ph.D. I'm sure we haven't heard the end of you. Thanks very much, and congratulations.

Thanks to all the witnesses.

We'll suspend now while we change witnesses.

We're bringing in the Department of Health officials, and I'm sure we're going to have some good questions for them.

[Mary Jane Vowles]

Thank you very much.

[The Chair, Liberal, Bill Casey]

Thank you.

[The Chair, Liberal, Bill Casey]

We will reconvene.

Before we start with our guests, I want to let everybody know that the unanimous consent motion for the correction of the premixed drink report will be tabled in the next few days.

As well, tomorrow, Mr. Roland Lescure from France, representing French residents overseas, will be visiting. He wants to talk to members of the committee about our role in the cannabis process. He's going to be available tomorrow at 3:15 p.m., for an informal chat, if anybody wants to participate.

We don't have a location yet. It might be in room 105 or room 107.

If anybody is interested in meeting with the gentleman from France about the role of the committee in the cannabis process, he wants to meet with us. I intend to meet with him. Anybody else who wants to come along can let me know.

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

What time was that again, 3:15?

[The Chair, Liberal, Bill Casey]

It's 3:15.

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

We have votes at three o'clock.

[The Chair, Liberal, Bill Casey]

Do we, tomorrow?

Well, we'll have to change it. It just came up and we just threw that time forward.

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

Maybe it could be right after the votes.

[The Chair, Liberal, Bill Casey]

If anybody is interested, let me know. We'll organize when and where.

We have members of the Department of Health here today. There are no opening statements, just questions.

We have Catherine Parker, director general of the biologics and genetic therapies directorate, health products and food branch; Karen Reynolds, executive director of the office of pharmaceuticals management strategies; and Dr. John Patrick Stewart, director general of the therapeutic products directorate.

We're going to start our questions with Mr. McKinnon.

[Ron McKinnon, Liberal, Coquitlam—Port Coquitlam, BC]

Thank you, Chair. I'll be splitting my time with Ms. Sidhu.

We've heard a lot of evidence certainly around rare diseases where there are trial drugs, and once they get approved, the price goes through the roof, or when another drug becomes an approved drug, the previous drugs that might be cheaper end up not being available anymore.

We're also hearing about the need for people on the special access program to reapply for coverage on a three-month basis.

I'll open it up to all of you to answer the questions, if you can.

What can the department do to streamline and facilitate access to these drugs for rare disorders?

[Catherine Parker, Director General, Biologics and Genetic Therapies Directorate, Health Products and Food Branch, Department of Health]

Dr. Stewart will answer that.

[Dr. John Patrick Stewart, Director General, Therapeutic Products Directorate, Department of Health]

A regulatory framework exists. We can't compel sponsors to come in and apply for market authorization. We can certainly encourage them. We can explain the process, facilitate, and provide incentives. However, ultimately it's a company's decision whether they market a product in Canada.

You probably heard over the last few testimonies that it is sometimes challenging to get products into market. We try to facilitate the conditions that would bring these products, through providing the framework for clinical trials, which supports clinical trials on rare diseases, and a process for priority reviews and notice of compliance with conditions, which I think we spoke about when we were here the last time, that incentivizes companies to come to market.

Once they're approved by Health Canada to be on the market, it's for other players within the system to decide on price and whether the provinces decide to ultimately fund it. It's beyond our role in that process. Maybe Karen can speak a bit about it.

As we talked about last time we were here, the special access program is one that is unique. It provides, on a probation basis, based on a request from a physician, access to an unapproved therapy that has not gone through the regular scrutiny for evidence around its quality, safety and efficacy. It's for the physician to explain why this therapy is the best choice for the particular patient in front of them, why it's a serious and life-threatening condition, why other available therapies, if they exist, have been considered and are not suitable, and evidence that supports its use.

Because special access requests are authorized in those kinds of conditions, they're not authorized for long periods of time. Typically, it's three to six months.

There can be a unique situation where a product receives an authorization, but it takes a company, once they get their approval, several months to get their labelling together and then actually put the product on the market. There's a period after a product gets market authorization where it's still not available, so the special access program will continue to provide access under that.

We shorten the time period because we know ultimately the product will be on the market. If there's an alternative product at the same time being accessed, we would shorten that as well, because we anticipate that most practitioners would transition to an approved therapy once it's available on the market.

[Karen Reynolds, Executive Director, Office of Pharmaceuticals Management Strategies, Department of Health]

I can speak a bit in terms of price.

As you're likely aware, the only federal lever that is able to exert any authority over price of patented medicines in Canada is the Patented Medicine Prices Review Board. I'm sure I answered a question related to the modernization effort from one of your colleagues when we were last here. They have authority under the Patent Act and its regulations to set what are termed “non-excessive prices” for patented medicines, and they do that based on the regulatory tools that are available to them.

That being said, as I'm sure you're also aware, Canada pays some of the highest prices in the world for patented medicines. We're third among the OECD countries, so it is acknowledged that prices of patented medicines in Canada are high.

The only other mechanism to bring prices down for Canadians is through negotiating mechanisms, largely through the pan-Canadian Pharmaceutical Alliance. That, as you're aware, negotiates prices for Canada's public drug plans. Prices would only be negotiated for those drugs that have received a positive recommendation for a formulary listing from the Canadian Agency for Drugs and Technologies in Health, better known as CADTH. That would not necessarily apply to all of the drugs that you're speaking about.