Health Committee on Oct. 4th, 2018

[Len Webber, Conservative, Calgary Confederation, AB]

Thank you.

Mr. McFadyen, you mentioned as well that you've had offers from pharmaceutical companies that do continue treatment for individuals on clinical trials. Have you heard of any pharmaceutical companies that do cut off their patients?

[Andrew McFadyen]

I've heard of pharmaceutical companies—and I have dealt very directly with pharmaceutical companies—that have levelled that threat. We've been able to work with them very collaboratively to ensure that our patients maintain access.

Part of the problem that the pharmaceutical companies are now facing, as I understand it after speaking with a whole host of them and a lot of my patients being on clinical trial drugs, is that the expectation now—even when there's a reimbursement deal in place for whatever drug has been approved—is that those patients who were part of the clinical trial aren't included in that reimbursement deal. Governments are still expecting companies to foot the bill for them.

For instance, there is a reimbursement deal in place for a rare disease drug that will be announced, I assume, sooner rather than later. The small details are that 20% of the Canadian population participated in the clinical trial. The reimbursement deal is in place, but governments still want the company to foot the bill. If you have 20% of your revenue stream being given away to those patients, even after that deal is made and they've been paying for them for the course of the last six years, it's really difficult for them to then start inching up into the profitable margins.

It's not the companies that I work with and deal with, and understand I'm very selective in the companies I deal with. I make sure that I am connecting only with companies that are in line with my moral values. They don't want to be removing patients by any stretch of the imagination. However, it does happen.

[Len Webber, Conservative, Calgary Confederation, AB]

Would you suggest a recommendation that any pharmaceutical company that wants to do a clinical trial has that moral obligation to continue after the fact and be forced to do that?

[Andrew McFadyen]

I do and I think it would be ideal, but once a reimbursement deal is in place with governments, I do feel that the burden should shift back over to the publicly funded health care system.

I do know that it is scaring companies away from wanting to do clinical trials here. That's really to the detriment, again, of our patients and our system.

[Len Webber, Conservative, Calgary Confederation, AB]

Doctor Midgley, you mentioned some stem cell research down in the States and Canadians' access to out-of-country treatment. Is it the case right now that Canadians cannot go down to the States to get this stem cell treatment? Can you perhaps elaborate on that, if you don't mind?

[Dr. Julian Midgley]

I'm not involved with any disease in the nephrology clinic with stem cell treatment currently. Some of the speculation was that this type of treatment, if it were successful, wouldn't be available in a Canadian centre. It may be, but one of the worries is that if you save a lot of money on a medication, it can't necessarily be transferred over to a different type of treatment coming from a different pot.

It's somewhat speculation.

[Len Webber, Conservative, Calgary Confederation, AB]

All right.

I don't know how much more time I have.

[The Chair, Liberal, Bill Casey]

You have another minute.

[Len Webber, Conservative, Calgary Confederation, AB]

All right.

All I'll say, Mr. McFadyen, is that I wish Isaac very well in his treatment in Italy. I just wish you very well and hope that it's successful.

Thank you.

[The Chair, Liberal, Bill Casey]

Next is Mr. Davies.

By the way, thank you very much for chairing.

[Don Davies, NDP, Vancouver Kingsway, BC]

Mr. Chair, welcome back; and thank you to all the witnesses for the excellent testimony.

Dr. Coyle, I want to start with you. This committee spent the better part of two years looking at universal, public, single-payer pharmacare. That was our recommendation. One of the central issues in constructing such a system would be the formulary and how it would be constructed.

Some of the difficulties of that are who makes the decision about what drugs ultimately get listed in a formulary. There are pros and cons of everything, but in my view, you don't want politicians making the determination about what gets covered, because that subjects politicians to pressure that may or may not be appropriate. You don't want industry to be unduly pressuring to make listing decisions.

In my mind, I came to the conclusion that what you really want is an independent evidence-based panel of experts, broadly speaking, who are making listing decisions based on efficacy and value for dollar.

That gets to the nub of the question. When you have a drug that costs, say, $500,000 a year and has a very small population that may benefit from it and the efficacy is unknown or perhaps marginal, yet that drug is very critically important to that small patient group who may have little other hope or few other options, how do we make those determinations?

[Dr. Doug Coyle]

It's really the crux of the question, isn't it? It comes down to what we want from our health care system. It also comes down to what we, as a society, feel we need to do for specific populations or problems facing us. I've come today and said that my idea is that maybe what we're trying to get from the health care system is to maximize the overall health of the Canadian population. That's what I think the objective should be, to maximize.

If there are other issues that you want to deal with, we need to deal with that outside of the health care budget, in a sense. Otherwise, funding a drug for a rare disease that costs $500,000 per year is going to take money from the health care budget and is not going to provide as much health benefit as would be provided to other individuals through other interventions, and therefore the net population health is going to decline. That's just a fact. We can argue about that, but that's just numbers, and math doesn't lie.

The issue then is whether there is something that we actually value differently about patients with rare diseases. Is there something that we want to create a special fund for or a special process for? That is for you guys to decide, not for me. That's a process issue or a values issue.

All I can say is that society has been asked, many times, to try to trade off different aspects of populations to see what they think they should put more money towards. The research shows that rarity isn't something that society has valued. Now, U.S. politicians might decide that this is not what we, as Canada, should do, and I applaud you for that. If you let the world decide what we could do, we'd still have the death penalty in Canada, but we think that's wrong. We might want to think about what society's values are. We know what's happened down south when we trust society to express their values. Maybe you want to have something special or different here. That's a decision that politicians have to make; I'm sorry. You would have to say that we need to set aside this amount of money to have a separate process for marginalized populations or for populations that we care about, but take it out of the health care system, because all you're doing by keeping it within the health care system is making decisions that don't lead to what the health care system should be doing, which is maximizing the health of Canadians.

[Don Davies, NDP, Vancouver Kingsway, BC]

Thank you.

Mr. McFadyen, I want to turn to you. You have obviously spent a lot of time thinking about this and are very personally affected.

You must have thought about that question, but as someone very personally affected, what advice would you give this committee about how we determine how we fund rare diseases when the efficacy may not be as—

[Andrew McFadyen]

If I listen to Dr. Coyle, I hear him saying that we value a society that will provide an exponential amount of money for people who make themselves sick over the course of their lifetime by smoking, by drinking. There's no question that you get lung cancer; we're treating it and we're putting all of those vast resources into it, but we're leaving behind those who have simply been born with a condition, and there's nothing else they can do, just as I was born with brown eyes or brown hair.

I like to believe that I live in a country where we protect everybody in need; where we protect the most vulnerable, where we have social safety nets in place that are meant to look after everybody who's sick.

In terms of looking at drugs that may or may not show appropriate efficacy, we also have to look at how we're evaluating what that efficacy is. When I sat with the minister of health one day, he said, “Listen, big deal: Billy can walk an extra 750 metres on a six-minute walk test. Why is he worth $500,000 for me?” I was able to express to that minister that it's not just an extra 750 metres; it's walking from his house to his car; it's walking from his car to his, now, place of work, if he can do it. At school, he's able to go from the classroom to the bathroom on his own, without an EA. He's able to fit more into society and become the type of person that the rest of the health care system values, and the value for dollar is actually there.

When we look at value for dollar as well, what we're not doing is looking at the overall ramifications on the health care system. We look at it as a $500,000-a-year treatment. What we're not looking at is parents leaving the workforce, mental health requirements for those parents who are dealing with these devastating consequences of these diseases, mental health of the siblings, or the amount of school time missed for siblings and families. None of that equates into that $500,000 a year; this is the value on these lives.

I feel we need to be able to look after everybody, and there are mechanisms in place to do that. If the efficacy of a drug is a little bit low or the data isn't available, we can look at funding on an interim basis and collect real-world data. Is it doing what the manufacturers say it does? Is it doing what the clinical trials are saying it does? I believe that's a system that can be put in place. I don't believe it will be a burden on the system and I feel that the overall health of all Canadians—not most Canadians—will be impacted and improved.

[Don Davies, NDP, Vancouver Kingsway, BC]

Well said, Mr. McFadyen, but I'll put a devil's advocate question to you. Surely we can't fund every drug for every condition, no matter what the efficacy or the cost. Are you saying we don't have to make any decisions in that respect?

[Andrew McFadyen]

I'm saying we should be able to look at those most critically in need—those patients, like my group of patients, who will be dead before we decide to fund it. I guarantee you, we will decide to fund this drug in this country. Most drugs that are granted priority approval move on to be reimbursed, or they move on to put a collaborative deal in place between governments and pharmaceutical companies.

What we shouldn't be doing is taking our time to make that happen, because we're losing a lot of people. If we have a drug that is granted priority review, it's granted priority review for a reason. We should be able to make sure there are mechanisms in place to reimburse that.

[The Chair, Liberal, Bill Casey]

Thank you very much.

Mr. Grewal is next.

[Raj Grewal, Brampton East, Lib.]

Thank you, Mr. Chair.

Thank you to all the witnesses for coming today. This is a very interesting discussion in terms of the role of government in this policy area. There are some very difficult decisions to be made.

I was on the finance committee for three years. Everything was dollars and cents. As a corporate lawyer, the bottom line always rang true in my work. At the same time, though, on the side of immigrants, we relied on social safety nets from time to time as I was growing up. Without those programs, I might not be a member of Parliament today.

As I'm hearing this testimony, I'm thinking to myself that I have this tremendous education and I'm a member of Parliament today, and I'm looking at it from the perspective of what's in the best interests of the nation. At the same time, when I hear Isaac's story, I'm thinking about my own family's story. My heart goes out to your family. Our prayers are with Isaac, and we wish him all the best. It's such a remarkable story that your family is going through.

My question is to Dr. Coyle. Does any jurisdiction get this right? In Canada we have a unique health care administration between the federal government and the provincial government. Is there any example of a jurisdiction, let's say in the G7 or the G20, that gets it right on rare diseases?

[Dr. Doug Coyle]

The fundamental problem with almost every health care system is that they actually haven't defined their objective. Unless we know what the health care system is trying to achieve, we can't address whether or not it has achieved that objective well.

The only health care system that does is Australia. Australia actually has a specific statement that the objective of their health care system is to maximize the health of Australians. If that's what they're trying to do, then they also have a process that makes those decisions, especially with respect to drugs, specifically based on whether this funding will increase the overall health of Australians.

However, even in Australia, we have the problem that we focus mainly on new interventions for which there is a commercial sponsor. That's where the pressure on funding comes in. Things that we used to cover, we no longer cover. In Ontario, for example, we don't cover physiotherapy appointments.

I have a chronic degenerative hip condition, so I go to see my physiotherapist every three or four weeks. It's horribly painful and difficult, but it actually helps. It stops me from requiring a hip replacement, which would cost the system thousands of dollars and probably mean that in 10 or 15 years, I'd be in a wheelchair and my quality of life would be pretty poor.

I can afford to pay for that physiotherapy out of my health insurance from my university. There are many Canadians, those from an immigrant background, who can't afford that basic health care, which is not very expensive. You're talking less than a thousand dollars per year. It would save money down the line in terms of the need for surgery, and it would greatly improve the quality of life of the individual.

I could say that no one does it right, because no one really considers health care as a whole. There are many things we can do to greatly improve the health of the population but that are just not funded anymore because there's no advocacy group. There's no commercial sponsor pushing for that coverage.

The easy answer is no. No one does it right. I'd say Australia goes the furthest by at least defining what they want from their health care system. In Canada it would be the first step that we could actually take; we could make a decision about why we have the health care system in the first place and what the underlying objective is that we're trying to achieve.

[Raj Grewal]

Thank you.

A lot of Canadians don't know individuals with rare diseases. I'm a new member of this committee. Funding for rare diseases isn't something that comes up on a daily basis in my constituency office, or in the calls I receive, in emails or on social media.

However, that doesn't mean it's not important. You get involved in politics to help make a difference. At a certain point, it's usually about protecting the most vulnerable people in society. The vast majority of us do well on our own and never need a member of Parliament's services. My riding of Brampton East has 130,000 people. I interact with maybe 5,000 of them over a four-year period, and that's an aggressive mandate.

What's the solution? Even my colleague from the NDP said that we can't possibly fund everything. Is there a solution to be found between the public sector and the private sector, maybe on a tax incentive basis or a research and development basis?

We have vulnerable patients in this country who need access to these drugs. These drugs are ready, FDA-approved and in the pipeline for Health Canada. Maybe they're stuck at the pricing stage, which must be beyond frustrating. Where are the small fixes we can make in the bureaucracy to make this thing a bit quicker?

I'll go to Mr. McFadyen, if you don't mind.

[Andrew McFadyen]

Well, for example, for these patients with cystinosis, the system is being forced into providing and reimbursing a drug that may cost $400,000. There's one in the pipeline with Health Canada, about to be approved, which will probably cost $50,000. As Dr. Midgley said, it does virtually exactly the same thing. You take four pills a day as opposed to two. We are no longer allowing our patients to gain access to that $50,000 or $60,000 drug. In fact, the cost of that drug at the moment is $10,000. There are savings right there, if you have a hundred patients across the spectrum.

Are there ways we can ensure that we have more collaboration between pharmaceutical companies and governments? You bet there are. With pCPA, we've pitched some really unique pathways that could help. For instance, there are certain ultra-rare populations of under 10 individuals: Alexion has some patients who require Kanuma, and BioMarin has patients with CLN2 or Batten disease. If one of these drugs is given priority review at Health Canada, the rare disease company would then foot the bill until pCPA negotiations are done, so long as there is an agreement in place within 90 days.

Pharmaceutical companies want to make this happen. Right now, it's a “We get everything or we get nothing” situation. Either the governments are happy because they've brought the price of pharmaceuticals down or the company is happy because they scored a big deal. The only people missing out here are patients.

It's a challenge, yes, but I'm sure there's a solution we can come up with, or our economists can come up with. I guarantee you that if we had what we've seen around the world, such as mining disasters in Chile where there are 21 people stuck down in a cave, we'd spend a hundred million dollars to go in and get them, because it's the right thing to do. An economist could sit there saying, “If we spend that hundred million dollars to go down and get those people, we're not able to buy a CT scan for this group of individuals over here, so let's not do it. Let's let those people die. Let's put the news cameras and news media on it as they waste away”—

[Raj Grewal]

I don't agree with that analogy from a public policy perspective.

[The Chair, Liberal, Bill Casey]

I'm sorry; your time is over.

That completes our seven-minute round. We're going to go to a five-minute round.

It's 10 o'clock. I understand Dr. Midgley has to leave at 10. If you have to depart, thanks very much for your contribution. We appreciate it very much.

We'll go to Mr. Lobb.

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

My first question is for Dr. Campbell.

If I have this wrong, please correct me, but in your opening comments you mentioned that you had worked with some pharmaceutical companies. One of the things that is obvious to me—but I want to get your perspective if you're able to give it—is that with these drugs there are so few people who are actually going to take the drug or benefit from it that the pharmaceutical companies say, “If we can do it through the SAP program, we will.”

To me, if it's been on the SAP program for 20 years, we should have a system whereby we make it very easy for these pharmaceutical companies to move it into a registered drug. Do you have any comments or thoughts on this idea—that we should do less through SAP and have more registration?

[Dr. Craig Campbell]

There's no doubt, I think, that this program, the SAP, can fill gaps a bit better than perhaps is happening right now. For really ultra-rare drugs.... I mean, let's face it: Canada is a small country with a wide geography, and sometimes converting an SAP access into an actual ongoing open-label clinical trial.... That's another option: to convert patients not just into an approved drug in the country but to actually enrol them in the longer term in a clinical trial.

It's not that patients who are enrolled in an open-label extension trial always must have participated in the initial randomized control trial or experimental trial, so rather than necessarily using the apparatus to push through a drug to its commercial realization, one could imagine other exit strategies out of the SAP.

Of course, ultimately, as you say, getting the drug approved in a formal process would be ideal. Where the SAP could be better, in my opinion, as I mentioned in my talk, is in trying to fill the gap between Health Canada approval and the pCPA process. I think we need to shine some real light on whether that's an access pathway in the short term during evidence review.

Those are my thoughts around the longer term. Let's move people into a clinical trial setting and gather data from them, or perhaps move it into a marketed product.