Health Committee on June 3rd, 2010

[The Vice-Chair, Liberal, Joyce Murray]

Thank you.

Ms. Ouellette and Ms. Coleman want to respond to Dr. Bennett's question. Then we'll go back to our list of speakers.

[Gail Ouellette]

The story of neuroendocrine tumours remains a story, and there are many like that in Canada. Unfortunately, it is often dealt with case by case. Fabry disease is another case that was dealt with to some extent. Often, the case was handled because parents or adults went to the media and managed to obtain a positive answer. But it is unacceptable that some people, in addition to being sick, have to go to the media to demand access to good medical care and treatments. There are many similar examples. Inequities happen all across Canada because each province offers different solutions.

For example, myosin is used to treat Crohn's disease. In our province, adults don't have access to it, but in our neighbouring province, in Ontario, adults can at least try this drug to see if it is effective, and can have an assessment done.

I could list a number of inequities like that. That is why we need a regulatory framework to standardize what happens in Canada with orphan diseases. The regulatory framework should address two issues: the development of drugs and access to them.

We know that drug benefits are under provincial jurisdiction. But, in terms of assessment, why redo the assessment of a rare drug, an orphan drug, in each province, when it is already something difficult to do? There must be a special way to review it. And that is what is going on at the moment.

We know that Quebec has excluded itself from drug review in general, but for orphan drugs, I believe in the need for a framework so that resources can be used better. We could do standardized assessments. We know that each province decides on the reimbursement, but at least the assessment would be properly done for orphan drugs, which are not the same as drugs for common diseases.

There is also the issue of developing drugs. I would like to give a positive example of an initiative that we could take more often in our country. In Quebec, a patient organization was able to have a company from California come to do a clinical study. Those people succeeded in turning the Montreal Children's Hospital into a site for international study. It is not an easy thing to do, but they managed because of those few patients with Morquio syndrome, who are actually more numerous in Quebec. In fact, that allows Canada to have expertise in a disease. That hospital could become a specialized clinic for that syndrome. It could become a site for phase 3 clinical trials for that drug. That is very beneficial for the patients, obviously, but also for drug research and development.

But, even if that drug has phase 3 trials in Montreal, we are afraid that we might be facing the same obstacles in terms of approval and access in Canada. There is also the question of quid pro quo in the research. Patients give their time and they travel to participate in the study. So, if they can't even have access to the drugs after that, it would be a real shame.

[The Vice-Chair, Liberal, Joyce Murray]

Thank you, Ms. Ouellette.

Ms. Coleman.

[Maureen Coleman]

Speaking as a patient, I want you to know that patients in Canada and around the world are connected very much by the Internet, and through the Internet we read about clinical trials in other countries. We read about approved procedures in other countries. So we read about how successful certain isotope treatments are, we read the abstracts. Our support network throughout Canada...it's not so much that we're doing things like yoga. We don't really have the luxury of just being stroked. We are out there hunting for research, reading research, learning about research, and then we discover that what's happening over here is absolutely wonderful. What's happening in Sweden or Germany with a certain type of scan, say, the gallium scan, is wonderful.

In my case, I'm a patient with what's known as an undiscovered primary tumour. I was discovered with distant metastasis 10 years ago. I go through imaging. When I go through imaging, the imaging is okay, but it's not that great. It can't find my primary tumour. It might find 80% of primary tumours. If I lived in Sweden, it would find 99%; it most likely would find my primary tumour. The current thinking is that it's good to have your primary tumour removed. It offers a better prognosis for the future, but if you can't find the primary tumour, then that chance of having an improved prognosis is much reduced. I'm just basically walking around thinking, so far, so good, but wouldn't it be nice to know some of the uncertainty could be taken out of that?

Meanwhile, our community has created a worldwide network. I'm on a steering committee called the Worldwide NET Cancer Awareness Day Steering Committee, and we have representatives from about 15 countries on that committee. We met in Berlin, we're meeting in New York, and we're going to have Worldwide NET Cancer Awareness Day on November 10.

A website is going to be unveiled next month with basically information from patients around the world, so we will be able to read what is happening in every country with regard to this cancer and how patients are faring and what's happening in the medical world, what developments are occurring.

Our particular neuroendocrine site will be connected to that and discussions will be going across borders all the time. At my last meeting I sat next to the representative from Poland, so I learned about what was available in Poland, first-hand.

[The Vice-Chair, Liberal, Joyce Murray]

Are there any thoughts about what could be in the letter to the minister?

[Maureen Coleman]

The key thoughts about what could be in the letter to the minister are this. Could we please relax the regulatory framework so that we can adopt some of those proven techniques, particularly the ones in Europe, maybe even in India? But we're close to Europe, so let's focus on Europe. Some of the isotope treatments that are used in Europe, the yttrium and the lutetium, and some of the scans, like the gallium scan, are so effective. Could we please look at them and adopt them, because they've worked year in and year out for many patients now without serious problems at all?

Thank you.

[The Vice-Chair, Liberal, Joyce Murray]

Ms. Leslie, back to you.

[Megan Leslie, NDP, Halifax, NS]

Thanks, Madam Chair.

I really appreciate this discussion. I'm wondering if you can help me understand what we can do with regard to diagnosis. Are there policy responses or regulatory responses to enhance, to improve diagnosis, or does it logically follow that if we can approve treatment, diagnosis will become better?

[The Vice-Chair, Liberal, Joyce Murray]

Ms. Wong-Rieger.

[Dr. Durhane Wong-Rieger]

Again, I think those are excellent concerns. If you look again in terms of what's happening internationally, I think it provides a lot of good commentary. As Maureen says, most of these are rare disorders, so you do have international expertise. You can't just rely on what's available in Canada. You do have to be connected internationally. The patients are connected internationally, and many of our clinicians, if they belong to international associations, are connected internationally. But our regulatory bodies and our approval bodies have also got to be connected internationally. It goes back to what Dr. Bennett is saying. We know we already have frameworks for approval in other countries. We've been talking about harmonization in terms of regulatory framework, so I wouldn't necessarily say we want to relax the regulatory framework--I understand what you mean--but I think what we want to do is to modernize it, and we want to be able to harmonize it so that we can take advantage of what is happening in other countries.

[Megan Leslie, NDP, Halifax, NS]

Harmonize internationally?

[Dr. Durhane Wong-Rieger]

Harmonize internationally; use international standards. We don't even have a definition that is harmonized. The diagnosis, then again, sometimes depends on having the knowledge and being able to get the right experts. What happens in many other countries is they have these centres of reference where if physicians suspect a disorder that might be of a particular type, they can actually send the tests and information, and sometimes the patient, to a centre to be diagnosed.

Many of our patients, fortunately, go to the NIH and they can get it, or they go to one of the centres in the U.S. Again, this is not acceptable for Canadians to just have that; we should have centres that are connected to centres that are international centres. So you have centres of reference where you've got experts, and they are virtual centres; it doesn't mean you have to build new infrastructure there.

The other thing that can happen in terms of diagnosis is, as you say, when you have treatments; that is in fact when you do stimulate a lot of diagnosis. There are lots of reasons--more is known about the disorder; there are more incentives for people to get tested for that particular disorder. Again, we need to be connected internationally, though, because as treatments become available, we do a better job in terms of diagnosis and we learn more about the disease.

The other thing we are doing a poor job of in this country--and we don't want to keep talking about the poorness of it--is newborn screening. I think as everybody has said, many of these disorders are genetic. In the U.S., even the most impoverished states will test for at least 50 newborn diseases with one single blood drop. Internationally, newborn screening is becoming one of the most important ways of identifying genetic disorders, many of which, in fact, if they were caught at birth, could be treated from birth without the devastating effects. Everybody knows about PKU, phenylketonuria, as one of the world's profound examples. We have many other disorders of that nature, which we do not test for. Except for Saskatchewan and Ontario, most provinces are identifying fewer than 10 of these at birth, and it just takes one drop.

We need to have a national reference centre, where once you've identified that, you can get the right genetic counselling and you can get the right genetic diagnosis. Again, it means that we need to be connecting these, nationally first and then internationally. The diagnosis is oftentimes difficult, but it is in fact improving tremendously because you've got these centres of expertise.

Unfortunately, Canada doesn't take part. And we can. Europe has its arms wide open. We go over all the time. They are telling us, “Come with us.” We're an affiliate in many ways. They're waiting for us to actually provide some expertise as well. We don't want to just be beggars at the table; we also want to be there with our own expertise. We have many tremendous pockets here; we just need to do a better job of taking advantage of our expertise nationally and internationally.

[The Vice-Chair, Liberal, Joyce Murray]

Thank you.

We have two more of our guests who wanted to speak to Ms. Leslie's question.

Dr. Urbain.

[Dr. Jean-Luc Urbain]

Megan, this is also a very good question.

We love acronyms in medicine, so I'm going to use two of them.

One is PPPT, which is for predict, prevent, pinpoint, and treat. One is the predictions biomarkers, the prevention is basically genetic counselling, the pinpointing is diagnostic imaging, and the treatment speaks for itself. We do not have this in place in Canada. This is personalized medicine. We just don't have it. We are basically dealing with patients based on a very empirical model, which is that we treat symptoms. Since we don't have the means to do diagnosis, we cannot really queue related diseases.

The other thing I want to mention is in terms of the regulatory frame. Another acronym we like to use is the AAA approach. The AAA approach consists of availability, affordability, and accountability. Unless Health Canada takes that approach, I think we are going to run in circles for a long time.

To answer your question, I think there are models, but we have to put the framework on Health Canada to embark into 21st century medicine. As I said earlier, I think the processes now are very antiquated.

Thank you.

[The Vice-Chair, Liberal, Joyce Murray]

Thank you.

Ms. Ouellette, can you speak to Ms. Leslie's question?

[Gail Ouellette]

To answer Ms. Leslie's question, I would say that we should expand on the second point in the letter to the minister; this is establishing a national plan for promoting the prevention, diagnosis and care of rare diseases.

I would like to give some examples from our patient associations and from our information and support portal. Many people call us or write e-mails to us to find out who the specialists in their diseases are in Quebec and in Canada. The situation is worse for people who live outside urban areas. Those people see a family doctor or perhaps a specialist once a year, but the doctor and the specialist do not seem to have in-depth knowledge of the rare disease in question. So the patients want to know if there is a specialist in Canada for that disease.

Patients will often find specialists outside the country through the Internet. I will give you another concrete example. A mother called us because her child has a chromosomal abnormality. She lives outside an urban area. She received the diagnosis for her child. She was told that nothing could be done for this chromosomal abnormality, the symptoms would be treated and she will be sent back to her region. On her own, she found a specialist in cytogenetics in France to help her. But there are cytogeneticists in Canada and in Quebec and she communicates through e-mail with a specialist from France. She even hopes to go there. It is absurd. All because we have not put our specialists in a directory for Quebec and all the provinces in Canada. We have expertise, specialized clinics and multidisciplinary teams for some diseases. We do not have enough, but the ones we have are not even in a directory.

Doctors have a hard time finding the resources, such as the resource centre for Fragile X Syndrome in Ontario, the clinic for tuberous sclerosis in Sainte-Justine or the clinics for Marfan syndrome in several provinces. They do not even know where to look for them. In Europe, and even in the United States, directories were created by patient organizations. In Europe, there is a tool called Orphanet where all that information is recorded for each European country.

So, a national plan that includes the creation of a directory would be very beneficial for care and early diagnosis. Shared practical guides, exchange and collaboration between health professionals would provide Canadians with better care. There are few patients in each province. There can never be a specialized clinic in every province for each of the 6,000 diseases, but there could be specialized clinics at the national level.

There could be what they call in Europe cross-border care, or if not, there are technologies like telemedicine or other ways to share knowledge. That is what patients are missing in our health care system, which is largely designed for common diseases.

If we had such a plan, all Canadians could benefit from it because the delay in receiving a diagnosis would be reduced, there would not be inappropriate treatments and we would not reach a critical point in care for those patients. There could also be support and social services that could help them, which would also lighten the burden on our health care system.

Almost 3 million Canadians have rare diseases, and many have serious, debilitating and fatal illnesses. We could take some of the weight off our health care system if we had a more standardized approach. It would already be a step forward to have them recognized nationally, with a definition of rare diseases that the provinces could follow. Working effectively together, we should adopt a plan that each province could implement individually without it being considered interference in their health care system.

Thank you.

[The Vice-Chair, Liberal, Joyce Murray]

Dr. Carrie.

[Colin Carrie, Conservative, Oshawa, ON]

Thank you very much, Madam Chair.

I want to thank the guests today.

You've really opened my eyes on a few of these issues, particularly that three million Canadians suffer with these conditions. It certainly doesn't sound rare. I guess individually it's rare, but it's a significant part of our population.

I like what's been said around the table—I think Peter brought it up first—about the federal challenges on the regulatory issues.

We talked about drugs in different provinces and how it seems to be a patchwork type of thing.

In a previous parliament, one of our Liberal colleagues, Mr. Bell, had a motion. I think Madam Bennett would probably know. Some of the things we're talking about, considering internationally accepted standards and how Health Canada's work on a progressive licensing framework could provide appropriate support for the design of clinical trials for small numbers.... My understanding is that Health Canada started an initiative and began meeting with the provinces. Of course, the motion was dissolved when Parliament was dissolved, but my understanding is that the work continues.

However, I wanted to perhaps get Madam Coleman and Madam Wong-Rieger to comment. This is obviously something you've probably followed. My understanding is that some of the provinces have lost interest as well. Could you tell us about that?

Madam Wong-Rieger, you mentioned that we're close and some of the work has been done. You talked about modernizing the regulatory framework and harmonizing the regulatory framework. Where did the process end? What did you learn from it? What happened to it? Could you bring us up to date?

As Madam Bennett said, we should work on seeing what we can do to get things done. What happened there?

[Dr. Durhane Wong-Rieger]

We were certainly very pleased when Mr. Bell brought forth the motion. As many of you will know, Mr. Bell had a grandson who had a rare disorder and who unfortunately passed away from his disorder a couple of years ago, in fact, before the motion was actually introduced. He continues to speak and work in the area of rare disorders. The motion had called for a report to give us a status in terms of rare disorders in Canada. Unfortunately, when Parliament dissolved, the report was not in fact continued.

We know there is work on the regulatory front. I hope I'm not speaking out of turn or getting anybody in the bureaucracy in trouble by saying that we know work is taking place. We were very pleased because we had an opportunity to provide some input into that work.

I think if you were to ask for a status report in terms of the drafting of a regulatory framework that's taking place right now, you would be pleased, because we are very pleased. We think there are some very good people who have put together a regulatory framework in terms of what the Europeans have and what the U.S. has. It's the best of the best, I would say.

If we had it implemented today, we would be far ahead of the game. We could put ourselves in a position so that when Peter, Gail, and I go to international meetings and say we're from Canada and we deal with rare disorders, we could speak proudly. I think we sometimes duck when we're there. We honestly do.

I think it's very close. Work has been started. I think it needs some official recognition in order to move forward. There's a lot that's been done.

[Colin Carrie, Conservative, Oshawa, ON]

Could you update us on the federal, provincial, and territorial aspects of it?

[Dr. Durhane Wong-Rieger]

Yes. That's a whole different issue.

[Colin Carrie, Conservative, Oshawa, ON]

It is a different issue. It is important.

[Dr. Durhane Wong-Rieger]

The federal-provincial-territorial aspect that I think Dr. Bennett started to allude to was the whole issue around what happens once a drug has been approved, some of the collaboration around making it available to patients. As we've always said, the funding of medications and the funding of even treatments and other diagnoses are pretty much provincial jurisdictions, and I think that part has not in fact continued to progress.

I would say that, unfortunately, part of it is a debate that patients have no appetite for, and that is the question of who is going to fund it. At the end of the day, Canada is going to fund it. And as we say, whether it comes out of our left pocket or whether it comes out of our right pocket, we as patients don't really care. We really urge the feds, the provinces, and the territories to get together, because we need to have you all at the table in order to do a national plan.

We, as the Canadian Organization for Rare Disorders, have been going around the country hosting patient consultations to talk about how to get better access. We've been educating patients on what the regulatory framework is, what the funding framework is, so they can provide input. We have been very pleased that whenever we held our round tables, we've had the feds and the provinces at the table, so we know you guys can get along with each other. We do know that can happen. And we do know that at the end of the day you have a shared vision.

We want to say that we need to have you talking about, as Gail said, whether there is a review process that you folks are actually intimately part of with the Canadian Agency for Drugs and Technologies in Health. I think you fund 75% of that body. Give a nod to them to get on with coming up with a mechanism for the approval of funding for drugs for rare disorders, for which, as you said, the recommendations can be used nationally, so we don't have different provinces coming up with different solutions.

We were talking about centres of excellence and centres of expertise. Yes, you've shown that you can make those kinds of strategic plans. You've done it for cancer, you've done it for cardiovascular disease, and you've done it for diabetes. You've put together these national strategies and said you will provide the guidance, the leadership, and the support for provinces to work together collaboratively to make it happen, and to work with the institutions--

[The Vice-Chair, Liberal, Joyce Murray]

Excuse me, Ms. Wong-Rieger, I have to interrupt because we're running toward the close of the meeting.

Ms. Coleman would like to make a comment, and then we'll have time for one last question. I ask the guests to keep their responses brief for the rest of the meeting.

Thank you.

[Maureen Coleman]

I would like to endorse everything Gail has said and everything Durhane has said, because in regard to networks of expertise across the country, what we lack is physicians who are aware of what is available in Canada or anywhere in the world. If somebody goes to a GP and.... For instance, it took ten years for me to be diagnosed. It takes the average person between five and ten years to be diagnosed. You run around to all kinds of people, but they don't know where to start looking. They don't suspect it, so they can't detect it.

So definitely we should have some kind of registry of physicians, and the existence of centres, the creation of specialized centres as well in Canada to treat our particular cancer is absolutely imperative.