Thank you for the opportunity to be here today to discuss Health Canada's role in maximizing the quality, safety and effectiveness of drugs for rare diseases and in making these drugs available to Canadians.
Joining me today is Dr. John Patrick Stewart, and Ms. Karen Reynolds, also of Health Canada.
Rare diseases are life-threatening, debilitating or serious and chronic conditions affecting a small number of patients. There is no international standard for the definition of a rare disease. Many definitions include prevalence or incidence of the disease being targeted. Within Health Canada we have adopted a definition similar to that used within the European Union of a rare disease being one affecting fewer than five in 10,000 Canadians. Some of these diseases are considered ultra-rare and may even affect fewer than 10 Canadians.
However, although the number of Canadians with a particular rare disease may be low, there are thousands of rare diseases, and it is estimated that as many as one out of every 12 Canadians is affected by one.
Many of these diseases are genetically based and appear at birth or in early childhood. They can lead to a shortened lifespan and reduced quality of life and can place significant challenges on patients, caregivers and the health care system.
I understand you will hear directly from patients living with rare diseases. You will hear first-hand about the difficulties they often face in getting an accurate diagnosis, which can take years and require several specialists.
I now would like to explain the role that Health Canada plays in making drug treatments for rare diseases available to Canadians in need. We are aware of the challenges associated with developing and bringing to market drugs to treat rare diseases, which Canadians often refer to as orphan drugs. These challenges include limited to no information on the natural history of the disease and small patient populations, which make it challenging to conduct the typical clinical trials that are normally necessary to support drug development.
Health Canada oversees the testing of new drugs in clinical trials and their eventual authorization for sale, as well as the post-market surveillance. This includes drugs for rare diseases. Clinical trials represent a good opportunity for rare disease patients to access treatments and to contribute to supporting research to further understand their disease and the potential therapeutic benefit of new therapies.
Health Canada reviews clinical trial applications quickly and efficiently and provides free scientific advice to drug manufacturers on the design of clinical trials in small patient populations. Once a clinical trial is authorized by Health Canada, the trial is included in the clinical trials database. This database aims to help patients and their primary care providers to find available trials and it supports the recruitment of rare disease patients.
Once a drug manufacturer has sufficient evidence of a drug's safety and efficacy, it may seek a market authorization in Canada. Health Canada issues market authorizations for drugs, following an assessment of a complete dossier of information showing that a drug is safe, effective and of high quality, that the benefits will outweigh any risks, and that the risks can be managed. Once the drug is marketed, we continue to oversee it in order to monitor the safety profile.
Drugs that are intended to treat serious or life-threatening diseases, as many rare diseases are, are accelerated through the regulatory review process and given priority status or conditional approval. These accelerated pathways provide earlier access to promising new drugs for patients suffering from rare diseases.
However, a market authorization alone will not ensure availability. Canada's health care system is complex and involves the participation of multiple stakeholders who all have distinct roles in the planning and delivery of health care services. Health Canada's decision-making role in approving a drug is distinct from the roles of those who make decisions about cost-effectiveness, price setting and drug plan reimbursements.
Unfortunately, many rare disease drugs are very expensive. Health Canada, while recognizing and protecting its distinct role, must work with all of its partners and key stakeholders to improve access to drugs for rare diseases by encouraging the development and availability of safe and effective products.
Health Canada is receiving applications and approving drugs for rare diseases. Currently 30% to 40% of all new drugs approved in Canada, as well as in international markets, such as the U.S. and Europe, are drugs for these rare illnesses. Recent research from the Patented Medicine Prices Review Board indicates that nine out of the 10 top-selling orphan drugs are available in Canada. In 2017, 16 of the 36 brand new drugs we authorized in Canada are classified as orphan drugs in Europe or the United States. Most of these were reviewed and approved using our accelerated pathways.
Drugs that are not marketed in Canada may be accessed through Health Canada's special access program. This program provides access to unapproved medications on an exceptional case-by-case basis for practitioners treating Canadians with serious or life-threatening conditions when conventional treatments have failed or are unsuitable or unavailable. Approximately 30% of the drugs authorized through the special access program are used for the treatment of rare diseases.
However, we need to do more. Many jurisdictions have specific legislation in place to incentivize the development of rare disease drugs. The orphan legislation in the European Union and the U.S. were put in place to support the development of drugs that would otherwise not be profitable to bring to market.
In 2016, Health Canada launched the regulatory review of drugs and devices initiative, a major effort to improve the availability of and access to prescription drugs, including drugs for rare diseases. This represented a significant funding initiative in budget 2017.
We understand the particular needs and challenges of Canadians with rare diseases and have made a commitment to improve access to medications that treat these conditions. The regulatory review of drugs and devices initiative will make the regulatory process more efficient and better able to meet the needs of the health care system. It will also help to ensure that patients have access to those important new medications approved by Health Canada.
We will do this by working closely with the health technology assessment bodies to reduce the time between Health Canada approvals and reimbursement recommendations. We will also work with our health technology assessment bodies to provide parallel advice to industry on clinical trial designs at an early stage of drug development.
We will consider health care system needs in making decisions about which drugs Health Canada should prioritize. We'll use existing and new real world evidence to support regulatory decision-making across a drug's life cycle.
We will determine the best way to incorporate patient input into the regulatory approval process. Health Canada recognizes the value of information gathered from patients as the direct users of these products. This is especially true for rare diseases, given the limited information available and the fact that, for some of these diseases, patients and/or their caregivers may be the best experts.
Furthermore, Health Canada is renewing and modernizing the special access program to better meet the needs of physicians and patients. Improvements have already been implemented to provide greater assistance to physicians, and other changes are planned, including a new electronic system that will streamline requests.
In addition to helping to support interested parties navigate the regulatory framework, Canada's regulatory approach to drugs for rare diseases is now described online on Canada.ca.
We have heard that recognition of the orphan status of these products is important to many stakeholders. We now identify drugs that are considered orphan drugs in Health Canada's annual new drug authorizations report, which we have brought copies of for you.
We have sufficient flexibility under our existing regulations and policies to accommodate the challenges posed by drugs for rare diseases and make them available in Canada. Our ongoing regulatory review provides us with an opportunity to continue modernizing our regulatory approach to help support getting Canadian patients the medicines they need.
Now more than ever, it is a shared priority of Health Canada and its federal, provincial and territorial partners to improve the affordability, accessibility and appropriate use of prescription drugs in order to better meet the needs of Canadians.
The short answer is yes. The special access program has been in operation for a long time and, as I mentioned earlier, it requires a dialogue with physicians, so the physician has to determine that specific therapy is the best choice for their patient.
They submit a request; it's assessed in the program and then it's ultimately authorized. There may, at times, be problems with documentation or lack of documentation, so there may be a further dialogue. If there isn't a response, it may ultimately be cancelled. If the dialogue with the physician is such that it is determined that it's not the best therapy, then it may be withdrawn.
Actually, formal denials are 0.3%. Last year there were 13,000 requests for the special access program, and only 48 were denied, so it's very rare.
Speaking to your question about process improvements, one of the things we've done is to look at when denials happen and ask how we can make that better. In the last year or two we've actually hired additional resources with clinical expertise, so we have a clinical pharmacist who actually works in the health care system as well as working for Health Canada. Before we make any denials, currently this individual, or someone with similar skills, will contact the physician and have a dialogue. Putting this in place has dropped our denials from about 1.5% to 0.3%, so there is a conversation.
You mentioned the situation with the drugs for the skin condition, larva migrans. We can't speak to specific requests, because that is private information, but I would just say that one of the bars that used to be met is that the request has to explain why the condition is serious and life threatening, and have the documentation on the use and safety and efficacy.
Sometimes requests will come in where that isn't clear. If you have certain conditions, like infectious diseases or the type of conditions that can be self-limiting or there can be aspects where it becomes life threatening, it requires a dialogue to determine whether the situation is serious and life threatening when it's approved.
Putting in place these additional clinical contacts I think will go a long way to avoiding situations where the problem is more clarity of information between the program and the practitioner.
As for other improvements, the communication is there. We've added clinical expertise. We've actually increased the collaboration across directorates and with the bureaus involved with approving drugs. You mentioned drugs that have been on the program for a long time, and this is an area where we are focusing and we've had dialogue with industry on why these drugs aren't coming in. Is it low volume or is it marketing decisions? We're putting in place a series of incentives to try to get companies that have had their drug on the program for so long to actually come in and market the drug.
We've had some success in the last year. We've had three of the top 10 drugs that were on the program for a number of years actually come in and get market approval, so that's a lot of help for pharmacists, for physicians and for hospitals, and we continue to work on that.
The other challenge you mentioned was patients who are on drugs for a long time on the program. Again, we've looked at this and we are changing some of our processes around situations where it's likely there will be a repeat request where the authorization period could be extended.
Having said that, the program is providing access to unapproved therapies that haven't gone through a formal assessment by the regulator to look at safety, quality, and efficacy. Some of these drugs are well known, and some are not, so the hesitancy to provide a long period of approval is that really they're not at the same bar. We don't have the same understanding as we do with approved drugs on the market in Canada.
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.
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.
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.
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.
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 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.
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 , 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.
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.
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.
Speaking honestly, as we were dealing with this regulatory framework, we kept getting promises that it was going to be submitted, and we kept hearing that the provinces are concerned about it because they're afraid that by having this framework, we're going to increase the number of rare disease drugs that they're going to have to pay for, which was a tragedy.
Part of what we also said was that we want to be able to support having research and development in this country. That's what this framework would have done. We do have drugs for rare diseases that are discovered in Canada and then by the time they get into clinical trials, they've been picked up and gone elsewhere because we do not provide the incentives to nurture that development, as you heard from Cathy and as you'll hear from Tammy.
At the end of the day, we're just net payers. We do not have a research infrastructure, but we also don't have a development infrastructure that says.... When Europe introduced their orphan drug act in 1999-2000, some 15 years after the U.S., they didn't do it just to serve their citizens. They did it because they said all the research and development monies for orphan drugs were going into the U.S. They needed them in Europe, and they developed that framework to make that happen. That was part of our hope. Can we support research and development in Canada as well so we're not just net buyers, so we're net contributors, and if we're developing some of those drugs, some of those profits come back to Canada? We have not put ourselves in that position, so this is a real challenge for us.
Yes, there are lots of challenges. Part of that framework would have supported that. It didn't even go far enough to do that. We thought it was a good starting point, so let's built on it. It was also a signal to the world that Canada is open for business in rare diseases and orphan drugs. Come and develop with us here. Let us support that.
We hear this innovation mandate from this government and we laugh and say that on the one hand, we're talking about it and on the other hand, we slap it away, including what's happened with the regulatory reforms on pricing. On the one hand, we say we want to make Canada first, that we want to encourage the innovation to come here, but on the other hand, we say let's put up big barriers so nobody wants to come here first. Really? You wanted me to speak honestly.
My problem is I think there's schizophrenia here. Can we get it together? Are we going to be number one? Are we going to support innovation? Are we going to encourage research and development in this country? Are we going to encourage drugs coming in and having clinical trials here and making them available to people or are we not? On the one hand, we say we are and we are doing some things to make it happen, and on the other hand, we keep creating more and more barriers and making it more and more challenging for us. At the end of the day, the poor patients are the ones who lose here.
Yes, you can imagine that as a loved one becomes gradually paralyzed, there are the very physical mobility aspects. That is where an organization like ours—and in your riding it would be our organization—would help to fill a gap left by the health care system.
These people are in their homes and as they're losing their independence, they require supports: hospital beds, wheelchairs, ceiling lifts, things to be able to get people in and out of their homes. If those aren't in place, then they're even further isolated, but again, as their care needs increase, our health care system isn't keeping up with that, so often a caregiver, someone within the family, will have to stay home.
Mr. Davies had asked about the costs associated with it. We had done a study and we know that the costs associated with ALS are between $150,000 and $200,000 in the course of the two to five years that someone will live with this disease. We're talking about loss of income as well. The social isolation as well as the financial constraints become even more significant, so our population is making very hard decisions: “Do I bother modifying my home for the six months I'm going to enjoy it? I'm going to have to take the equity out of my home, out of my child's education fund to be able to support this,” or unfortunately, “Will I have to make other choices about how I'm going to live out my final days as a result of those hard financial considerations?”
Social isolation is an important aspect that we also help to support. Again, we are a donor-funded organization that has people in our communities around the province. We work hand-in-hand with the ALS clinics, but once somebody is diagnosed, they're immediately signed up with our societies and our people will go to their homes and help them to start navigating their journey. We're providing psychological support groups so they're able to be with people who understand what they are going through. We have many supports in place like that to be able to, but we're a donor-funded organization. Should a charity have to fulfill this role in our society?