Health Committee on Sept. 27th, 2018

[Tammy Moore, Chief Executive Officer, Amyotrophic Lateral Sclerosis Society of Canada]

Mr. Chairman and honourable members of the Standing Committee on Health, thank you for the invitation to appear before you today.

We appreciate this opportunity to address barriers to access to therapy. It is a reality our community finally has the opportunity to deal with, but it is a growing risk we see for the future. We were pleased to hear about some of the advances Health Canada is making, but I'm also going to share with you today an illustration of some of the issues that even in our limited experience we have seen give us further concern for the future.

I am here today representing Canadians affected by ALS. I wanted to invite Carol Skinner, a young woman and strong advocate from the ALS community, to join us here today. In fact, many of you have met her in the past, but she has to send her regrets because to be part of today's proceedings exactly highlights some of the issues associated with rare diseases like ALS.

Carol has a terminal illness. It's robbing her of her mobility, her ability, and her independence. To be here today, Carol would have needed her personal support worker to come in extra early to attend to the daily routines that most of us take for granted, such as, brushing our teeth, washing our face, getting our clothes on, tasks that would further be challenged by the fact that this meeting is happening early in the day and her muscles do not respond as readily as they might later in the day.

Carol's husband, Travis, would have needed to take the day off work to physically support her in attending resulting in lost wages in a family already burdened by the heavy cost of a costly disease. Carol's reality underscores that ALS is not just a terminal neurodegenerative diagnosis for 3,000 Canadians who are currently living with the disease, but it's a disease that impacts many more people. The emotional, physical and financial costs are devastating.

Each year, 1,000 more Canadians will be diagnosed and they and their loved ones will find themselves on a journey with the disease that in the course of two to five years will take away their ability to eat, speak, move and eventually to breathe as their motor neurons die.

Only 5% to 10% of people who are diagnosed have a hereditary link, and in those families, the disease devastates in each and every generation. We each in this room have a one in 400 chance in the course of our lives of having an ALS diagnosis.

The current situation in which there is almost nothing in the way of therapeutic options leads to an urgent desperation as people consider any options that will provide them hope, including those that put them at greater risk and financially drain their resources and challenge a health care system that has to pick up if something goes wrong.

With this context, for the remainder of my remarks, I ask that you consider what it would be like to be paralyzed by ALS. Please try sitting still and not moving a muscle until I'm done speaking.

We have several recommendations that we would like you to consider in access to therapies.

One is to create an environment that makes Canada a country of choice for new therapies throughout the spectrum from research and development, through clinical trials, through new drug submissions, through approval process and reimbursement. Another is to coordinate, streamline and increase transparency associated with those processes and timelines that enable patients to access drugs both before and after market access is granted. We also recommend that you please work with the provinces and territories to address the inconsistencies that currently result in the inequitable access across our country.

Our recommendations are grounded in the following principles: equity, timeliness of access, affordability and patient partnership. Every Canadian should have equitable and consistent access to high-quality treatments that are appropriate to their individual needs.

In regard to timeliness, Canadians should have access to the treatments they need in a timely manner. My population doesn't have time to wait. In terms of affordability, they should be able to afford both the treatment and the means to administer the treatment. Patient partnerships must be meaningful. They must be thoughtful and they must make a difference. Above all, we have to have transparency in the process.

As a member of the Canadian Organization for Rare Disorders, we broadly support the call for extensive stakeholder engagement and a strategy for the management of rare diseases. These recommendations and guiding principles are a result of the experiences we've recently had in our community and our engagement with organizations like CORD and the Health Charities Coalition of Canada, HCCC.

I would like to share with you some specific barriers recently experienced by Canadians living with ALS.

Some of you have met Norm. His situation demonstrates the lack of a streamlined process in clinical trials and pre-market access, which results in physical, emotional and system costs. Norm participated in a clinical trial in which there were no adverse events. Once the clinical trial concluded, the company was willing to provide the drug to him, but an application had to be made to Health Canada for an open-label extension. The process of approval took six weeks.

While this may not sound like a long time for any one of us, this could mean the difference in terms of the ability to speak or to move your hands. Unfortunately for Norm, during this gap in treatment his disease progressed with a loss of function. It directly resulted in two significant falls. The resulting injuries required hospitalization, including epidurals, to deal with the pain from the back injury.

Finally, after having to act as an active liaison between the two different decision-makers that were not directly communicating, Norm was granted an open-label extension. When safety of the therapy is not a concern, this type of delay and regulatory inefficiency is not acceptable. It creates uncertainty and unfairly and unnecessarily impedes the access to therapies.

In comparison, in the U.S., when the FDA approves a clinical trial, as long as there is no safety signal, that open-label extension is immediately available through the conclusion of the clinical trial protocol. This process avoids a potential gap in treatment, like the one Norm experienced that caused injury and loss of function. In Norm's case, that function cannot be regained.

Right now we are also seeing lengthy timelines and a lack of transparency in Canada's regulatory and reimbursement process, both of which affect patient access. In May 2017 the FDA approved Radicava as a treatment for ALS in the United States. After considerable lobbying of the company by patients, by ALS Canada, and yes, even by Health Canada, eventually, in March of 2018—it took almost an entire year—the company decided to put their drug through the regulatory process in Canada. Currently that drug is under priority review. Given the 180-day timeline, we expect that the decision is imminent.

In those 17 months since the FDA's approval, those in our community who could afford to do so have utilized Health Canada's personal importation process. They have paid out-of-pocket to import the drug. This method of access is not in the spirit of equitable access within a universal health care system, and it has put people at risk.

We are also very aware that even if this drug is given a notice of compliance, or an NOC, with conditions, it will not mean that the treatment is readily available, as we heard earlier today. We expect that CADTH may provide reimbursement recommendations to the provinces and territories by the year's end and that it will be sometime after that before actual decisions are made. But with no defined time frame and no transparency in the process, companies may find the lack of clarity not worth the business risk to consider a Canadian marketplace, which means that Canadians will not have access to new therapies.

In the 180 days during Health Canada's priority review period, 500 Canadians have died of ALS. How many will die awaiting the CADTH decision? After that, how many will have to die while they're awaiting the availability through a publicly funded drug program? We are dealing with a community that measures time by loss of their own function and by the number of members who will die during this process.

Access issues do not end even once the drug is in hand. It also means creating a system where patients can have the drug administered equitably, regardless of where they live or their financial means. Even though many in our population were able to access this drug through their own initiative, many face challenges with getting it infused. This drug requires administration through an IV. It's typically 10 days out of 14, followed by 14 days without the drug, and then the cycle repeats. Provinces had different policies and approaches to managing the infusion, and many people had to pay additional costs to have the drug infused by private clinics or nurses. In some provinces, the situation was so dire that the health care system wouldn't support the drug administration. While people had the drug sitting on their kitchen table, they were on Kijiji looking to see if they could find someone who would be willing to infuse the drug.

This puts an already vulnerable and desperate population in a risky situation that could end up having an even higher cost to the health care system due to adverse events.

Our concern as we look to the future is that even with the pCPA process, which is designed to establish a consistent funding approach across the provinces, we will continue to see differences not only in reimbursement decisions, but in standards of practice.

Also, of course, a new effective treatment cannot improve the health outcomes of Canadians if the drug is delayed in coming to Canada or, alternatively, does not launch in the Canadian marketplace at all. Canada, with its relatively small population, must be a competitive player in attracting manufacturers to bring their therapies here throughout all stages of the therapeutic pipeline.

There are more ALS therapies on the horizon. We do not wish to see the challenges of the last 18 months repeated as other therapies come forward. We cannot leave a desperate and vulnerable population without hope when they can see it just across the border but don't have the physical or financial means to access a therapy that could save their lives.

One thousand Canadians are dying of ALS each year. How many more Canadians will die before our health care system responds to the needs of Canadians who are unfortunate enough to receive an ALS diagnosis?

[The Chair, Liberal, Bill Casey]

Thank you very much.

We'll go to questions now. We'll start with Dr. Eyolfson for seven minutes.

[Doug Eyolfson, Liberal, Charleswood—St. James—Assiniboia—Headingley, MB]

Thank you, Mr. Chair, and thanks to all of you for coming.

Ms. Wong-Rieger, thank you for coming. It's good to see you again.

You've made some mention of our pricing policies and how there have been concerns that they are making this a non-competitive environment. As you may be aware, Canada pays the second- or third-highest costs for drugs in the world. Many other countries are paying less and have universal pharmacare systems. Some are a mixture of public and private. Others are completely public. New Zealand, for instance, is a country with a small population over a reasonably large area. They have a universal program. They pay much, much less for drugs. The government buys commonly used drugs for probably less than a tenth of what we pay.

Are countries like this that are paying much less for drugs having problems with the marketplace not wanting to develop new drugs? Are people in New Zealand having trouble accessing them? Are people throughout the EU, which is paying much less for drugs, having trouble accessing drugs for rare disorders?

[Dr. Durhane Wong-Rieger]

The challenge in terms of accessing drugs for rare disorders are many, as you say, and they vary in different countries. As we say, nobody has an ideal system.

When we look at New Zealand, let's be real clear. Nobody with a rare disease gets access. We know the New Zealand Organisation for Rare Disorders very well, and almost nobody gets access to any treatments. For his two children, the past president never got access to a drug that actually was quite readily available both in Australia and in Canada. If you want to use New Zealand as an example, it's absolutely not going to be supported in terms of rare diseases. Their formulary is very limited. New Zealand is just off the table in terms of any kind of comparison.

You can look at other countries. Obviously, there are some countries that do it better. Partly what we will recommend is looking at the countries that do it well.

How do we bring these in early on? How do we negotiate a price? Part of the challenge in terms of pricing in Canada, as you know, is that we do have a private market system that actually does not negotiate as one in the same way that the public system could. The difference in what we pay privately and what we pay publicly is huge.

Quite frankly, the reason we get such great prices publicly—and we know when we look at those prices that they are much lower than many other countries would get for those drugs—is that they can offset it with the private. If we're going to have a harmonized system, one system, then we're going to have to look at somewhat of a blended price. We agree.

We do agree that the prices can come down lower in Canada, and they should come down lower. We do not agree with what the regulatory reforms are suggesting, that is, that we put a value-added proposition up front to look at the list price. Again, this is not what other countries do. It will put us at a disadvantage.

We agree. Negotiate better in order to get a price that's actually going to bring us down, no doubt about it, but one of the ways to do that is to negotiate as a country, to negotiate as one drug plan. We believe that would be helpful. Also, definitely, bringing in the drugs earlier on and then negotiating those prices, the way Germany does, the way France does, the way the U.K. has been able to do, and where we've seen Australia going...these are some of the models. We think Canada could do quite well if it follows what it's doing but recognizes that the differential in pricing has to do with the fact that we do have a large private component.

That's a different question in terms of how we want to address this, and it's something that Dr. Hoskins is trying to address for pharmacare, but quite frankly that is not actually the situation with regard to public prices in terms of rare diseases. We negotiate pretty hard on those and we get them down pretty low. The problem is that it takes two to four years to negotiate them. There's where the tragedy lies. I think we can have a better process that can do it in a much shorter period of time if in fact we look at the negotiations at the time of the NOC, with the HTA having been done appropriately at that point.

[Doug Eyolfson, Liberal, Charleswood—St. James—Assiniboia—Headingley, MB]

If you were to pick a nation that would be the ideal for the best approval for rare diseases, which nation would you pick?

[Dr. Durhane Wong-Rieger]

I think we could do a great job in Canada by choosing the best from all of the countries. We can look at what—

[Doug Eyolfson, Liberal, Charleswood—St. James—Assiniboia—Headingley, MB]

Let's nail it down to one.

[Dr. Durhane Wong-Rieger]

I don't think we can nail it down to one, because nobody does it perfectly.

Canada, as you know, is a wonderful hybrid. We can learn from everybody. I think that's where we want to go.

We can look at what France does in terms of providing it immediately. We can look at what Germany does: have a managed access scheme that brings in the drugs, and we'll renegotiate the price after a year. We can look at the U.K., which says it has a highly specialized approach for ultra-orphan drugs.

We can adopt all of those in our proposal for a managed access scheme in Canada. Bringing in those drugs and making them available to people under a monitoring system, with information that will allow us to decide who can continue and what the right pricing is over a period of time, we think picks up all the best of those.

Canada can step up, and I think other countries can say, “Why don't we do what what Canada does?”

[Doug Eyolfson, Liberal, Charleswood—St. James—Assiniboia—Headingley, MB]

Thank you.

We've talked about pharmacare, and I know you gave us some testimony on that. I won't bother there.

There's a recommendation regarding the government supplying CADTH with additional funding to undertake its capacity to review high-cost specialty drugs and develop expertise to support these negotiations.

What other recommendations would you ask this committee to make to improve access for rare diseases?

[Dr. Durhane Wong-Rieger]

First of all, we want to make sure we do not have the barriers that the proposed reforms to the PMPRB would have, and that is to discourage countries from coming in.

I think we want to talk about having a national program that would allow us to bring in all of those patients. The numbers are small, so we're not going to be able to....

Most of these drugs have to come into specialty clinics, so we have a proposal for a rare disease strategy that would also include centres of excellence. They would include the ability to do what we call a life-cycle approach. We can bring in the drugs. We can identify early on, as we're bringing them in, which patients are going to be immediately eligible—like those with ALS—and which ones are not at all going to be eligible, set up a monitoring program for them to have access to it, and then over time, as we are learning from those patients who are on it....

In the old days, we would call it post-market monitoring. As Cathy talked about, under the new bill, Bill C-17, we can enforce those kinds of post-market monitoring programs that we can learn from.

We have a lot of tools at our disposal and, quite frankly, we do not necessarily need to invent them. We can look at some of the model programs we've put together. Canada knows how to do this. We already have these kinds of monitoring programs with very specific drugs, but we want to be able to make that the standard, make it so we can do it nationally, which we don't necessarily do well, and then not wait until we've gone through two more years of negotiations before we implement it.

We know how to do it. We just need to make sure we put that template in place and have the specialty clinics that are there also able to support the use of them, and, as she talked about, the administration of them, and the ongoing data collection to make sure they're used appropriately and, at the end of the day, price readjustment.

[Doug Eyolfson, Liberal, Charleswood—St. James—Assiniboia—Headingley, MB]

Thank you.

[The Chair, Liberal, Bill Casey]

Thanks very much.

Mr. Lobb.

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

Thank you very much, Mr. Chair.

We can maybe see where this goes, but I think it would be great if we could have the officials back to conclude this round of meetings and studies to see if they have had any thoughts after we've gone through this and whether they'd like to add to this discussion.

Durhane, you made a great point in the beginning about Dr. Stewart. I know they're here to do a great job and everything else, but I can tell you, the number of people who call my office who are not happy with the way Health Canada.... It's not to blame a government; it's just to say the state of the situation. They're not happy. Then I have drug companies that come to visit and have meetings with me. They're not happy. Yet, Health Canada sits here and tells us everything is going pretty well. It seems to me there has to be a disconnect here.

There are 13,000 SAP applications, and I think they made a comment today that they're knocking off about three per year or something like that. They're getting three of these companies to come in and have licensed drugs.

How do we convince these companies to go from being in the SAP to being licensed? What do we have to do as Canadian parliamentarians, Health Canada or the Government of Canada?

[Dr. Durhane Wong-Rieger]

Certainly we believe that the modernization of the regulatory frameworks has provided some tools that can make that happen.

You need to look at where the different barriers are. In some cases companies don't come, because these drugs have been around for a long time, and there's no new evidence in terms of how well they work. If you have a process that says, “We need to have you submit evidence based on clinical trials on how these work”, these companies are saying, “I'm not going do a clinical trial for Canada; we have 20 years of evidence, in terms of real-world usage.” That hasn't been enough sometimes to get them to get an NOC.

That's number one. We need to be able to accept real-world evidence the same as the FDA does, to say, “Okay, that can happen”.

The second thing we need to look at is that in some cases these companies aren't coming in because it costs a lot. This is a generic drug by this time. They're making peanuts on it. In fact, sometimes they're making less than peanuts on it, because it's lower than their costs. Now they would have to pay an application fee of how much in order to get this drug approved. After it gets approved, we have companies that are caught in a loop that they must now submit to CADTH in order to get it reimbursed. Well, hello, it's already being reimbursed as an SAP drug. Now we want them to pay 70-some thousand dollars to get us to reapprove it. We're not going to negotiate a price, so why would they do this?

We put in these bureaucratic obstacles that do not need to be in there. I just had a company that called me again and said, “Can you please just get Health Canada to accept the IND that we have in the States, developed for a clinical trial? Do not make us write a separate protocol for you. I have five babies that are waiting for treatment. Can we not do this right away?” I'm kind of thinking that it should be able to do it, but we haven't been able to figure out how to remove those kinds of barriers. It can be done, but I think we need to have an even more enlightened approach.

I don't fault the individuals who are there. I really have huge respect for everybody who was here at this table. They work with us as hard as they can. Good God, we keep throwing land mines in their path and tell them to keep jumping.

I think this is the challenge, and at the reimbursement end as well. This is a problem for us. We have companies.... Actually, let's talk about the drug Cystagon. The company brings it in. It's been here for years. They have a certain price, and now we're saying to them, “We'd like you to have an NOC on it so that we can get it out of the SAP.” They're saying that in order to do that, they are going to have to raise the price of the drug. Then, we have one province—I won't tell you which one; I should tell you which one it is—that says, “Oh, you've raised the price of that drug. We don't want to reimburse it any longer, because it used to be cheaper.” Now we have to intervene with the province, to say this is ridiculous.

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

I would call it a Ponzi scheme. I think that's what you're going to see, a rare disease Ponzi scheme amongst the pharmaceuticals.

A drug company visited me about this. It bought a company that had one product and paid $800 million for it. They said to me that they would now have to charge a certain amount to make their money back. Who told them to go and spend $800 million on this product?

That's in the same breath with Mylan. That's what they've done. They sold the rights or whatever. Whoever it is who bought them, Recordati or whoever it is, can now justify saying that it is going to cost more money, that it is going to be extra.

I hear what you're saying, and I can imagine all my colleagues here are so frustrated that it's right there; I don't understand how it can't get done. I'm sure we're all very frustrated with that.

I'd like to ask Ms. Moore a question in regard to the clinical trials.

Any member of Parliament who's been doing this long enough, whether it's in our social circles or just out and around, we've all met people who have had ALS. It's terminal, and it's pretty tough to see that take place. When it's in a situation like that, what do we have to do to get it from six weeks to virtually immediate?

[Tammy Moore]

It would be a simple alignment, the same as what they have with the FDA in the U.S. When the clinical trial protocol is approved, it automatically gets open label extension, unless there has been an adverse event or something to signal safety issues associated with it. There should be no reason that a separate application has to go through for an open label extension once the clinical trial protocol has been met. It seems like a simple solution. I'm not certain.

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

I have another question for Durhane.

One comment that Dr. Stewart made—it was kind of interesting the way the two witnesses were talking—was that we'll respect the commentary from the physician and the patient, because in some cases they know best. At the same time, Dr. Stewart was saying, and I'll paraphrase, that may be, but you're still going to have to have your physician, etc., prove without a shadow of a doubt that they have to stay on this drug.

You see thousands of people throughout your career. Is that the right approach to take?

[Dr. Durhane Wong-Rieger]

Again, I think the way in which the process works penalizes the clinician and the patient, and we don't have to do it that way. I think that if you have patients, as you've rightly said, who are on a lifetime drug, the necessity to reapply every three months because it has not actually been given NOC makes no sense whatsoever. It's the anomaly in which the SAP is written. We get it, in terms of the way the law is written, but that can actually be changed.

Our recommendation is to bring those drugs into a national rare disease pharmacare program. We can deal with them in the same way as we deal with all of the drugs that have uncertainties. You have a panel. You set up the guidelines in the protocol, and you make sure that the patient and the clinician are fitting within that protocol. The monitoring sits within that program. It doesn't have to be bumped up. As long as you say there's no adverse event, as long as the patient is responding appropriately to it, and as long as the physician can attest to it, then the patient just stays on the therapy.

There should not be the necessity of having to go back every three months to reapply. As all the clinicians say to us, they don't get paid to do this. I'm not saying that in any kind of derogatory way. They have hundreds of patients. This is actually taking away from them being able to do something else.

So yes, we could do that, and I think what we want is to go back and ask what the real experience is here. In some respects, I think what we often hear is a request for a modicum of common sense when we're doing these things. If we could just introduce that, I think that would help a whole lot in terms of what we're able to do.

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

I know my time is up, but I would just like to say that many issues on Parliament Hill are political, whether it's your views on taxes or something else, but I don't think this one is. Whether you're NDP, Conservative, Liberal, or other, you should want this to be best for everyone who lives in your area.

[Dr. Durhane Wong-Rieger]

Thank you.

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

Let's hope we have some good recommendations that come out of this.

[The Chair, Liberal, Bill Casey]

Thank you, Mr. Lobb.

Mr. Davies.

[Don Davies, NDP, Vancouver Kingsway, BC]

Ms. Moore, ALS Canada's website says, “There are no significant sources of Canadian ALS research funding other than ALS Canada, resulting in a need for directed support from other sources like the federal government.”

How much funding does the federal government currently direct towards ALS research?

[Tammy Moore]

There is nothing specifically directed towards ALS research in Canada. We were fortunate in the past to be able to secure matched dollars through the Canadian Brain Research Fund, in partnership Brain Canada, but those were matched dollars, and the only reason we had those dollars available was because of the ice bucket challenge. Other than that, we are a Cinderella disease, and we can't get out of that cycle.

We provide for gaps in the health care system. Our societies across the country work in a very strong collaborative model, and we are filling a gap within the provincial health care systems to provide hospital beds, wheelchairs, ceiling lifts, ramps, and things that enable people to stay in their homes, where they are best cared for, and out of the health care system.

We're grassroots fundraising organization, and we're now back to the fundraising levels we were at prior to that one anomaly, so we are back to about $2 million that we have to direct towards research. There is not the same opportunity going forward for the $10 million that we secured from Brain Canada, because we don't have a massive pool to draw from.

Aside from that, our researchers have the opportunity to apply to programs like CIHR. However, because we are a relatively small population, both in terms of the number of Canadians living with ALS and the number of researchers, we have a very small opportunity for success within CIHR. Once again, it becomes this Cinderella disease. How can we possibly break this cycle using population-based research funding models or population-based research support?

I would even challenge that as we're talking about the support of clinical trials, we're talking about registries. The Canadian Institute for Health Information does not get down to the level of ALS when looking at a neurodegenerative disease, so the data collection on ALS within Canada is done by societies like mine, in partnership with donor-funded, volunteer-based organizations across the country. In P.E.I. there are three volunteers supporting the people living with ALS in their province. They are trying to help collect data to support advocacy efforts and to support clinical trial information. We need other systems in place to support rare disease, and diseases like ALS.

[Don Davies, NDP, Vancouver Kingsway, BC]

Thank you.

Ms. Moore, you mentioned some of the difficulties with patients facing significant out-of-pocket costs.

Ms. Smith, have you faced any significant out-of-pocket costs? I'm curious about what the financial impact has been on you. Also, what recommendation would you give this committee if you were health minister or prime minister?