Proceedings of the Standing Senate Committee on
Social Affairs, Science and Technology
Issue 15 - Evidence - May 3, 2012
OTTAWA, Thursday, May 3, 2012
The Standing Senate Committee on Social Affairs, Science and Technology met this day at 10:30 a.m. for a study on prescription pharmaceuticals in Canada (topic: Clinical trials).
Senator Kelvin Kenneth Ogilvie (Chair) in the chair.
[Translation]
The Chair: Welcome to the Standing Senate Committee on Social Affairs, Science and Technology.
[English]
My name is Kelvin Ogilvie, and I am a senator from Nova Scotia. I will ask my colleagues at the table to introduce themselves, starting on my right.
Senator Seidman: Judith Seidman, from Montreal, Quebec.
Senator Martin: Good morning. Yonah Martin, from Vancouver, British Columbia.
[Translation]
Senator Demers: Good morning, I am Jacques Demers, from Quebec.
Senator Verner: Good morning, Josée Verner, from Quebec.
[English]
Senator Callbeck: Catherine Callbeck, Prince Edward Island.
Senator Merchant: Pana Merchant, Saskatchewan.
The Chair: Thank you, colleagues.
I am pleased to welcome our distinguished witnesses here today. We are continuing our study on prescription pharmaceuticals in Canada, dealing purely at this stage with clinical trials. This meeting is to focus on patient and disease advocacy groups, and we have two presenters. I will introduce them as I call them to speak. By predetermination, I will start with Dr. Kathy Kovacs-Burns of the Best Medicines Coalition.
Kathy Kovacs-Burns, Operations Committee Member, Best Medicines Coalition: Good morning, honourable senators. Thank you very much for this opportunity to talk with you about clinical trials for pharmaceutical products. As patients, we take this topic very seriously, particularly since clinical trials are critical to ensuring we end up with prescription drugs that have positive health outcomes.
We do have some general issues with clinical trials. As patients, the issue is not that we think all clinical trials can be framed the same way or even critically analyzed the same way. Sometimes there is a feeling on our part that early Phase I and even Phase II clinical trials are questionable as to whether they are gold standards, particularly when healthy volunteers are selected and sample sizes are very small. However, we realize that there is really no other way to assess or determine the effectiveness, efficacy and, equally as important, some of the safety considerations for those products that will be targeted as prescription drugs.
The issue for patients is the entire presentation of clinical trials to and with patients, some of whom actually have no other treatment choices available to them. First, the awareness to patients that clinical trials are starting is actually quite poor. Either they will take part as suggested by their physicians directly or announcements are ineffective in reaching them so that they can actually participate in clinical trials.
Second, being a subject, or as some would say, a guinea pig, can be demeaning, certainly without the consideration and respect of being a contributing, engaged partner in the clinical trial. This includes being part of the clinical research teams. That includes being part of the research design and discussions for ethics and consent discussions, recruitment, implementation, data collection approaches, and the results and reporting of them. This latter is the third issue: the holding back of clinical trial results, especially those that are negative and contain potential safety considerations.
We have asked to be engaged at clinical trial research tables. We are still waiting.
We read about, talk with and hear the evidence of other patients in other countries, particularly the U.K., the United States, and other European countries, who are engaged in clinical trial research. We have to wonder why Canadian patients generally are not part of the clinical trial research in Canada and why this seems to be such a stumbling block when it could be a mutually beneficial partnership.
Our recommendations are that, first, Health Canada needs to take more of a leadership role in implementing and enforcing the progressive licensing and life cycle model for drug review, starting with early clinical trials to submission trials for review and finally post-marketing monitoring and evaluation. Second, as part of legislative reforms, the consideration is not only for the life cycle approach and progressive licensing of drugs, but making it mandatory for all clinical trials to be registered. Only then can there be more focused follow-up on each clinical trial and monitoring of results and reporting. Investigators, along with companies, manufacturers or other research institutions, should be held accountable and liable for withholding any clinical trial results, either negative or positive.
Third, Phase IV clinical trials should become the norm, not the exception. Without an adequate drug safety surveillance system in place, making Phase IV clinical trials mandatory may be the only other recourse to monitoring the safety of new drugs on the market and being prescribed.
Fourth, Health Canada, together with clinical trial investigators, patients and other stakeholders, needs to learn from the experiences of clinical trial leaders in other countries where patients are considered more as partners in clinical trials and clinical trial research. This includes the capacity building through research training and orientation provided not only to patients but also to clinician scientists and research teams. The reports from other countries are clear: There are more benefits to this partnership than drawbacks, and any challenges seem to be dealt with as part of the process. They have worked out the kinks in their processes, and we can certainly learn from them.
We can never stress enough the importance of safety related to clinical trials or the post-market safety surveillance. Part of this safety consideration includes the release of negative clinical trial results, along with the positive results. Any investigator or research institution who does not comply with releasing negative and positive results should be held accountable to their ethics board as per the Helsinki Principle 30 and to Canadians through legislated progressive licensing accountability.
Health Canada should work closely with the Network of Networks and patient groups to develop a patient engagement framework for clinical trials, research and monitoring of results, and this should be initiated and supported by Health Canada.
In conclusion, we have some opportunities in Canada to build a model that looks at drug review as a progressive life cycle approach and to apply learned applications from other countries. Patients want to be part of the process and solution to making clinical trials in Canada a top priority.
Legislating the monitoring of clinical trial research through the life cycle of drugs more critically and including patients as partners we feel will strengthen clinical trials as gold standards in Canada.
The Chair: Now I will turn to Kelly Gorman, who is a board member for the Canadian Organization for Rare Disorders.
Kelly Gorman, Board Member, Canadian Organization for Rare Disorders: Thank you. It is a pleasure to be here today.
Clinical trials are the keystone to access to new therapies. By participating in clinical trials, patients get access to new therapies sooner and clinicians gain experience with emerging therapies. The implementation of clinical trials can lead to creation of centres of expertise and to the screening and diagnosis of patients affected or at risk.
The things learned from clinical trials are also the cornerstone for advancing evidence-based therapy and evolving best practices. A major purpose of clinical trials is to assure that new therapies meet the standards of safety and efficacy and have a positive benefit-risk profile. Those clinicians who participate in clinical trials are better prepared to identify appropriate patients, participate in the development and implementation of evidence-based treatment guidelines, and monitor patients to assure safe and effective use when the therapy is available to patients beyond the clinical trial setting.
Sadly, clinical trials, or the lack of Canadian participation in clinical trials, can also be the millstone that delays or even denies Canadian patients access to new therapies. This is especially true with respect to rare diseases. Rare disease patients in Canada are much less likely than their counterparts in the U.S. or Europe to be included in clinical trials. Subsequently, companies will be slower to file for approval and slower to make the therapies available to Canadian patients because there is a lack of experienced treatment sites and a lack of demand. This is especially tragic when a new drug is the first effective therapy for that condition.
Clinical trials are inherently challenging for rare diseases. In the decade prior to 1983, there were only 10 new drugs developed for rare diseases. This was primarily because the small number of patients made traditional clinical trials difficult to conduct and the high cost of development meant it would be challenging to achieve a return on investment. In 1983, the United States passed the Orphan Drug Act, which provides financial incentives for companies to invest in drug development for rare diseases and, as importantly, provides researchers with protocol assistance to develop clinical trials that are appropriate to small patient numbers and poorly understood disease histories.
Over the years, clinical trial design has been adapted to demonstrate safety and efficacy with very small sample sizes and short clinical trial periods. Clinical trial outcomes often rely on surrogate measures that are agreed-upon substitutes to clinically relevant outcomes.
Since 1983, the U.S. Food and Drug Administration has approved close to 400 new therapies for rare diseases, many of which are life-saving or disease-altering treatments. In 2000, the European Union also passed orphan drug legislation and since that time has made available more than 75 innovative therapies.
Canada is still the only developed country without an orphan drug policy. In 1996, Health Canada reiterated there was no need for an orphan drug policy because drugs were being developed in the U.S. and these were available in Canada. In reality, fewer than half of the rare disease drugs available in the U.S. or the EU are brought to Canada. Companies do not apply for market approval, so many Canadian patients never get access to these life-saving or life- altering therapies.
The lack of orphan drug regulations has been a disincentive for companies to do research and development in Canada. According to data from the Pharmaceutical Research and Manufacturers of America, as of April 2012 there are more than 460 drugs for rare diseases in clinical trials. Unfortunately, fewer than one half of those trials have a Canadian site. Previously, Peter Brenders, president and CEO of BIOTECanada, spoke to this committee about Canadian biotechnology companies with successful research in innovative orphan drugs. What you may not know is that in many cases, once a company has a promising drug that goes into clinical trials, the clinical trial administration and sometimes the whole company leave Canada.
When a Canadian company discovers a drug targeted at a rare disease, it will file for orphan drug status in the U.S. and/or Europe. When development reaches the stage of clinical trials, some companies will relocate to the U.S. or Europe to take advantage of financial incentives and technological support. For example, Aspreva, one of the most successful Canadian drug biotech companies, relocated from Vancouver to Switzerland, in part to take advantage of a preferable research and development environment.
Similarly, a professor at the University of Montreal discovered and developed a drug for a rare bone condition. The company, Enobia, still has an office in Montreal, but the operations and clinical trials are headquartered in Cambridge. Fortunately, in this case, Health Canada approved the clinical trial protocol, and one of the most active clinical trial sites is at Children's Hospital Winnipeg. As a result, not only do we have a high number of Canadian children enrolled in the clinical trials, but this site also treats children from all over the world. The company has now been sold to another U.S. biotech firm, Alexion, but there is no doubt that Canada will be a site for the Phase III clinical trials of this drug because of the clinical team expertise.
The Canadian Organization for Rare Disorders, CORD, calls upon the federal government to implement the orphan drug regulatory framework as quickly as possible. Since 2010, Health Canada has been committed to developing regulations and hopefully legislation to facilitate research, development and approval of orphan drugs for rare diseases. The elements of this framework would allow Canada to be in a position whereby drugs applying for orphan drug status could come to Canada at the same time as going to the U.S. FDA and the European Medicines Agency. This means that the clinical trials could be approved in Canada at the same time as elsewhere and Canadian patients would have a fair chance to be enrolled.
We have been told that Health Canada considers the orphan drug regulations to be a top priority, but we have yet to learn when they will be completed and submitted for approval. We know, however, that without agreement from Industry Canada and legislative changes we cannot have all the elements of a comprehensive orphan drug policy similar to those of the U.S. and the EU. For example, the proposed regulations do not include the period of exclusivity for orphan drugs, seven to ten years, that is built into the EMA and U.S. FDA legislation. We understand that the regulations do not include the extended patent protection for drugs that are repurposed for rare diseases, and this will also be a disincentive for some drugs to come to Canada.
Nevertheless, CORD believes that the first step, orphan drug regulations and an orphan drug committee similar to that of the EMA will help bring Canada in line with the rest of the developed world and facilitate much-needed access to clinical trials and to new therapies for Canadian patients. Thank you.
The Chair: Thank you both very much. I will now open up the floor to questions from my colleagues.
Senator Callbeck: I thank both of you for your presentations.
Dr. Kovacs-Burns, I have a few questions on your presentation. In the second paragraph you say healthy volunteers are selected and sample sizes are very small. What size are we talking about here?
Ms. Kovacs-Burns: A lot of the initial clinical trials have a very small sample size, less than 100 usually, so it is based on what actual participants are available at the time to be able to conduct that clinical review or trial, but it is a small sample size to make any informed decision.
Senator Callbeck: Might they go with less than 100 in some situations?
Ms. Kovacs-Burns: Definitely. The clinical trials will run at the early stages, Phase I, sometimes Phase II, with less than 100; sometimes decisions are made on that, but from our perspective, it is not a gold standard, particularly when for some diseases there are thousands of patients or even volunteers who might be accessible to be involved in a clinical trial.
Senator Callbeck: You mentioned the problem about patient awareness that clinical trials are starting is quite poor. How can we improve that?
Ms. Kovacs-Burns: It is important when clinical trials are just beginning that announcements reach not only the physicians but also the many patient groups available now, coalitions such as the Best Medicines Coalition, who can reach out to those patient groups and make them aware that clinical trials are starting, that they should look at them and consider being participants in those clinical trials. That is certainly one way, and it is a very effective way. That certainly has not happened.
Senator Callbeck: Whose job is it to see that happens?
Ms. Kovacs-Burns: It is usually the investigators. Anyone who is an investigator for a clinical trial, or the institution that supports that clinical trial, whether it is a pharmaceutical company or even a university, should make the effort of reaching out to as many of the patients as possible.
Senator Callbeck: I would think they would. I do not understand why it is a problem.
Ms. Kovacs-Burns: Yes, we assumed that they would too, but it does not happen.
Senator Callbeck: Second, the subject, or what some would call being a guinea pig, would be demeaning. How do we change that?
Ms. Kovacs-Burns: It has to do with the whole presentation of clinical trials. Our premise is that if patients are good enough to be subjects within a clinical trial, they should be good enough to be part of the clinical trial research. By being part of that research team, they probably can enhance the credibility for patients to be engaged in those clinical trials and in fact increase those recruitment numbers for sample sizes as well. It has to do more with how it is presented and how patients are engaged in that whole research design.
Senator Callbeck: You want patients to be engaged in the clinical trial research tables, as they are in the U.S. and the U.K. and Europe. What are you told when you ask for that? Why is that not happening here?
Ms. Kovacs-Burns: Many times we do not get any response. That is the usual response, just no response at all. Sometimes we are told that patients will probably be more of a hindrance at the research design table because they will not understand the research process and be able to contribute to the discussion.
We are saying that, in fact, many patients today are quite educated and have the capacity to be able to learn the language around research — it could be research 101; but it is not necessarily being part of the technical pieces that we are concerned about as being there to talk about whether the research question is relevant, how is it that patients can be recruited in a better way, those kinds of things.
Senator Callbeck: Ms. Gorman, you say, at the bottom of your first page, that in 1982 only 10 new drugs were developed for rare diseases. Is that in Canada, in the U.S., in the world?
Ms. Gorman: I would have to confirm, but my understanding is that would be in the United States, prior to that.
Senator Callbeck: Then the legislation went through, the Orphan Drug Act, in 1983, and we now have 400.
Ms. Gorman: Close to 400, yes.
Senator Callbeck: It mentions that this legislation provided financial incentives for companies. Could you comment on what those financial incentives were?
Ms. Gorman: I do not know the exact details, but part of it was the fees that you have to pay in terms of filing your drug, and also that it helps with protocol assistance too, for developing some of the clinical trials and the market exclusivity.
Senator Callbeck: Are we talking about large amounts of money here?
Ms. Gorman: It can be, depending on the product. A lot of it is the expertise and advice provided to the development of these drugs. I work with Cystic Fibrosis Canada, and for cystic fibrosis the new drugs that have been made available are because of the Orphan Drug Act in the U.S.
Senator Callbeck: You said we are the only developed country without an orphan drug policy. One policy I want to ask about is Health Canada's Special Access Programme. How does that work? That is for a drug that really has not been approved in Canada.
Ms. Gorman: Has not been approved or has not been applied for. If a company has not come to Canada for market approval, sometimes they would make that decision not to come because of some of the challenges that they would face here and the cost to bring the drug to market here. Yes, special access is available to people to apply to Health Canada to access drugs that do not have market approval in Canada.
The issue with that is that then there is still the cost factor in terms of who pays for the drug. Some companies will have a compassionate care program where they will help cover the cost of the drugs, but with other companies you have to pay for the drug yourself.
Senator Callbeck: What about the time frame? How long does it take?
Ms. Gorman: Through the Special Access Programme? I think it depends on the product. In our experience with my organization it was fairly quick, but that was a product that had been made available and it came off the market. I know for some it is quite labour-intensive because for each individual patient the doctors have to fill out an application form.
Health Canada, with my organization, one time, was quite willing to work with us so we could do it through a clinic where they could submit one application for the whole clinic. However, my understanding is that a lot of paperwork has to be done.
Senator Callbeck: Once the doctor fills out that application, roughly what is the average length of time before the patient has access to that drug?
Ms. Gorman: I would have to get you some statistics. I do not know that offhand. I can just speak to our experience. It was within a couple of months. My understanding is that it can take longer, depending on whether it is a drug that has not been reviewed at all by Health Canada and depending on the evidence that has been presented to Health Canada.
Senator Seidman: Ms. Kovacs-Burns, I would like to ask you something about what you spoke of on the issues of accountability and transparency. You talked about the need to register all clinical trials, and even the need for reporting of clinical trial results, especially negative trials. I would like to ask you what you think the stumbling block is to this, why in fact it is not happening when it is happening in other countries.
Ms. Kovacs-Burns: I do not think I have quite all the details there, but certainly what we have been able to find out, or have been told primarily, is that when companies do their clinical trials, right now there is not a mandatory requirement for them to register. If we are talking about all the different phases of the clinical trials, from I through IV, a company can actually start a clinical trial but not necessarily register it.
That means, then, that if that clinical trial ends up with negative results, they do not have to report it. However, if it was registered, if all were required to be registered, then there would be a mandatory requirement for all results to be submitted and reported publicly.
I think the stumbling block is not having the registration of clinical trials, all clinical trials, as a mandatory component through all the phases of the life cycle of the drug.
Senator Seidman: How do you think that should happen?
Ms. Kovacs-Burns: As I mentioned, the progressive licensing of a drug, which includes the life cycle components, is the one way that we have been talking about with Health Canada. If that progressive licensing were legislated, it could incorporate the different components, one of which could be the registration of clinical trials through all the different phases, and that would be significant progress made in terms of how any company or investigator files for a clinical trial.
Senator Seidman: Other than the actual registering of the trial, what other information do you think Canadians need to encourage their participation in trials and, of course, to ensure safety?
Ms. Kovacs-Burns: Certainly the legislation would help. We have been at the table talking about the legislative reform and the progressive licensing components. We have talked about where patients could be involved.
We are not sure exactly why we are not being included in some of the discussions around how we could actually contribute to the different clinical trial research pieces. We do believe that manufacturers, pharmaceutical companies, other investigators, may feel a bit threatened to have patients involved in clinical trials, but we also have tried to convince them that in fact there would be a lot more benefits than risks with us at the table. It is not that we are out to disclose any information around that clinical trial; it is really to make it much better in terms of access to patients and even the running of the clinical trial.
Senator Seidman: There is more than just registering clinical trials, so do you, as an organization, have a list of information — data, in other words — that you would like to be collected and reported on publicly in the clinical trial process?
Ms. Kovacs-Burns: Yes. With a clinical trial itself we feel that it is very important to report all aspects. Even the design of the research question, the manner in which patients are recruited, the data collection approaches that are used, the results and how they are analyzed, and then finally the reporting of all the results — both negative and positive — are the pieces that we feel are critical for patients and their prescribers to have access to.
Senator Seidman: That is good. I really appreciate that.
Ms. Gorman, it is clear from your presentation that Canada is very slow in conforming with the European Union and the United States on orphan drug trials. Why do you think that is the case? Why do we not have any orphan drug regulation?
Ms. Gorman: That is a really good question. I do not know the answer. It definitely seems that Health Canada is saying it is a priority. We are now waiting for it to get approval. I think it is up to the government, at this point, to make it a priority.
A lot of the newer drugs that are coming are targeted. Sometimes it is very specific patient populations, and how we design clinical trials — having strong regulations around that — will be important to how we target these patient populations. The Canadian Organization for Rare Disorders is trying to push hard for this. We are just waiting for the government.
Senator Seidman: Other than the necessity for government regulations, is there something else that is not working in the system, that is not allowing this to happen?
Ms. Gorman: I think the government is important. Also, we hear from cystic fibrosis researchers, clinicians and patients that, when they are setting up these clinical trials, having to go through the research ethics boards at multiple sites is problematic. For these small patients groups, it is hard to have that expertise from the clinicians. They are looking for resources and capacity building to educate them and to have better coordination, especially in developing clinical trials for rare diseases. The locations and smaller sites are problematic too because there are people who would like to participate in clinical trials, but it is difficult at some of these smaller sites.
Senator Seidman: Thank you.
[Translation]
Senator Verner: I also want to talk about rare disorders and personalized medicine. You mentioned the Orphan Drug Act, passed in the U.S. in 1983; the European Union is also active. You say that you are awaiting a Health Canada decision on that. I don't want to qualify the political will in that area, I'll keep some things for myself for the time being, but has the pharmaceutical industry shown any interest in such a policy here?
[English]
Ms. Gorman: I think the pharmaceutical industry has shown great interest in having something in Canada, yes.
[Translation]
Senator Verner: It's good to know. A few weeks ago, we heard the deputy minister of Health, Mr. Paul Glover, who said that in terms of personalized medicine Canada was as advanced as its American and European partners for establishing policies and regulatory procedures. In general, do you agree with this statement regarding our situation and the situation in the U.S. and European Union? And has your organization proposed some changes that Health Canada could bring to clinical trials regulations in order to better regulate the development of personalized drugs?
[English]
Ms. Gorman: The Canadian Organization for Rare Disorders would have recommendations around clinical trials in Canada and around personalized medicine. The organization probably does not think that we are at the same level as the U.S. and the Europeans, either.
We at Cystic Fibrosis Canada see this seeing happening with cystic fibrosis too because of the number of mutations that exist within cystic fibrosis. Recently, there was a breakthrough drug. It was the first time that there really was a drug that targeted the basic defect for cystic fibrosis, and it is specific to a mutation. Our organization has a national registry. It is our registry that we have managed since the 1960s. Virtually all people with cystic fibrosis in Canada are part of that registry. Most of them can be identified as having that mutation. We are not sure when that drug will be available in Canada because, again, it will probably only affect about 100 people in Canada. It is now available in the U.S. Registries will be another area that will be important. That is something that our organization has, but a lot of the other rare disease groups do not have that in place. That is a role that the federal government could help with as well.
Ms. Kovacs-Burns: When we were looking at personalized medicine and how it relates to clinical trials, we believe, for most patients, that when you are thinking about a clinical trial you are thinking that it will be personalized because that particular drug that is going to that clinical trial could be for me.
It is personalized, even at the very beginning. However, we know that, for example, Phase I clinical trials use volunteers who are healthy. They are not even patients. That part is excluded. When we talk about real personalized medicine and how the drugs impact patients, it comes after the approval given by Health Canada. It is in the last phase, when it is being marketed and prescribed to the average Canadian patient. Only then can we say, as patients, "That drug works for me, or that drug does not work for me. There has to be something else that has to work for me better than this drug that I am trying." We always think of it as personalized, but it is not that simple, not with clinical trials anyway. However, we keep hoping that one day there will be a drug that will help everyone in that disease group be able to say, "My health outcomes are a lot better than they were before I had this particular drug." It is not quite that simple.
Senator Demers: We have been talking a lot about personalized medicine. We are hearing more about it than ever. We see it, from time to time in magazines and in newspapers. It seems quite interesting in terms of advances. Is it a preferred approach?
Ms. Kovacs-Burns: Is it a preferred approach? I am not sure that we can say that fairly easily. I think that most patients would say, "Yes, I would like to see personalized medicine because I would like to have the choice, and my prescriber would like to have a choice in terms of looking at a drug that is on the market that will best meet my needs and improve my health outcomes." In that respect it is. When it comes to clinical trials, as I said earlier, it is not quite that simple. I think most patients would absolutely like to have personalized medicine because it means that you will be looked at specifically and that all your needs will be met in a particular way. Follow-up will be done in a particular way, and outcomes, hopefully, will be much better for you as a patient.
Ms. Gorman: For CF and any rare disorder, every treatment is personalized because of your own uniqueness as an individual. Personalized medicine for certain things is coming. We hear that especially with genetics, but, even with having certain mutations for some diseases, there are modifiers, our environment and other things to take into consideration. There is a lot of talk about personalized medicine, but it will involve a bunch of different factors. However, I think we will get more advanced in treating individuals.
Senator Demers: Thank you.
Senator Martin: Obviously, the advocacy work that you do for patients is very important. Without the patients, industry would not be needed. Therefore, it is an important relationship that you need to maintain.
I go back to what you were discussing about wanting to be at the table and fully engaged in the clinical design. However, I wonder about the limitations of many patients to be part of that process. Some would be very informed. Yet, if it involves the technical design and all of the technical elements, that would be left to the researchers themselves. At the same time, as you said, it is important to know what the response will be and how you can best design something to get that kind of response and participation.
I am curious about the actual relationship of the industry with your organization and those you represent. I do not mean for you to go into the whole history, but what can be done to improve the communication? What are you doing in that regard? Have you had ongoing discussions? Are there surveys that come to you that allow you to weigh in?
You are saying there has not been the kind of response from them that you desire, but what would you be looking at and how could we improve that relationship? That is a critical process because the industry is serving you and what happens to you affects them directly. I can see why there could be potential adversarial elements but, at the same time, it is such an important interrelated relationship.
Ms. Kovacs-Burns: We think that the relationship with any manufacturer, pharmaceutical company or even a university with investigators has that obligation to consider how patients view the clinical trial that will involve them as subjects. This goes back to the philosophy that we have a vested interest in the outcomes of that clinical trial. We want to know that the testing of a product is done in such a way that it is truly earmarked for patients in the best way possible.
Aside from looking at the ethical issues, which we also consider to be part of that relationship, to be respected as partners means that we contribute. I am a researcher by background, too, so I might be able to understand the technical parts of it and be critical. Many patients are there because of their disease and have nothing to do with the health care system but would still be able to say how the research question benefits them generally, whether they are seniors or pediatric cases or people from ethnocultural groups. All of those things are not necessarily caught up in the technical language.
How do we consider including patients in the clinical trials? How do we best recruit them? Those are the kinds of questions that patients might bring to the table. They are pretty significant because it is part of society. It is part of the fabric of Canada. It is foundational to try to get to an understanding of how those clinical trials will effectively and efficiently involve that broad range of patients across Canada. That also means there are some groups for whom perhaps it will not work. Where are the gaps in terms of that relationship? How is data gathered from these people? Is it only the numbers we are interested in or are we interested in their quality of life and the other qualitative aspects, like their experience with the drug? What if they get really sick on the drug? How is that reported and captured?
For us, being at the design table, that relationship means asking all of those pertinent questions that sometimes escape in the technical aspects of designing that clinical trial because they might be interested in looking only at the receptor sites or the changes in blood pressure or numbers. It is not always focused on the whole person who is part of that particular clinical trial and the outcomes. When we are talking about personalized medicine, part of the process is how it affects patients everywhere and in every way.
That is the kind of relationship we would like to see happen. So far that is not of interest particularly to the pharmaceutical companies, who tell us that we still probably would not be able to contribute. They may be more concerned about their intellectual property rights, which does come up on occasion, but for patients that is not the issue. Why would we talk to anyone about any of that when really our concern is to ensure that the clinical trials are run with respect to the patients who will be subjects in the clinical trial?
Senator Martin: Do you publish journals, documents or reports that you may, perhaps, send to the industry? Is that something you would do on a potentially regular basis? If you know that a clinical trial is taking place, do you inject your input through documents?
Ms. Kovacs-Burns: Certainly there is lots of literature coming from Europe, the U.K. and the United States regarding that kind of partnership and the results. With the cancer groups, they have guidelines for how to build those partnerships and how to train and educate the patients and the clinician scientists so that that partnership becomes a lot more solid. There is lots of evidence out there. We certainly have shared it with the companies that we have spoken to, and we have probably spoken to all of them at some point or another about the kinds of relationships we would like to see happen in Canada. They certainly are aware that we are trying to be part of the clinical trial research team. That is not new to them. However, it has not gone too far.
Senator Martin: Those models do exist, then.
Ms. Gorman: I want to add, from a rare disease perspective, too, that EURORDIS, which is the organization in Europe, actually has a charter for clinical trials. I have a copy of it and I would be happy to send information about it to the senators. They are trying to get companies to sign on to their charter when doing their clinical trials. CORD is starting to look at what they have done in Europe and proposing something similar in Canada.
The Chair: We would welcome that.
Ms. Gorman: Great. I would like to echo what Dr. Kovacs-Burns was saying, which is that having patients at the beginning of a design of a trial will become more important. I do think governments will start to look at this because some of those outcomes are important when it comes to funding decisions, too. We must ensure the clinical trials are designed in a way to get the information that is important to patients because that will come back to patient adherence to therapies.
Some research has been done in the United States as well. I was at a conference talking about the importance of having the patient up front in terms of deciding and designing a clinical trial because the outcomes that physicians might think are important often are not the same when you ask for patient input. There have been some examples in the U.S. where that has been important and invaluable to the design of the trial.
Senator Martin: Ms. Gorman, you talked about Children's Hospital in Winnipeg that is a model example of a site that is doing well. I was wondering what they are doing — and perhaps this is something you may submit depending on the time we have today — that perhaps other hospitals in Canada could or should adopt. There are other major centres, like Vancouver, Toronto and other places, and perhaps we should be sharing these promising or best practices. What makes this particular place so successful? I am curious to know about that as an example.
Ms. Gorman: We could provide further information about that. There are other sites in Toronto, the Hospital for Sick Children there, and the BC Children's Hospital, too, for other rare diseases where there are good practices. One of CORD's recommendations is having somewhere to share those best practices and whether that could be an office for rare diseases where people can go in order to get that clinical advice.
The Chair: We would welcome the examples. Do you interact with CHEO here in Ottawa with regard to the genetic disease developments that are occurring there?
Ms. Gorman: I can check with Durhane Wong-Rieger around CORD. I do know from cystic fibrosis that we interact quite a bit with CHEO. I am sure we would be able to get some additional information.
The Chair: Could you give us some examples of the best practices in following up on the question?
Ms. Gorman: Sure.
Senator Merchant: I want to deal with the ethical issues and whether they are addressed appropriately in clinical trials. I believe that each clinical trial site must have the approval of the research ethics board. Can you tell me something about the composition of the REB? How many members are there? You are with cystic fibrosis. Do you sit on this board?
Ms. Gorman: No.
Senator Merchant: Who sits on this board? Can you tell us something about the composition of the board?
Ms. Kovacs-Burns: There are standards for research ethics boards that stipulate the membership. Most of the membership consists of physician and dentist scientists — primarily clinician scientists. As well, administrators from the institution are present. On some boards, we have been trying to talk with Canada Standards about revising some of the requirements for research ethics boards to include at least one community member. That is as far as we were able to get. It could be a lay person from the community; it could be a patient; or it could be a retired health professional. It would be someone who is external from and or recently involved in any type of clinical trial or that type of situation at all.
There are no patients involved in the research ethics boards, not even called as witnesses primarily. Certainly, clinician experts can be called in, but I have never heard of a situation where a patient has been called in.
Senator Merchant: Are you saying that there are several boards across the country?
Ms. Kovacs-Burns: There are hundreds of boards across the country. There may be a central research board. For example, in Alberta, where I am from, we tried to merge all of the ethics boards into one because we have one health system. However, that was not successful, and we have two. There are other smaller research ethics boards. It depends on the province and the institution. There are many of them. They are not all exactly the same and may have processes that are slightly different. That has been the challenge across the country as well.
Health Canada and the Canadian Standards Association have been looking at trying to develop a standard for all research ethics boards with those kinds of things in mind to ensure that there is consistency in their membership and in their process. That has been a challenge.
Senator Merchant: I would like to ask a question about the way patients are recruited. For instance, there is an obligation to obtain written consent. Is there a consistency in that across Canada? Do all the provinces have the same kind of form?
Ms. Kovacs-Burns: Yes. They do not have the same kind of form across all provinces or institutions, but they would have similar kinds of language in a consent form. The goal, of course, is not only to inform the patient what the clinical trial is about — and some of that is really onerous because there is so much information in the information letter that has to be repeated and reviewed verbally — but also to ask some basic questions. One is does the patient understand what the clinical trial is about and what are the expectations of the clinical trial? There might be some similarity in those concepts, but they are not exactly the same across the country.
Senator Merchant: My next question is about recruitment, whether there are any concerns about coercion of patients and the practice of competitive enrolment, where people may have a financial gain if they can recruit patients. Can you make a comment about that?
Ms. Kovacs-Burns: We have heard and know that those things do exist. Frankly, I do not want to hear too much more about them, but patients have said to us that when they were in their physician's office, it was suggested that they would be part of a clinical trial. Are they asked? Probably not in that case. I would view that as coercion. That does exist.
The other aspect of how patients should be recruited is quite different. We all know that patients need to be asked and then to take some information, mull it over and consider it. There are some side effects or potential for that or other complications, and they need to consider everything before they sign. I do not always think that is done, particularly when patients are — and I hate to use the word — desperate, whereby the clinical trial might be their last choice or option for a good treatment that might work, might save their life. They are more inclined probably to not listen to everything or hear everything, so although they will consent, their consent is less informed. We are aware that those things happen. I am not sure that we can necessarily correct everything except that it does have to go back to the ethics board for review. That is what should happen, but I am not sure that everything is done that way either.
Ms. Gorman: For rare diseases, often a patient's only choice is to participate in a clinical trial. It is important that they understand the risks before they participate. Also, on patient recruitment in terms of cystic fibrosis, there is a small patient population in Canada of about 4,000 people, which is large for some of the rare disorders. They already take so many medications, too. We are trying to work with our patient community to explain the value of being involved in clinical trials and what it means to them. We have developed a brochure around clinical trials. We are working with the clinics to send this out to the CF community.
The other issue is people knowing about clinical trials, especially for some who have rare disorders. CORD is also trying to make people aware of when these clinical trials are taking place. Education is an important component.
Senator Seth: I would like to talk about orphan drug policy. The government currently uses Health Canada's Special Access Programme to allow practitioners to request drugs not available in Canada, including drugs for their diseases or orphan drugs.
Can you describe some of the orphan drug policies in other countries? Do incentives for drug development include government assistance for clinical trial funding?
Ms. Gorman: Yes, in Europe and the United States they provide some assistance to help with clinical trials conducted. I do not know the exact numbers, but we can provide information on what the FDA does.
The Chair: Ms. Gorman, are you familiar with the extended licensing protection?
Ms. Gorman: Yes.
The Chair: Would you comment on that because it relates to her question.
Ms. Gorman: In the U.S. there is extended patent protection. It is seven to ten years for a drug classified as an orphan drug in the United States. In terms of clinical trial, they get protocol assistance from the Office of Rare Diseases in the U.S. for developing the trial and giving advice. The office also has reduced fees for when they file for a drug submission, and they get priority review as well. The breakthrough CF drug was extremely fast-tracked and made available sooner than the company had thought it would be available.
Senator Seth: Typical clinical trials are designed with a control group that is compared to the test group to determine the efficacy of the candidate drug. In your view, can the value added of a candidate drug be appropriately measured with the control group receiving a placebo? Is it ethical to give a placebo to a control group if there is a standard drug therapy available?
Ms. Gorman: Do you mean if someone is already on a standard drug?
The Chair: If there is a disease identified for which there are drugs on the market to deal with it, the question is, is the best clinical trial that versus just the placebo or one that would involve placebo plus the concept of against an existing treatment?
Ms. Gorman: They are maintaining their treatment that they would be on.
The Chair: Yes.
Ms. Gorman: Yes; sorry.
The Chair: This is a complex area because there are those situations where the disease is one where the individual has a life-threatening or otherwise serious disease whereby taking them off the drug would be unethical. Let us back away from that example and go to the broader example of a general disease treatment for which drugs are on the market and then a new entity comes in. In that situation, what would you consider the ideal clinical trial?
Senator Seth: I am asking about ethical as well.
Ms. Gorman: I am not sure I can give a complete answer from CORD's perspective. I can speak with the CORD board, but I believe that they would agree that you have to continue to do your standard treatment. You cannot take someone off something that is potentially maintaining their life. When you are developing a trial, if you are adding something to it, you will have to compare it with their standard medications.
Senator Seth: Do we get the proper results? The patient is already on medication and this is just a control trial. We are giving a placebo.
Ms. Gorman: If you are going to do harm to someone by taking them off medication they need to sustain their life, I do not think you would do that.
We talked about the Phase IV trial and monitoring people long term. That is where the evidence becomes important, because the longer that you monitor people the more health outcomes you will see, and that is where registries become very important too in determining what your markers are to show improved health outcomes. You would be able to see it with someone who is on a different combination of drugs versus someone who is on the standard therapy or a placebo.
The Chair: This is a good question area, Ms. Gorman. I would like to drill back and take the situation separately.
Let us take your last example of the 100 cystic fibrosis patients with a personalized characteristic who have a treatment already available. A new drug comes into a test phase and evidence indicates it might be of benefit to them, possibly even more benefit than the existing treatment, but there is an existing treatment.
In those cases, the trial currently would normally be some patients on the new drug but not on their previous medication. The comparator group would be the patients who are receiving their existing medication but not the new drug as well. That would be one example. That is the very narrow case of clinical trials. In the area that Senator Seth was talking about, the ethics, it is generally considered unethical to remove such a person from all treatment, but it is considered ethical to remove them from their existing medication to receive the new and potentially even better medication; is that not correct?
Ms. Gorman: That is my understanding, yes.
The Chair: That is good. We have the answer to that.
Since you are representation groups, I want to broaden the question into the larger clinical trial area. Everyone gets headaches. A new headache treatment pill is proposed by a pharmaceutical company. From a patients group's perspective, would the best trial be the historic gold standard, some on the new pill, some on a complete placebo, or would it be a trial that has some on the new drug, some perhaps on a placebo, but others who are given clearly identified doses of existing headache drugs on the market? From a patient's perspective, do you have a view on which type of trial would be better in that situation?
Ms. Kovacs-Burns: We certainly do, and it does come back to the ethical part of it. When there are products that are already on the market that are working to some extent and a new product comes out that claims to be even better, our position is that you can do a comparative study. We would not suggest a placebo. In fact, we would not even see that the placebo at this point would necessarily add value to that particular clinical trial. We would want to compare the two products that are out there and see which one in fact is more effective. You might even consider the cost.
You would consider a range of things with the new product versus the product that is already on the market. That, to us, is a clean, ethical clinical trial, not one that takes people off their standard medication and puts them on a placebo. That, to us, is not value added at that stage at all.
Ms. Gorman: In talking about doing comparative studies, that is something we hear a lot about with rare diseases and also cystic fibrosis, especially in children. That is another one we are starting to hear when something is on the market, because often for children it is off-label use, so comparative studies to do with traditional therapy and also the new therapy are important.
Many people with cystic fibrosis are on multiple medications. When new ones come out, it is good for funders to know how the new one compares to a current therapy, and there are not a lot of those kinds of trials being done. Definitely I would say that is an important thing to look at.
The Chair: We have been dealing with important issues here today, and I want to try to broaden them in certain areas and be more specific in certain aspects. I have a direct question on one of the last issues that came up.
Dr. Kovacs-Burns, you were talking about the numbers of ethics boards and so on. From a patients group's point of view, do you think it would be to your advantage to have a standardized ethics protocol across a system where trials are being conducted? You could take a province, for example. We know that trials generally cross provinces and so on.
Would it be in the better interest of the patient if wise people came together and developed a standardized ethics board approval process rather than, as we hear about today, the hundreds of different boards that have to be met? There are two aspects to that question. One is the fact that there are, in some cases, literally up to 100 different ethics boards for which a different submission has to be made by the pharmaceutical company or the research group proposing the new drug, which can substantially delay the ultimate approval of the drug and benefit to patients. The second is direct issue to the patient. If the patient were aware that there is a standardized, ethical protocol, from your organization's point of view, would that be a benefit?
Ms. Kovacs-Burns: Yes to both questions. We certainly think that a standard protocol for all research ethics boards, whether within one province or even across the country, is extremely important. We would want to ensure, first, that if clinical trials are multi-site across the country, the same protocol for ethics is being applied in Ontario as in Alberta for that particular clinical trial.
It is important to have a standard protocol for the ethics boards, particularly when it comes to those kinds of questions about all the standards, the applications, the monitoring of clinical trials and so forth. That certainly is critical to us.
I am sorry; I have lost the second point.
The Chair: There was standardization across the board, and the speed of access to the ultimate approval of the drug is dependent upon how many hoops a test has to jump through, right?
Ms. Kovacs-Burns: That is correct, yes, and that does happen. A reality for most researchers, including clinical trials, is that if they wanted to do a trial across several provinces, they would have to go to separate ethics boards and go through the same process, which, you are right, would delay the starting time for the clinical trial. It is absolutely a problem.
Ms. Gorman: I agree with Dr. Kovacs-Burns' comments. For example, we wanted to test different methods for something that was an airway clearance for cystic fibrosis. It is not a type of medication. It was at 14 sites and they had to go through each of the research ethics boards, and it delayed the start of this trial by over a year. We need something that is standardized.
I also think it is important to educate patients. That is something CORD is trying to do with the health technology assessment, once it has the approval and you are looking at funding. There is a role to play in terms of educating people about clinical trials. Patients want to be engaged, and some of that is around the education piece that can be done through webcasts, conferences and things like that.
The Chair: Thank you. I want to come back to your experience on behalf of patients with regard to orphan drugs — let us take those first, because it is probably more visible. I would like to know about their being introduced into the country.
I want to get you to also clarify for the committee the two things you have been talking about in this regard, so I will ask you a specific question to begin with: When you talk about rare disorders and then you refer to orphan drugs, can you please tie those together for the committee?
Ms. Gorman: They are interchangeable. One of the biggest issues — and I probably have not said this — is that we do not have a definition in Canada of a rare disease or an orphan disease. "Rare diseases" can mean different things. The definition CORD goes by is that it affects 1 in 2,000 people, and that is in line with the European Union. The United States has a slightly different definition. However, it is usually fewer than 200,000 people in the United States that it would affect. That is a rare disease.
An orphan drug is something that would be used to treat a rare disease. "Orphan" implies that people tend not to want to develop them because often there is no financial incentive, so they are orphaned.
Senator Martin: That is quite a difference. Did you say 1 in 2,000?
Ms. Gorman: Or fewer than 200,000 people are affected by it, but not 1 in 200,000.
The Chair: I want to build up from there to the access to orphan drugs in Canada. You gave an example where, from your perspective, there is more incentive for companies to test drugs for rare or orphan diseases.
Ultimately, let us take an example where one is approved for a orphan disease: The trial has not occurred in Canada; there has been no activity on this drug in Canada. I would like you to give us your perspective about the difficulties of bringing that drug into Canada. It has been approved for an identifiable rare disease in the United States. There are half a dozen persons in Canada who might well benefit from that orphan drug. No trial of the drug occurred in Canada. There is no direct activity of the company proposing the drug in Canada. What are the barriers to getting that drug into Canada?
Ms. Gorman: The barrier in terms of the company bringing it in or the patient getting it?
The Chair: Ultimately, from the standpoint of the patient getting it. The first will probably affect the latter.
Ms. Gorman: For the patient getting it, if it is not approved and does not have Health Canada approval, there is no market access here.
The Chair: Can I stop you right there? That is an important aspect. It has not been tried in Canada, so Health Canada has not approved it. What I want you to tell us a bit about on that, from your perspective, is why Health Canada cannot recognize the data that was developed in the trial, say in the United States, and then, by carefully reviewing the protocol to ensure it meets Canadian standards, accept that result and then grant access to the drug.
Ms. Gorman: That is Health Canada's decision, but what I have been told and what I know through CORD and other organizations is that Health Canada is working with the FDA in terms of sharing information and harmonizing some of their review processes, and in terms of the data that they accept from other agencies. They are also working with the European Union. I do not know a lot of detail about that. I can get more information, although I think Health Canada would be able to answer those questions better.
It also depends on the company bringing it to Canada. Some of what we have heard is that the process to bring it to Canada is very time-consuming and very costly because they do not get the reduced fees that they do in the European Union or the U.S. The market exclusivity issue is also another concern for some of these companies. We have been told that there is hesitation from certain companies around bringing their product to Canada and going through that regulatory process.
From the patient perspective, if a product does not have a notice of compliance from Health Canada, then no one will fund it in Canada in terms of giving that drug to you. The provinces do not fund it and private plans do not fund it. Then it would be up to that individual to actually know that the drug exists. That is another thing, because it depends where you live in Canada, who your health care provider is, and your knowledge. The Internet is a great thing, and many people are becoming more educated.
Regardless, assuming that the patient knows that product is available, then they would have to know whom to go to. Again, you have different levels of knowledge and education about their own disease and resources available to them. Let us say it is someone who is well aware on the Internet. They have a rare disease and are seen by someone who understands what their disease is. You can hope the health care provider knows about it, or the patient does. They will speak with the health care provider, and then your options are to apply to Health Canada through the Special Access Programme, which can be time-consuming and a lot of paperwork.
Alternatively, you could go to that company directly to see if they have any type of patient assistance program in place to help you access that drug. Some of them do. Then it becomes an issue, though, of the company deciding what they want to do, because they may be thinking about bringing it to Canada. If they are thinking about bringing it to Canada, they may say, "I will give it to you through our patient assistance program until we get market approval in Canada." Therefore, the patient may be able to access it that way, or they may have to pay for part of it, too.
Then it comes down to the ability to pay. My understanding is that some provinces maybe have helped certain individuals access these drugs, but sometimes these are lifetime drugs, too.
If the company is thinking of bringing it to Canada, they may. Then they have to decide what price to charge, or give it through patient assistance. Once it comes to Canada, and if they do get approval in Canada, then you have to also look at the funding mechanisms available at the provincial level or through private plans. Sometimes provincial governments decide not to fund the drug. Then that patient is kind of left out by himself or herself, trying to figure out a way to access that drug. It can potentially become an ethical situation.
The Chair: Thank you. You have covered a lot of material here. The latter part — who funds it or whatever — is not directly part of our direct issue here today. The issue for us is how we get the identity of drugs that can benefit a particular disease, even the possibility of Canadians gaining access to it. Who pays and whatever is an important issue, but is a subsequent technical issue. What we are concerned with is getting them approved for use in Canada through process.
We can understand why a company would find it very difficult, perhaps, or not even productive, to carry out a clinical trial in an orphan disease in Canada or some other country. We have had evidence on the difficulty identifying patients. If there are only half a dozen people in the world or in a given country, how do you carry out a trial? You have referred to that sort of thing. We can understand that part.
The issue I am drilling into here today is barriers. When trials have been carried out, such as in the United States, which is a huge market and for which there is incentive for companies to do trials on even orphan diseases, and once something is approved there, we need to identify the barriers to then bringing that into Canada. You have helped us with some of those in terms of it being at Health Canada's level with regard to their regulations in recognizing the protocols that were used elsewhere to ultimately approve that drug. You have indicated that you understand that there are discussions going on to try to harmonize some of those aspects.
Is there anything else in that area that you would like to add at this point, or have we covered it pretty much?
Ms. Gorman: I do not have anything else to add.
The Chair: Thank you. I wanted to finish off with a little bit of clarification on the rare disease area because, when you were responding to some of the broader questions — and one could interpret your comments for a larger or smaller number of people — you actually used a small example. The cystic fibrosis one was the case in point. That is the kind of thing we tend to be thinking of. In the evidence coming before us, there seem to be two clearly identifiable areas of so-called personalized medicine. One is the area that you identified. You have a small group of people who have been identified as being susceptible to a particular type of disease. It is personalized because medical science has been able to identify that they carry a particular gene, or they are clearly showing symptoms or whatever. It is a rare disorder and therefore, as you very well described — both of you dealt with this issue — it is personalized because you have to deal with that individual in their circumstance.
There is another area of personalized medicine that we have heard some evidence on that may be on the horizon or even active to this point. That is where you have a very large portion of the population that may benefit from a given drug, but there is a subset of that population that may have a serious adverse reaction. The Vioxx and Celebrex situation is a case in point. There is another example of personalized medicine where, through genetics, we may be able to predetermine, ultimately, if the evidence occurs at trial — as you so well indicated, it is important to see these adverse reactions at the trial stage — where a drug that is off the market because of adverse reaction may well be brought back to the market to benefit a wide range of people. You can identify that subset that would be adversely impacted. Those are two kinds of highly distinct aspects of personalized medicine.
Are there other areas of personalized medicine that, from a patient's perspective, either of you could identify for us this morning?
Ms. Kovacs-Burns: There is a whole new class of drugs that we are seeing with the biologics that are very specific. They are targeted, and they certainly are a protein that could be genetic. It could be a different kind of biologic. That range of product seems to be coming more and more on the market not just for rare disorders but also for a lot of other diseases as well. That is truly going to be a personalized kind of approach. Clinical trials will be so critical through the phases where they may be doing comparative studies with standard drugs but even more so in terms of Phase IV, post- market. They will not be able to determine exactly what the side effects or adverse reactions might be until 10 or 15 years later when there might be an immune response or something else happens. That is truly a personalized kind of product. Arthritis patients use biologics right now. They are a prime example of cases where some of them have been on a biologic for a while and then suddenly developed an immune response or reaction to that product. Yet, they were doing so well. That is a very fine area, one that I think is starting to increase in terms of the drug market and one that we have to be very conscientious about.
The Chair: Thank you very much. In fact, we will be looking at the post-approval surveillance as a whole phase of our study. We will probably be hearing from you again on that. You have referred to it as Phase IV of a clinical trial, so I have allowed you to continue the discussion. It is a clinical trial term. We will be looking at that area specifically, and I think that will be a very interesting phase.
Senator Callbeck: I think the question I had has been answered by Ms. Gorman, but I just want to restate it.
I take from what you say that, if a Canadian gets access to a drug through the Special Access Programme, there is no private or provincial drug plan that will cover any of that. It has to be a drug that is approved by Canada.
Ms. Gorman: For coverage, yes. My understanding is that there have been exceptional circumstances where people have been able to get the province to pay for it, but that would be an exception. If it comes through the Special Access Programme, it is up to the individual, generally, to pay for it or to get assistance through the pharmaceutical company.
Senator Callbeck: There are no private plans that even look at it.
Ms. Gorman: I am not aware of any. To my knowledge, no.
Senator Martin: I had one question for Ms. Gorman that I do not think has been asked by anyone. You made a comment regarding how the U.S. and Europe take advantage of financial incentives, which you have talked about. However, you also mentioned technological support. Could you expand on that?
Ms. Gorman: That is around the clinical protocols and trial design.
Senator Martin: I see. Thank you.
The Chair: Perhaps you could enhance that a little bit for us because it is not likely to come up elsewhere, Ms. Gorman. You are referring there to the fact that you have a very rare disease, and the expertise may be difficult to acquire or exceedingly expensive for an individual drug company or an inventor or whatever to identify and bring forward. The U.S. has a plan to assist in bringing that expertise together to help properly design a very limited trial. Is that what you are referring to?
Ms. Gorman: Exactly. It is around the capacity and design of some of the clinical trials. I know from clinicians in Canada, especially for cystic fibrosis — we are one of the bigger rare disease groups — that that is a huge issue. To have some type of expertise that they can tap into to help them, and also in terms of the trial design and recruitment and things like that, would be critical. That is one of the things that CORD is strongly recommending — to have some type of office for rare diseases to which people can reach out.
The Chair: Thank you both very much. This has been extremely helpful to us. I have no doubt that your input will be even more encouraged in subsequent phases of our study. As you may know, we will be looking at off-label use, post-approval surveillance and adverse drug reactions. I am certain those are things of considerable interest to groups that represent patients in these areas.
You have been very clear and helpful to us today. As we discussed before we came on the air, if you go away and think of particular benchmark approaches to these issues — as Senator Martin referred to — and examples of best practices or other examples that occur to you when you leave, we would welcome your following up and providing that to us. On that note, on behalf of the committee, I want to thank you and declare the meeting adjourned.
(The committee adjourned.)