Health Committee on Sept. 27th, 2018

[Catherine Parker]

Thank you for your question.

It is correct that we are not seeing that every medication which goes to the U.S. or Europe is coming to Canada at the same time.

There are two issues: first of all, getting the medication here, and also getting it at the same time as those other countries. We are seeing some significant improvements in that. There used to be long lag times between filing to the U.S. and to Europe and then Canada as the third country of filing, but it would sometimes be a very long lag period. We are seeing significant improvements in that, and we're seeing that many submission filings are concurrent or overlapping.

That is our first priority, that—

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

What is the reason for this improvement? What is it that makes some cases improve, while others do not? What makes success not fully offset failure? Why doesn't this work for all drugs? Why are we not at the same level as all other developed countries?

[Catherine Parker]

We are in dialogue with the pharmaceutical industry as to why there is still a difference in times of filing. They give us a number of reasons.

They find it difficult sometimes to work with more than one regulator at the same time, so we are trying through international co-operation and work sharing to minimize that obstacle. We are actually encouraging through a number of our projects that companies can file to Canada and another regulator at the same time, and we will work share on those submissions so that they are basically only dealing with one party.

There are other factors, of course. Unfortunately, it is a business decision for manufacturers, the whole drug marketing, so we are doing as much as we can to encourage that information.

We are internationally harmonized with respect to the requirements for drug approval so that companies can file one dossier that is applicable to all regulators. Also, we have what we call a common portal with the U.S. FDA so companies can now file simultaneously for approval to Canada and the U.S. through one common filing portal. That has improved the situation.

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

I accept your answer, but it doesn't reassure me about short-term improvement.

I put myself in the shoes of Canadians with a rare disease who tell their MP and government that they can't get treatment in Canada, that they must go to the United States or Europe. They must seek hope of being treated outside of Canada. But we can't accept this. If Canadians are going to be treated outside Canada, it is because we haven't been able to provide them with the service they need.

I understand that an effort is being made to reduce the gaps, but what is your plan to completely eliminate this gap, despite the economic pitfalls and marketing difficulties of pharmaceutical companies? Canada must become a leader and be among the first in the world.

[Catherine Parker]

Yes.

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

Having said that, I don't have a plan, I haven't found one, but you aren't reassuring me, at least not today. I would like you to reassure me. You have one minute left to do so.

[Catherine Parker]

Did you want to add...?

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

Mr. Stewart.

[Dr. John Patrick Stewart]

I have one thing to add. Our regulations do not give

authority to compel a sponsor to come to Canada. We cannot compel a company to market a product in Canada, so we work to encourage them to come. We work to provide incentives at the level of clinical trials. We work to ensure that Canadians get access if there are rare disease trials going on. Canada punches, I think, above its weight from the point of view of academia and research centres, so we try to get these trials in so Canadians get access at the development stage and therefore we have evidence on Canadian patients when the submission comes in.

At the end of the day, we cannot make a company come to Canada. One of the realities is the population in Canada is not the same as the population of the U.S. or in the EU, so some of the decisions of these larger companies when they come in in their sequencing is the size of the population.

As Cathy pointed out, we are trying to work internationally, work sharing, so that companies can come in and get an approval for more than one country with one application. We're hoping this will be an incentive for companies to look to Canada and our partners we're work sharing with to come sooner.

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

Thank you.

[The Chair, Liberal, Bill Casey]

That completes our questioning for this panel.

You've completed your chore here today and we thank you very much. We only scratched the surface of this very complicated issue, but we do appreciate your sharing your information with us and your expertise.

[Catherine Parker]

Thank you very much.

[The Chair, Liberal, Bill Casey]

Thank you very much.

We're going to suspend for a minute while we change panels.

[The Chair, Liberal, Bill Casey]

We will now begin with our second panel.

Today we have Tammy Moore, chief executive officer from the ALS Society of Canada. From the Canadian Organization for Rare Disorders, we have Maureen Smith, Board Secretary, and we welcome back Durhane Wong-Rieger, President and Chief Executive Officer.

Welcome. We're looking forward to your testimony.

I'm not sure who's going to start. Do you have an order?

[Dr. Durhane Wong-Rieger, President and Chief Executive Officer, Canadian Organization for Rare Disorders]

We'll go first.

[The Chair, Liberal, Bill Casey]

Perfect.

[Dr. Durhane Wong-Rieger]

First of all, a huge thanks to the committee for having this very special panel and certainly, for inviting us again. We are really pleased that you're undertaking, for the first time, a focused look at Canadians with rare disorders. This is a huge asset to us.

I especially want to thank Ben Lobb—who I hope is somewhere around here—and also ex officio member John Oliver for their leadership within this caucus to make this study happen.

To those of you who are health professionals here, and I know some of you are, we also would like to build on what you've already heard from your constituencies and your patients as well around the first-hand impact of rare diseases.

Certainly, we're very pleased with the way you've asked the question. What are the challenges and barriers, but also, what are some of the solutions? I think that's what we all really want to get to.

We very much appreciated hearing the Health Canada presentation as well. We'll add a little bit to what you've heard and to some of the very cogent questions that you folks raised with that.

This is, in fact, as you have seriously addressed here, a long-neglected part of Canada's health care system. I think, as you've already heard, and I don't need to reiterate, rare diseases affect nearly three million Canadians directly, and that's not including family members. Two-thirds of those are children. Most of these diseases are disabling, and that's why they're so important. They're lifelong conditions, and many of them are life-threatening unless we diagnose them and can intervene quickly.

The good news, of course, is that we are doing a much better job in terms of diagnosis. There are, in fact, many more treatments coming. We just did another survey this past June, and we will be able to break down the results for you to look at, but about 80% of people with rare diseases who responded to our survey were clear. They have challenges getting access to the medicines they need. I think Maureen, who appeared with me last time, is going to be talking with you about her challenges of getting diagnosed and treated for her rare condition.

At CORD, we've been working for many years to address these challenges. One of the things that we did in the context of this was the launch in 2015 of Canada's rare disease strategy, which we were very happy to do here at Parliament in order to have the recognition of the members of Parliament in support of that launch.

One of the key goals—I'm not expecting you to remember, but I will reiterate for you—is, in fact, access to promising therapies. We're very pleased to be here today to talk to you about some of the challenges that patients face in accessing those therapies and about some of our recommendations, especially how the federal government can help improve access.

Before I get started, I will turn it over to Maureen Smith. Not only is Maureen a patient, but she has also been for many years the secretary of the board for CORD. She's going to share her story.

[Maureen Smith, Board Secretary, Canadian Organization for Rare Disorders]

Mr. Chair, committee members, thank you for inviting me to appear before you today.

As Ms. Wong-Rieger said, my name is Maureen Smith, and I am a Canadian with a rare disease. It was diagnosed when I was eight years old, after four years of tests and visits to specialists.

For the past five decades, access to treatment and drugs has played a key role in my life.

Over the past 10 years, as a volunteer with the Canadian Organization for Rare Disorders and many other groups and advisory committees, I have heard from many Canadians with rare diseases. I have also been able to learn about the difficulties faced by other stakeholders.

I am delighted to share with you my perspective as a patient.

I thought long and hard about having this opportunity and giving you one message, if I could. I'm going to give that to you.

When you have a rare disease, you face medical uncertainty, and often there's nothing that can be done about that. In my own case, I was on the first Canadian clinical trial—four children with an ultra-rare disease. I was the only girl. You live with that medical uncertainty your entire life. Often, even with the best interest, there's nothing that can be done about that.

When you add the burden of uncertainty of access to treatments, that just adds another layer to your life. If you're fortunate enough to have a treatment, then you constantly worry about whether you're going to be able to access that treatment. If you do get it, then you worry about it being taken away.

I wanted to share that with you. That is one of the most difficult things for patients to deal with. A lot of medical uncertainty can't be dealt with, but I'm hoping that you will try to lessen that burden on Canadians who have rare diseases, and their families.

[The Chair, Liberal, Bill Casey]

I just want to caution you that in your opening remarks you were quite long. You only have about five minutes left to go.

[Dr. Durhane Wong-Rieger]

Let me pass over some of it. From listening to your questioning before, I understand that this committee is actually quite cogent with some of the initial challenges, so I won't go through all of them with you. I will highlight a few.

We do endorse what Health Canada is doing right now in terms of an orphan drug regulatory approach. They brought it together, but we do reiterate that having an orphan drug regulatory framework would actually bring some additional supports and advantages for bringing those therapies into Canada. That is a very important consideration still, but as I say, we do endorse what they're doing now.

The big challenge, as I think you have heard from Cathy as well, is that having them approved does not mean access, and a lot of the access is in fact in the reimbursement system. Certainly, we think that aligning the review process between Health Canada now and the health technology assessment agencies could reduce the timing. Quite frankly, what we are very concerned about is the health technology assessment process that we use in Canada by CADTH and by INESSS is not appropriate for rare diseases.

What we do know is that the majority of those drugs which go through that process are in fact.... Originally, they were being denied. Now they're denied because they're not deemed cost-effective. They do not meet the standards of cost-effectiveness that have been set for more common drugs. I think you have already indicated that you can understand why there are so many challenges in terms of drug pricing, the uncertainty, and the long-term benefits of those drugs. Those are the two parameters that have CADTH and INESSS coming back with not only negative recommendations but also recommendations of a discount, sometimes as much as 97% of the price, in order for them to be approved.

It isn't a matter that they've taken a few more months in getting there, as Dr. Stewart says. It's a matter of two years. It's a matter of four years for them to negotiate that price. That's a tragedy. That's a time when, for patients with life-threatening, debilitating disorders, either their diseases get worse or, in many cases, they get to the point where the drug is no longer working for them because they've progressed too far, or they die. This is not good.

We've asked patients what's worse, not having any drug, or having a drug that you can't get access to. You can imagine what the answer is. Sometimes it's worse to not be able to get access to a drug that could in fact be beneficial to you, and that's the case.

We do want to make sure that there is more put into the assessment of these drugs, but we need a separate HTA pathway, like the U.K., France and what Australia has just come out with, and to recognize that we need to do that differently. Again, we think the federal government can help support that tremendously.

We do want to highlight the fact that, as you've heard, the PMPRB price and regulations are in fact.... We have no doubt about it, because companies have talked to us about it. It will have a devastating effect in terms of bringing in those drugs in a timely way, as we've talked about. We will not have them available to us. We will not be a first-tier country anymore. We would definitely not get the clinical trials in, because no company is going to come to Canada where the pricing is set in such a way that it's different from every other country, and where they are not going to be able to launch that drug early. We know that in most cases, once the patients are on a clinical trial, you don't take them back off after their drug has been approved. Now companies are looking at two to four years during which they are going to pay for the patient. I don't think so. And they are not going to want to come to a country where the prices are set in such a way that they are going to be at a disadvantage. That's not to say we don't negotiate new prices. We can't negotiate in a way that's different from other countries.

We do want the federal government to reconsider what's happening in terms of those PMPRBs. We support wholeheartedly lower drug prices. We support wholeheartedly the necessity of making sure that Canada does not pay more than other countries. However, we cannot do it in such a way that it's different from other countries. That's going to actively discourage companies from coming in.

Sorry, I'm making this up as I go along now.

[The Chair, Liberal, Bill Casey]

You're doing great.

[Dr. Durhane Wong-Rieger]

I do want to speak to the special access program that was brought up. I think this is a huge problem for us here. Quite frankly, as Dr. Stewart said, we had 13,000 requests and only 48 rejections. Really? Should we have a system in which we have 13,000 requests through SAP? Is this the way to run access? I don't think so.

We need to consider how we can bring many more of those drugs into the approval process. I think the pathway that Health Canada has put forth, the modernization of the regulations, could in fact encourage that, but I can tell you—and this is not the time to tell you—in great detail why the companies hesitate and why, even after they now apply, there are such barriers in terms of the approval and reimbursement process that a company looks up and says, "I don't think so." That's a problem. I think this is a challenge for us.

Definitely, as my final comment, we would like to propose a national pharmacare program in which the federal government actually can take a huge role. It was promised to us initially in 2006 when the Liberal government was in place. We actually had a proposal put in front of us from the federal-provincial-territorial committee around a program for expenses for rare diseases. In 2012, 2014 and 2015 we've had continual promises that this would be happening. We've yet to see anything materialize, even though we advocate on their doorstep at almost every health minister's meeting.

This is something we would like to see as part of this national pharmacare that's coming forth. We've had great discussions with Dr. Hoskins, and they have encouraged it. We need to have a program that is going to be publicly owned. It could have a private component to it if they choose to, but patients should have the opportunity to sign up for a public program funded by federal and provincial governments that's going to be one program, one set of guidelines, one approval process, one set of databases in which we can enrol patients and provide drugs in a managed fashion that would allow patients to get access to drugs as they are brought in.

If we have a parallel process with HTA and Health Canada, great. At the time of NOC we should be able to open up this program. The federal government, we believe, needs to take a huge role in doing that and we can bring the SAP into it. There's no reason that SAP drugs should not be adjudicated as part and parcel of that. You have a clinical panel. You have experts. You have databases. We can actually manage an entire rare disease program, and it will put us at number one. It will be something. Nobody has an ideal program. We think Canada is in a good position to actually step up to that spot.

[The Chair, Liberal, Bill Casey]

I don't want to miss a word you have to say, but I have to cut you off. We're a little over time.

[Dr. Durhane Wong-Rieger]

I have finished. That's great.

Thank you very much, again.

[The Chair, Liberal, Bill Casey]

We do appreciate your comments, and we look forward to more when the questioning starts.

Now we go to Tammy Moore from the ALS Society.