Health Committee on Oct. 4th, 2018

[Ben Lobb, Conservative, Huron—Bruce, ON]

Okay. Thanks.

Mr. McFadyen, we had our Health Canada officials here. They paint a very rosy picture of the landscape and all the great things they claim to have done, but we see the example of the patients who have cystinosis. They get one drug through this program, the SAP, for many years, and it works. Another pharmaceutical company comes along, sees a tremendous opportunity to make a lot of money, gets their drug registered in Canada, and then Health Canada starts to force patients onto that drug at a higher cost.

I know they say that it's one in a million that this is the case, but I know that can't be true. That's not the complete issue with rare diseases, etc., but why can't we go to the drug company that has been providing that drug for 22 years and just ask what we have to do to get this into the country so that we don't have to force people off it? Why can't we say to them that for these rare and ultra-rare ones, we will make it worth it for them to register and not go through...? In the last meeting we had with Durhane, she said that there are 13,000 applications in Canada per year through the SAP.

[Andrew McFadyen]

Yes. In that particular case of cystinosis, I can speak confidently about it, because once that drug from the other company—the more expensive drug—became approved and was working its way through the negotiation process, I had a lot of cystinosis patients reach out to me. They said that they didn't want to lose access to the cheaper drug, that it worked for them and they didn't want to have to move over, and they asked what I could do.

I actually met with Health Canada. I met with the special access program director and said that I felt there was a unique role I could play in this, and that I would like to visit the company that was making that cheaper drug and do what I could to see if they would apply to Health Canada for standing, and then they could go through the reimbursement negotiations as well. That has taken place.

I met with that company and said that if they believed their drug was of value for patients in need, if they were a company that believed patients come first, would they please step forward and apply to Health Canada. They've done that. It's working its way through the system. At some point, whether they get a priority review or not, we should see that second drug approved. Then we'll have a very expensive drug on the market and one that's not as expensive, both of them doing the same thing.

[Ben Lobb, Conservative, Huron—Bruce, ON]

I know that there—

[The Chair, Liberal, Bill Casey]

I'm sorry, Mr. Lobb. Now we'll go to Ms. Sidhu.

[Sonia Sidhu, Liberal, Brampton South, ON]

Thank you, Chair, and thanks to all of you for your testimony.

One in 12 Canadians, two-thirds of them children, are affected by rare diseases. What strategy can Canada implement in the short term so we can fix that problem instead of going through the planning for a long-term solution?

Anybody can take that.

[Dr. Doug Coyle]

To start with, I'd say that the figure of one in 12 Canadians having a rare disease emphasizes maybe the wrong message that the Canadian organization for rare diseases is giving. It seems to imply that rarity isn't very rare and that therefore the impact of making decisions to fund drugs for rare disease which don't increase the overall health of the population might be much bigger than we actually think it is.

Part of the problem there is, what is the definition of “rare”? When we think of rare, we think of diseases for maybe one in 100,000 Canadians, or one in 50,000 Canadians or even one in a million Canadians. Part of the problem is that CORD uses a definition of rarity which I think is maybe one in 20,000—I can't remember—but it's not what people think of as rare.

We really need to realize that rarity is not a binomial issue. It's not “your disease is rare” or “your disease is not rare”. There are different levels of rarity. I can very much understand that a lot of us are thinking about those ultra-rare diseases—those one in 100,000 and one in 200,000 individuals—and that's where I think Canadians might think that there's a value in having some separate process for those ultra-rare conditions.

I think the definition that CORD uses in throwing around those figures like one in 12 Canadians having a rare disease—or one in 10 Canadians, they've even said—is really, really unhelpful to this debate, because we're not really talking about having a disease for one in 2,000 or one in 5,000 Canadians. We're really talking about those ultra-rare conditions that maybe 100 Canadians have or that 50 Canadians or even 10 Canadians have. That's the dialogue that I think we're supposed to be having here, and it's not the idea of the one in 12 figure, which really is unhelpful.

To be honest, if one in 12 Canadians has a rare disease, then we can't treat rarity as special, because almost everybody has a rare disease in those contexts. I think this definition that CORD uses is really counterproductive to this argument.

If we can focus on those ultra-rare conditions, then maybe we might think that Canadian values should reflect some special process or some special funding envelope for that, but that's for you individuals to decide. Clearly, if you've left it up to the general public, the general public doesn't think we should have that special process or special funding envelope.

[Sonia Sidhu, Liberal, Brampton South, ON]

Thank you.

Mr. McFadyen, you said the bioethics innovative group in the United States makes quick decisions. Was that type of group created here in Canada?

[Andrew McFadyen]

The bioethics group? No, we exist in the United States. There is a panel of about nine of us led by Dr. Art Caplan, who is probably one of the foremost medical bioethicists on the planet.

We take action when needed. For instance, if there's a grey area in terms of decision-making and a company can't decide whether they should be providing expanded access for a patient, they don't make that decision: it comes to us. We hash it out based on our experiences, and then we send that recommendation back to the company. A hundred per cent of the time they take action and follow up on what we do.

I have pitched this in places like the pCPA to say that there should be that independent ethical panel that can work out disagreements between a government side and a pharmaceutical side. It may not be binding, but at least a recommendation will come forward, and it will be part of those negotiations so that what we don't see—and we see it often—is that negotiations for these rare diseases drugs come to a standstill and nothing happens. If this type of bioethics panel were to take place, we could at least advance some innovative solutions for them so that the process can continue to move forward.

[Sonia Sidhu, Liberal, Brampton South, ON]

Thank you.

My next question is for you, Dr. Campbell, on off-label use of medications. How do we measure the efficacy of those medications?

[Dr. Craig Campbell]

In my mind, there is no doubt that we need to make sure that people who stand to gain the most from a medication get access to it. However, you're right: in the real world, things often start to spread out, such that other patient groups or patients who are maybe not the focal point of the treatment benefit will get these drugs.

I think that's where I would say that investing in rare disease registries will be very informative and is a systematic way to approach this, so that we can follow patients over time who are getting access to treatments and who are perhaps using off-label treatments. It's a little bit different from drug-based registries. I think investing in these kinds of infrastructure is really critical.

If you don't mind my making a short comment on Dr. Coyle's initial suggestion, I think using cumulative figures around describing rare disease is really an effort to do two things.

One is to demonstrate that when you put together all rare disease, in fact it's not that uncommon. We spend a lot of health care resources—extraordinary health care resources, sometimes—on some very common things like heart disease, stroke, and cancers. I think that in the rare disease space we want to get some skin in the game here, so describing it in that terminology helps people to recognize that it's a big problem.

The second thing it does is show that solutions that crosscut all rare diseases are needed. Yes, you may have an ultra-rare disease versus something that's one in 20,000, but there are deficits in our way of approaching that in the health care system and in the evidence review. If we actually develop effective paradigms, it would be useful across all these rare diseases.

[The Chair, Liberal, Bill Casey]

That concludes our session with our witnesses.

We have to go into committee business now. We have quite a bit of business to do before the week off.

I want to thank the witnesses very much for contributing to our study, and also for the good work you do, and your commitment. It's obvious that you really care and contribute every day.

With that, I want to suspend for five minutes while we go in camera, and we'll return.

[Proceedings continue in camera]