THE STANDING SENATE COMMITTEE ON SOCIAL AFFAIRS, SCIENCE AND TECHNOLOGY
EVIDENCE
OTTAWA, Wednesday, October 8, 2025
The Standing Senate Committee on Social Affairs, Science and Technology met with videoconference this day at 4:15 p.m. [ET] to consider Bill S-201, An Act respecting a national framework on sickle cell disease.
Senator Flordeliz (Gigi) Osler (Deputy Chair) in the chair.
[English]
The Deputy Chair: Welcome to this meeting of the Standing Senate Committee on Social Affairs, Science and Technology. My name is Senator Flordeliz (Gigi) Osler. I’m a senator from Manitoba and the deputy chair of this committee.
Before we begin, I would like to do a round table and have senators introduce themselves.
Senator Senior: Hello, everyone. Senator Paulette Senior from Ontario.
Senator McPhedran: Welcome. Marilou McPhedran, independent senator from Manitoba.
Senator Burey: Sharon Burey from Ontario.
[Translation]
Senator Arnold: Dawn Arnold from New Brunswick.
Senator Petitclerc: Good afternoon. Thank you for joining us. Chantal Petitclerc from Quebec.
[English]
Senator Bernard: Welcome. Wanda Thomas Bernard from Mi’kma’ki, Nova Scotia.
Senator Muggli: Tracy Muggli, Treaty 6 territory, Saskatchewan.
Senator Greenwood: Margo Greenwood from the most beautiful province in all of Canada, British Columbia.
The Deputy Chair: Thank you, senators.
Today, we are continuing our study of Bill S-201, An Act respecting a national framework on sickle cell disease. Joining us today are, from the Royal College of Physicians and Surgeons of Canada, Dr. Catherine Moltzan, hematologist, Hematology Specialty Committee; from the Health Standards Organization, by video conference, Sandra Young, Executive Director, Standards and Education; and from the Canadian Hemoglobinopathy Association, Dr. Robert Klaassen, pediatric hematologist/oncologist, Fellow of the Royal College of Physicians and Surgeons of Canada, Division of Medicine; as well as Dr. Alan Tinmouth, physician and Director of the Ottawa Hospital Hemoglobinopathy Program. Thank you all for joining us today.
We will begin with Dr. Moltzan. You have five minutes. The floor is yours.
Catherine Moltzan, Hematologist, Hematology Specialty Committee, Royal College of Physicians and Surgeons of Canada: Good afternoon. Thank you for the opportunity to discuss the intersection of Bill S-201 and hematologist training in Canada. I’m a hematologist from Winnipeg. I’m also an associate professor in the Department of Internal Medicine at the University of Manitoba, and I’m here as Chair of the Hematology Specialty Committee at the Royal College of Physicians and Surgeons of Canada.
The success of this framework depends upon policy, infrastructure and the physicians who deliver care for patients with sickle cell disease, that is, hematologists. As you are probably aware, hematologists specialize in blood diseases. To become one, a medical school graduate must spend at least five to six years in postgraduate residency training, including three years in internal medicine or pediatrics and two to three years in hematology. The committee that I chair oversees the standards of training of residents in university programs who have completed internal medicine training. There is a sister committee that does the same for pediatrics training. Many hematologists pursue further fellowship training in specialized areas, especially for careers in academic university centres.
The Royal College of Physicians and Surgeons of Canada has modernized hematology education through the Competence by Design model, and the competencies, the training experiences, the entrustable professional activities and the standards of accreditation align closely with Bill S-201’s pillars. They ensure that hematology residents develop expertise in diagnosing and managing blood disorders like sickle cell disease as well as in coordinating care for these patients. This includes pathology, diagnostics, transfusion medicine, pain management, interprofessional collaboration, scholarship, patient advocacy and health system leadership.
This bill calls for national standards of care, training and data collection. The training requirements already mandate quality-improvement projects, multidisciplinary collaboration and registry-based research, which are all needed to build the evidence base envisioned in the bill. Residents must also demonstrate competencies in advocacy, equity and culturally safe communication to address the disproportionate burden of sickle cell disease in racialized communities.
The standards of accreditation require hematology training programs to reflect Canadian society’s needs and collaborate with other health professions. This means that each hematology residency program must provide exposure to community care, diverse patient populations and interprofessional teamwork, which are essential to equitable national service delivery.
In practical terms, our hematology training programs would help operationalize Bill S-201 by providing consistent training for all hematology residents in Canada; encouraging more residents to consider sickle cell disease/hemoglobinopathy as a subspecialty as we build national infrastructure; encouraging residents to consider quality-improvement projects and research in sickle cell disease to build a national database; and serving as regional knowledge-translation hubs, sharing best practices across jurisdictions.
The educational infrastructure is already in place. Canadian hematology training has evolved to produce clinicians skilled in molecular diagnostics, complex therapies, health policy implementation, research advancement and health equity advocacy. This bill would provide a national mandate, and hematology education would help to provide the workforce to fulfill it.
Thank you for recognizing the need for training excellence and national policy to improve outcomes for Canadians with sickle cell disease.
The Deputy Chair: Thank you, Dr. Moltzan.
Ms. Young, you have five minutes.
Sandra Young, Executive Director, Standards and Education, Health Standards Organization: Thank you very much and good afternoon, senators. I’m very pleased to be here. I’m joining you today from Calgary, the traditional, ancestral, unceded territory of the Indigenous Peoples who have lived in the area for generations, including the Blackfoot Confederacy, the Stoney Nakoda, the Tsuut’ina Nation and the Métis Nation of Alberta.
My role at the Health Standards Organization, or HSO, is as the Executive Director of Standards and Education. The Health Standards Organization is a health and social services standards development organization, and we are accredited by the Standards Council of Canada and the International Society for Quality in Health Care.
Evidence-informed standards are developed in collaboration with technical committees with equitable representation from across Canada, inclusive of product users, policy-makers, people with lived experience and general interest holders, namely, researchers and thought leaders.
The Health Standards Organization comes to you today to speak to Bill S-201, An Act respecting a national framework on sickle cell disease, in reference to a national framework on sickle cell disease, specifically citing the evidence-based national standards for the diagnosis and treatment of sickle cell disease, including measures to institute universal neonatal screening; postnatal diagnosis, when necessary; and the provision of results for affected individuals and organizations.
In the upcoming year, 2026-27, the Health Standards Organization plans to develop perinatal, pediatric and laboratory standards. These standards will be updated, and the pediatric standard will be a net new standard. We are very proud of the standards that we publish. We are proud of the first pediatric pain standard in the world, as a national standard of Canada, as well as the first Integrated People-Centred Health Systems, or IPCHS, standard. Currently, we are working with an Indigenous-led distinctions-based technical committee to develop the first national standard in Canada for cultural safety and humility. These standards have uptake and are implemented through Accreditation Canada. Revisions to these standards may integrate criteria requirements outlined in the bill for neonatal screening, postnatal diagnosis, laboratory testing, blood transfusions, and pediatric management of pain and sickle cell disease. This integration would then be translated into practice and continuous quality improvement through Accreditation Canada.
The Health Standards Organization welcomes the opportunity to collaborate as an interest holder in the development of a national framework on sickle cell disease. We thank you for your attention to this important issue.
The Deputy Chair: Thank you, Ms. Young.
Dr. Klaassen and Dr. Tinmouth, you each have two and a half minutes on behalf of your organization. Dr. Klaassen, the floor is yours.
Robert Klaassen, Pediatric Hematologist Oncologist, Canadian Hemoglobinopathy Association: Thank you very much. I am a pediatric hematologist and oncologist at the Children’s Hospital of Eastern Ontario, or CHEO, and it is a pleasure to be here today as a witness representing the Canadian Hemoglobinopathy Association, CanHaem, in support of Bill S-201.
I have been practising pediatric hematology for more than 25 years now, and, during that time, I have noticed an exponential increase in the number of patients affected by sickle cell disease, with the numbers increasing far above what I see in the other patient groups I take care of. For instance, when I first started, I was following about 60 patients in our clinic, and we now follow more than 300, which is a 500% increase during that period.
Sickle cell disease is an inherited condition that results in distortion of red blood cells, which is why it’s called sickle cell disease. This change results in red blood cells breaking down, causing anemia and damaging the blood vessels throughout the body. Due to the damage, even young children are predisposed to serious complications, such as stroke, eye disease, lung problems and painful damage to their bones. This is a serious life-limiting condition, and during my career, I have seen a number of children die from this disorder.
Fortunately, there has been steady improvement in care through newborn screening and early hydroxyurea use, but a significant proportion of patients still have debilitating disease. We currently only have one disease-modifying treatment that we use for this condition, called hydroxyurea, and in Canada it is prescribed off-label because it is not Health Canada–approved for sickle cell disease. While the U.S. Food and Drug Administration, or FDA, and the European Medicines Agency, or EMA, have approved it, it’s not done in Canada. They have approved two other drugs for sickle cell disease that are not approved by Health Canada.
A stem cell transplant can cure eligible patients but is limited by donor source, toxicity and a system already overloaded with cancer patients. Gene therapy has now come in and is approved by Health Canada, which occurred in September 2024.
However, only recently have we started discussions about how to roll this out. The Canadian Hemoglobinopathy Association is very concerned that access to this therapy is going to be very limited due to capacity problems among the transplant centres. We may end up with access being totally dependent upon a patient’s postal code. For example, I work in Ottawa and have patients in Quebec and Ontario, and access to drugs is very different in each province. That could be a real problem. I have a colleague who works in the U.K., and they have already been rolling this out for more than a year. Canada is significantly behind the rest of the developed world regarding this very important treatment.
Over to you, Alan.
Alan Tinmouth, Physician and Director, Ottawa Hospital Hemoglobinopathy Program, Canadian Hemoglobinopathy Association: I’m an adult hematologist at the Ottawa Hospital, and I’m the Medical Director of the Ottawa Hospital Hemoglobinopathy Program. I’m also representing CanHaem, the Canadian Hemoglobinopathy Association.
Similar to what Dr. Klaassen said, I’ve been taking care of sickle cell patients for about 20 years. We initially established a clinic about 20 years ago for 10 to 15 patients due to their complex and unique disease. We now have over 350 patients in our clinic, with 30 to 40 new consults per year. This exponential growth has been seen across Canada. Most large cities now have comprehensive care clinics, but, despite this, patients with sickle cell disease still often don’t get the care they need. We don’t have the resources to provide optimal care.
As Dr. Klaassen has highlighted, there are many short- and long-term complications related to sickle cell disease due to abnormal red blood cells and impaired oxygen flow. Patients can suffer strokes, vision loss and organ failure, including the brain, the heart, the liver and the kidneys. Often, these occur in patients in their twenties and thirties. Patients have severely shortened life expectancies and markedly decreased quality of life.
When we started our clinic 20 years ago, we really had three options that we could provide for patients: hydroxyurea, blood transfusions and pain medications. Twenty years later, we really only have the same options: hydroxyurea, blood transfusions and pain medications. We haven’t seen similar advances in sickle cell disease that we’ve seen in other patient groups with chronic medical conditions.
Additionally, patients with sickle cell disease face systemic biases and barriers that prevent them from accessing care and receiving the appropriate treatment and supports that they require and need. This results in our patients having regular — often daily — pain, multiple hospitalizations per year and challenges completing school or receiving the work accommodations that they need.
There is hope, as Dr. Klaassen mentioned. Curative therapies do exist. Stem cell transplantation and gene therapy are licensed under Health Canada, but access to these novel therapies is limited. In Canada, only a handful of patients with sickle cell disease have received a stem cell transplant, and no one has received gene therapy outside of a clinical trial.
Bill S-201 represents a historic opportunity to establish a coordinated, national framework for sickle cell disease in Canada, and it’s greatly needed. CanHaem strongly supports this bill, and we stand ready and look forward to collaborating with members of the Senate, Parliament, Health Canada, provincial ministries and patient communities to ensure that the framework delivers on its promise of equitable, evidence-based and transformative care for Canadians with sickle cell disease. Thank you.
The Deputy Chair: Thank you very much, doctors. We will now proceed to questions from committee members. For this panel, senators, you will have five minutes for your question, and that includes the answer from the witnesses. Please indicate if your question is directed to a particular witness or to all witnesses.
Senator McPhedran: Thank you, all of you, for being here in person and on screen.
One of the points that I’ve heard in relation to the most recent treatments is that the circumstances seem a little unclear as to why, when it’s approved by Health Canada, patients aren’t able to receive gene therapy, for example. I wonder if you could talk to us a little bit about what’s underneath that statement.
Dr. Klaassen: I could just say that gene therapy has to be administered in a specialized unit. We’ve established that it should be in a bone marrow transplant centre. The unfortunate situation is that bone marrow transplant centres are all overwhelmed. As I said, we compete with cancer patients in particular, who often need bone marrow transplants, and also other conditions. They tend to get prioritized over the patients with hemoglobinopathy.
Dr. Tinmouth: That’s definitely one of the key aspects — limited centres that can provide it. The second factor is cost. Gene therapy has been approved in Canada since September of last year. At this point, it’s still being considered by the different provinces how they’re going to fund this therapy, which is very expensive but, at the same time, curative. Really, as one of my colleagues describes, it demedicalizes patients’ lives. It takes them from being in hospital regularly for pain episodes or transfusions, and it basically removes them from hospital and puts them back to having a normal life.
Senator McPhedran: Are you aware of any private insurance schemes that could potentially be helpful in accessing this treatment?
Dr. Tinmouth: I think private insurance schemes are very challenging given the cost and our patient population. As highlighted by Dr. Moltzan, it’s a disadvantaged, racialized community. Most of the patients in our clinic don’t have private health insurance or family doctors. It would be very challenging to have private groups funded.
The other factor is that within the Canadian system, the model of care is this is primarily an in-hospital therapy that’s being delivered as a bone marrow transplant. They’re admitted into hospital, get high-dose chemotherapy and then receive the treatment. Most of that cost is borne by the hospital, and private insurers wouldn’t be paying for patients who are covered under provincial health care.
Senator McPhedran: Thank you very much.
Senator Petitclerc: Thank you for being here. I have one more question following up on Senator McPhedran, and then I have my own questions.
Just to wrap up on access to new treatment and options that exist and are competing and the cost, what do you believe would be the role of framework legislation like this one to help bring sickle cell disease a little higher up on the priority list? Is this the sort of legislation that can help?
Dr. Klaassen: I certainly think so in that, right now, it’s a bit of a competition as to who gets a bed for a bone marrow transplant, for instance. We need to have dedicated beds and dedicated infrastructure for patients with sickle cell disease. I also take care of a group with thalassemia who also benefits from this treatment. We need to have dedicated beds for patients with sickle cell disease and thalassemia that are protected so that they’re not bumped or pushed out of the way by patients who obviously have needs, but I think we need to make sure that these patients have access as well.
Dr. Tinmouth: I think even the point around the other therapies that we have, most of the time the therapies don’t come forward for approval by Health Canada unless they’re brought forward by the companies. I think that a national framework would ensure that these do come forward when they’re available therapies, or it should be built into it. Otherwise, without that, it becomes like the case of hydroxyurea; it has been our primary treatment for 20 years, and it’s still not a licensed indication, which means some patients can’t get it covered under insurance.
I think there are things that a national framework could do to ensure that the therapies are being delivered from that point of view. One of the things we haven’t spoken about is that there is universal access to patients and not just in very specialized academic centres as well.
Senator Petitclerc: I want to take you in a bit of a different direction to get your expertise on the specific impact of this disease on individuals. Clearly, you have that expertise.
We’ve talked about this being a rare disease. You’ve talked about it as a life-limiting condition. One of the things that came up — it’s in the legislation, but the sponsor emphasized it — is that the challenge is that sickle cell disease is not recognized as a disability for some reason. We’re not talking about treatment but access to services, access to support, accommodation for students, for example, and access to the Disability Tax Credit.
From a medical standpoint, does sickle cell disease qualify as a disability?
Dr. Klaassen: I’ll talk from the pediatric perspective, and I’ll let Alan Tinmouth talk from the adult perspective.
There’s a spectrum of the disease. Actually, because of newborn screening and early administration of hydroxyurea, I have a number of patients who do quite well. They’re taking a medication every day and, fortunately, they can go for years without problems.
But there’s a significant proportion of patients who have, for instance, what’s called avascular necrosis. We have a teenager who has no blood supply to their hip, so they’re hobbling around like an elderly person because, basically, the blood supply to their hip has been affected. They definitely have a disability. There’s a big spectrum, and it would make things much clearer.
For instance, in Quebec, if you have sickle cell disease, it is eligible for the Disability Tax Credit. It’s not eligible in Ontario, which is kind of crazy. Again, this disparity between provinces is quite dramatic.
Dr. Tinmouth: The only thing I would add to that is that every week in our clinic, we see patients who are struggling in some way because of difficulty maintaining employment or struggling with school. While I probably couldn’t quote to you a definition of disability in my mind, out of all the patients I see, they qualify. They need support and help with this condition, which is lifelong and one they were born with. I can’t see how it isn’t a disability, and it needs to be recognized as such.
Senator Bernard: Thank you all for your testimony today. I appreciate it. I have a couple of questions, first to Dr. Klaassen and Dr. Tinmouth. What accounts for that significant increase in the number of patients you’re seeing in this 20-year period?
Dr. Klaassen: It’s certainly very clear that it’s from immigration patterns. We frequently have patients who recently immigrated to Canada whom we are seeing. Even more of a challenge is that those patients have often been inadequately treated because of resources in the country they came from.
As I say, with Canadians who are diagnosed in the newborn period and started on treatment, they tend to do well. The patients who are most affected by sickle cell disease in my population in particular are the newer immigrants who have gone through their whole life and have not been properly treated, have many of the complications and are struggling. Added into that, they often have many difficulties with social supports. They may not have housing and the support to back them up, and they don’t have the funds. For instance, getting access to hydroxyurea, which is a relatively cheap medication, could be a barrier to them.
Dr. Tinmouth: I don’t really have anything to add. In our case, we see patients coming from CHEO to the Ottawa Hospital, but it probably represents a third of our patients that we see, and most of them are immigrants who have come into Canada.
Senator Bernard: My next question is for Ms. Young. Is newborn screening now a national standard? Is that happening across the country?
Ms. Young: It is happening across the country, but it is currently not a national standard of Canada under HSO. Newborn screening is definitely described in the perinatal standard, but there are other requirements at the provincial level with regulation and standards for neonatal screening. But from an HSO standpoint, neonatal screening is not currently a national standard for Canada.
Senator Bernard: You both mentioned barriers and systemic biases. I’d like you to drill down a bit on that, please. I’d like to ask specifically what role, if any, do you think that race and racism may play in these systemic biases and barriers, and what are you seeing and hearing from patients who come to see you in your clinics?
Dr. Tinmouth: There is no question that biases and race play a significant role. Sickle cell disease predominantly affects individuals who are from an African background, also from the Middle East and other areas. When they present, they present with episodes of severe pain, and they present to emergency departments. They are usually presenting because the pain medication they have at home, very strong medication, isn’t working. So they’re coming in asking for IV narcotic medication because it’s the only thing that works for their pain and the only thing we have to offer them.
Most of the time, if you have a patient who knows what medication works, how often they need it, is paying attention to when their next dose is scheduled and they’re asking for it, you think that’s a great patient. In the case of sickle cell disease, they’re often thought of as a drug seeker, and this is a case where we don’t have other medications to offer. This is the most cogent example I can think of as to how it affects us. We know this is a racialized group that it affects, and it just multiplies the effect of a terrible chronic illness.
As Dr. Klaassen says, most of the patients who start on hydroxyurea do well when they’re young. When I see them as adults, they are still struggling with a lot of issues and problems as well. It’s not something that if we just treat them young, then we get ahead of it. We are controlling it at best.
Dr. Klaassen: One of the unintended side effects of this opioid crisis is that there has been the message that opioids are horrible, terrible things, and you should not prescribe them to patients. You should avoid them because you will turn patients into addicts. That is the treatment for pain in sickle cell disease. The hard message that we have to try to get to emergency doctors, family doctors is that in this particular condition, opioids are the right treatment, so they should not be holding back because of all the other messaging they’ve been getting.
Dr. Moltzan: From an education standpoint, it’s really important that not just hematology specialists but different types of physicians who may interact with these patients actually do receive appropriate education about pain management in sickle cell disease. This framework would help raise that profile.
A side effect of the opioid crisis as well is that physicians are almost afraid to prescribe narcotics to these patients, even though it is very necessary. I think anything we can do to heighten the awareness of the needs of these patients would be very important.
Senator Burey: Thank you for your passionate testimony and for appearing before our committee today.
I want to drill down on the training, on the standards, because we’ve heard from so many people with lived experience, families, that when they ask — just like you said, Dr. Klaassen — people think they are drug seeking. They don’t know about sickle cell disease.
Given the mandate of the Royal College, and I know that you’re a hematologist, and this is just going back to the piece about education and the fact that you need to have — I’m not going to read what your website says — training, how does the Royal College of Physicians and Surgeons of Canada plan to integrate sickle cell disease into its training standards, not just for hematologists but for every doctor, every health care provider in your purview and in continued education?
That’s the first part of my question. I think you answered the second, which is whether this framework will help that.
I want us to go back and see. You’ve been given that mandate to train. What have the barriers been that have prevented it from disseminating to health care providers?
Dr. Moltzan: That’s a very good question that doesn’t have an easy answer. What I would say is that in terms of the barriers for other physician groups, other than the hematologists, I think it really does come down mostly to appropriate pain management education. The reason why I say that is when patients present to the emergency room, as has already been elucidated by my colleagues, that’s usually what they present with.
The increased awareness of this group of patients and their need for very specialized pain management and probably, as well, early consultation with specialists — not just hematologists but also pain management specialists — are needed to actually make sure that these patients get the care that they need.
To the earlier point of my colleagues, this is the one situation where narcotics are the treatment of choice.
Senator Burey: Just building on that, would it be the Royal College saying, “You need to spend X number of hours on sickle cell and pain management”?
Dr. Moltzan: It’s challenging, but I would say, for specific physician groups, certainly time spent learning about pain management in this group of patients would be very important.
For example, I’m thinking of emergency physicians, ICU physicians at times, anaesthesia physicians, all of whom would fall under the purview of the Royal College. In fairness, family physicians are under the College of Family Physicians of Canada, but I would argue that appropriate pain management education — and although, as an overall message, I think the message to avoid narcotics for most types of pain is appropriate, given the crisis we’re in, for this particular group of patients, it’s not the right way to go.
Senator Burey: This is for the oncologists and hematologists. What effects do blood donation restrictions have on blood transfusions and not having enough blood for transfusions as well as for stem cell transplants and that sort of thing — so the restrictions on blood transfusions?
The Deputy Chair: Can I put you on the second round? I think that’s an important answer we would want to leave time for. Hold your answer, please; we will put you on the second round.
Senator Muggli: Thanks for being with us today. I’d like to direct my question to Ms. Young. Others may answer, too, if they have thoughts on it.
Ms. Young, you mentioned standards for pediatric pain management. What drove that, and what drives the development of other standards? Was there a framework bill related to that or other standards? It just made me curious: Have there been attempts to create national standards for sickle cell disease, and if not, why not?
Do you have any thoughts on why we need a framework bill to drive the creation of standards for this condition?
Ms. Young: Thank you very much, senator, for the question.
Standards are definitely levers of change. The advantage to having a national standard is that it sets the requirements across Canada. As I said, they come to life through Accreditation Canada, and Accreditation Canada is in every hospital in Canada. So that would be the impact of a national standard of Canada.
The Pediatric Pain Management National Standard of Canada came forward from the strong work of Solutions for Kids in Pain, or SKIP, and their partnership with Health Canada. When there is a requirement for a national standard of Canada, we are very responsive when national groups, clients, patients and policy-makers come to us with requests for national standards of Canada, and we prioritize those accordingly.
The pediatric pain standard, as I said, was the first in the world, and we’re actually embarking on work regarding an adult pain standard right now, in partnership with McMaster University.
Senator Muggli: Have you had people coming forward asking for standards on sickle cell disease? Has there been a push, and if there has been — obviously, you have to prioritize — is there a reason this condition would not have been prioritized?
Ms. Young: It hasn’t been brought forward, to my knowledge. I made sure to check that before I presented today. However, certainly, many of the requirements that are outlined in the national framework and in the evidence-based national standards in the bill would be easily integrated into the standards that we’re updating in 2026 and 2027.
We haven’t been asked for a specific one, and we don’t usually write a standard that is specific to a disease, but that’s not to say that neonatal screening, which is covered under ISO 15189, or a specific disease would not gain attention. Generally, we look at service standards, like palliative care, primary care, et cetera, and would integrate the needs of sickle cell disease and pain management into standards related to perinatal care, pediatric care and laboratory care.
Dr. Klaassen: I will add one thing, if I may. The Canadian Hemoglobinopathy Association has put together standards for the management of sickle cell disease, but it’s very specific to the subspecialty of hematologists. It’s not designed for emergency doctors or family doctors. I’ll just mention that.
Senator Muggli: Thank you.
Senator Senior: I think it was you, Dr. Klaassen, who mentioned that 25 years ago you were planning a particular approach to patients with sickle cell disease, and 25 years later, you’re doing the same thing; is that correct? Or was it you, Dr. Tinmouth? Okay. It was a statement I remember. Clearly, I’m curious about that because, as was mentioned by others and, I think, by you, there have been developments in other parts of the world but not in Canada. Can you talk about that?
Dr. Tinmouth: It is very true. Dr. Klaassen mentioned we only have one therapy that we use in Canada, which is hydroxyurea. We have had that for 20 years. There have been a number of other medications that have been licensed in Europe, as Dr. Klaassen mentioned, or by the FDA. Those have not received Health Canada approval. I’m not sure if that’s because they weren’t submitted by the drug companies to receive it or there were other issues with the submissions to Health Canada. Therefore, in many ways, we haven’t been able to offer those additional therapies that can help to reduce the number of pain crises and to increase hemoglobin to our patients.
So, in many ways, Canadian patients with sickle cell disease have not had the same access to the same care and treatments that are available in other countries that have similar health care systems to us or are offered similar systems. That has been a great loss.
We are lucky with the curative therapies available, particularly the gene therapy; that is something that is approved and licensed by Health Canada — at least one of the gene therapies — but it’s not something that is currently being offered to Canadians. It’s currently being offered, as Dr. Klaassen mentioned, to patients in the United Kingdom and the United States. Even now, when we have them licensed, we still haven’t had them, and there are delays to getting access to those therapies. We’re hopeful they will come, but there are definitely delays.
Senator Senior: Is there a particular lever in the framework that you think can bring that about?
Dr. Tinmouth: That’s a good question. I’m not sure what the lever would be. In my mind, the framework does ensure that we would be providing care to the patients, but what the specific lever would be within that — I must admit I’ve never seen a national framework for a disease in place, to my knowledge, so I don’t know how it would work. At the same time, I know that what we have had for the last 20 years hasn’t allowed those therapies to come to the front. I think there need to be additional frameworks put in place that allow us to raise the bar of care for our patients.
Dr. Klaassen: I think gene therapy, in particular, is a really good model for this because not every place in Canada can do gene therapy; it is a fairly specialized thing. There needs to be a national framework for this to work, or else patients in different provinces will not have access to this very important treatment.
It’s even more specialized than bone marrow transplants. We’re building upon the bone marrow transplant network because they have the expertise and some of the infrastructure, but it’s even more specialized than that. So I do think a national framework would be very important in this particular instance.
Dr. Moltzan: From an educational standpoint, it’s heightened awareness of the severity of the disease, how many patients are affected by the disease and the need for these therapies. I say that both from an educational standpoint and from a clinician’s standpoint.
Senator Senior: Ms. Young, I was quite surprised that we haven’t heard from the sickle cell community or associations with regard to their concerns around the lack of treatments or standards. That might be for the second round, but I just wanted to plant that question.
The Deputy Chair: Ms. Young, may I suggest we leave it for the second round? It will give you some time to think of an answer.
Ms. Young: Okay. Thank you.
Senator Greenwood: The two questions I had were asked, so I’m going to ad lib here.
I don’t know much about sickle cell disease. Let me say that. I’ve learned a lot, but I wouldn’t say I know. But I did hear one of the comments earlier about there being a related blood disease to sickle cell, thalassemia. I’m curious: If we were doing this for sickle cell disease, would it include thalassemia? Is it the same treatment for those patients as it is for sickle cell? So then if I looked at this framework, and if I were a physician or somebody, I could say, “Well, actually, that’s included.” I don’t know. Can you talk a little bit about the relationship there?
Dr. Klaassen: I would say that CASGEVY, the gene therapy, is approved for both sickle cell disease and thalassemia. I would say they are quite different conditions and they have different treatments, but in this particular instance, the gene therapy is equally effective in both. They just came out at the same time. It’s fairly unique, the gene therapy.
Dr. Tinmouth: Speaking to the bill, as it is titled “National Framework on Sickle Cell Disease Act,” thalassemia is a hemoglobinopathy, and sickle cell disease is a hemoglobinopathy, but thalassemia is not encompassed within sickle cell disease.
There is certainly a need for the treatment, as Dr. Klaassen said, with gene therapy for that population, but they have a very different set of complications. They’re not routinely hospitalized. They do have decreased life expectancy, but they don’t have severe episodes of pain. It’s a very different disease. It just happens to be that the curative therapy is the same.
Senator Greenwood: Thank you for that.
I have a question for the HSO and the Royal College. This is around discrimination and racism. I’m wondering how the work you do in training and education and in setting standards can combat discrimination, racism, bias and those sorts of things. How do you see it?
Dr. Moltzan: What I would say from an educational standpoint is that I think in all residency programs there certainly is a lot of attention being paid to diversity, equity and inclusion. That’s not only for this patient population but also for Indigenous patients and patients of other marginalized communities.
Certainly, the standards of training that we have in the entrustable professional activities do reflect that. I think in many training programs, there is certainly attention being paid to culturally appropriate communication. I know that doesn’t necessarily specifically speak to sickle cell disease, but I think that there certainly is attention being paid to individuals and patients who are in marginalized groups. That would include patients with sickle cell disease.
Senator Greenwood: I think you made a comment earlier about how the more we educate those who are treating this and recognize it in emergency rooms, it is a strategy in and of itself of combatting discrimination.
Ms. Young, could you comment on that, please?
Ms. Young: Absolutely, Senator Greenwood. It would be my pleasure.
Throughout all of our standards, we integrate equity-based principles. In fact, with the new, sixth edition of the requirements that accredit us — we too get accredited from the International Society for Quality in Health Care — we’re required to have equity-based principles integrated throughout every standard.
We also have anti-racism and anti-Indigenous racism criteria throughout our governance of leadership standards. We are in the process of developing a cultural safety and humility national standard of Canada that also addresses cultural safety and humility and equity-based principles. Cultural safety and humility are also integrated throughout all of our standards.
The Deputy Chair: Senators, that concludes the first round. We have four senators on for a second round of questions.
Senators, for this round, you have three minutes for the question and the answer.
Senator McPhedran: In previous testimony, we received some reference to some problems for patients in terms of preserving their fertility and the impact of this from a young age on.
I wonder if you could tell us a bit more about what damage, if any, to reproductive functions the existing care and, when possible, the gene therapy and other care may be causing.
Dr. Klaassen: I can speak a little bit to that.
The one medication we have to treat sickle cell disease, for instance, we know that it decreases sperm count in males. Fortunately, it seems that when they get to the age where they want to reproduce — and I’ll let Alan talk to that because he deals with that side of it — if they stop the hydroxyurea, then the sperm count comes back up. Unfortunately, that means that because they’re stopping hydroxyurea, they’re at risk of having complications from the disease. It is a bit of a problem. I would just say that for the hydroxyurea.
Gene therapy and bone marrow transplant can definitely impact fertility. I think this is really important because getting access to fertility preservation is a real problem, and it needs to be addressed.
With males, it’s relatively simple because you do sperm collection, and you can freeze it. For females, it’s much more complicated because egg preservation can be very challenging and is very expensive, and it’s not supported. My friend in the United Kingdom says it’s not a problem. They actually pay for fertility preservation for their patients going for gene therapy. I have a lot of conversations with him, and I’m very surprised at how far we are behind the national health system in the U.K.
Dr. Tinmouth: Just to echo, obviously, hydroxyurea, while patients are on it, may affect fertility. Yet, if they come off it, they may start having more pain crises, so they are really left in a bind. That’s a very difficult situation. It speaks to, potentially, the need for additional therapies from that point of view.
There is even some question about longer-term effects. I don’t think it’s completely clear that there aren’t longer-term effects from hydroxyurea around fertility.
Just to re-emphasize what Dr. Klaassen said, both stem cell transplant and gene therapy involve patients getting very large doses of chemotherapy, and, essentially, without either harvesting eggs or sperm collection, individuals may not be able to have children afterwards.
Senator Bernard: This question is for Dr. Moltzan.
You mentioned interprofessional teamwork, and I wonder if you can tell us a bit more about that and who is included in the interprofessional training that’s offered.
Dr. Moltzan: What I was referring to in my statement about interprofessional education is that it’s not just the education of hematologists, but it’s also the education of and the interactions with the teams that we work with. With the complexity of the care of this patient group, it can’t just be a group of specialists. There need to be nurses, sometimes orthopedic surgeons, transfusion medicine specialists, pain management specialists.
I think there is an analogy here to cancer care in the sense that — in my mind, at least, and when I’m training residents — we should be thinking in terms of multidisciplinary care, not only different specialties within medicine, but some of these other professional groups, particularly nursing.
Senator Bernard: You might include other allied health professionals as well?
Dr. Moltzan: Yes, and sometimes professionals like social workers would also have a very important role. Hopefully, as treatment options increase for these patients, pharmacists and other professionals will become increasingly needed in this patient population.
Senator Bernard: Would this framework help to create the standards for that to happen across the country?
Dr. Moltzan: Yes, I would agree.
Senator Bernard: Thank you.
Senator Burey: I will reread my question. This was the impact of blood donation restrictions on blood transfusions and, perhaps, matches for bone marrow transplants relating to the malaria exclusion. In the African-Canadian community, many have been exposed to malaria, have gone to endemic countries and are not able to donate blood. What is the impact of that?
Dr. Tinmouth: As part of the work that I do, I do research in transfusion medicine and clinical research, so it’s very close to my heart.
Transfusion therapy is a life-saving therapy in patients with sickle cell disease. They can present acutely ill, with respiratory failure, with multi-organ failure, and the treatment for them is to give them what we call an exchanged blood transfusion. We remove probably 70% of their red blood cells and replace them with red blood cells from donors.
As you said, the problem is that most of our donors are White, and patients with sickle cell disease are not. They have different markers on their red blood cells, and they can develop antibodies or proteins that react against those markers that they are being exposed to that aren’t as frequent on their blood cells. So there is no question that having increased representation from the diaspora, as you say, in blood donors will improve the availability of that blood.
The malaria is a difficult question, but it’s one we need to answer. There isn’t currently a licensed test, to my understanding, for blood operators such as Canadian Blood Services or Héma-Québec to be able to test for malaria, because that’s a potentially life-threatening transfusion reaction. If we transmit malaria to a patient, that could actually kill them if it’s in the blood, and that’s the concern.
We need the ability to test that blood, and I think a national framework will provide the leverage, the tools to ensure we get that testing and that we drive forward that we’re getting the testing to improve the availability of blood for patients with sickle cell disease.
Senator Burey: I’ll just go to the research, then. I was just reading an article on indicators of inequity in research. Can you comment on funding for sickle cell disease compared to cystic fibrosis and hemophilia? Can you comment on funding research in Canada for sickle cell disease?
Dr. Tinmouth: We participate as part of the Canadian Hemoglobinopathy Association. One of our desires is to participate in more research. I would say in terms of national funding through agencies like the Canadian Institutes of Health Research, or CIHR, we have had very few projects that have been funded. Granted, these are competitive and need to be submitted by scientists and researchers, but I think there’s something where there needs to be an emphasis and an opportunity to specifically fund this patient group and calls to go out for those, as there are in other groups. It’s definitely something that has not benefited from research over the years, even within Canada and outside of Canada, to advance care of patients with sickle cell disease.
Dr. Klaassen: It is very frustrating that CIHR, in particular, will have calls out for very specific things, but blood disorders are not part of that. We’re being basically ignored by the research community. I strongly support what you’re getting at, yes.
Senator Senior: Ms. Young, just for context, I have chaired a couple of hospital boards and been part of the preparation for accreditation. What a time!
I’m curious what opportunities there would be for you to hear from the public or associations like the sickle cell associations that are formed nationally and regionally in part of that process.
Ms. Young: Yes, we’re always open to that process. I would just like to clarify that the national associations and the professional associations would be the experts in writing clinical practice guidelines, whereas the standards for health and social services for quality and safety, as you experienced, are implemented around programs or services in hospital, long-term care communities and primary care. At any time, we would be most open to being approached, and we also try to be advocating and present on a national forum with many of our pan-Canadian organizations and work closely with the Children’s Healthcare Canada association as well.
So we’re more than open, always open to feedback on where we should be developing standards. Generally, we haven’t written standards that are disease-specific but would write standards that support neonatal screening, postnatal diagnosis, laboratory testing and pediatric management of pain, because those are more program- and service-based.
Senator Senior: Is it an issue of access? I hear that you’d be open, but are communities able to actually access, to basically discuss or register their concerns regarding being heard?
Ms. Young: Yes. We have mechanisms on our website. Certainly, requests come to me and come through our executive and come through our teams, requests for standards from associations across the country. We certainly take the feedback that perhaps we should look at making that more known.
The Deputy Chair: Senators, this brings us to the end of today’s panel. I’d like to thank Dr. Moltzan, Dr. Klaassen, Ms. Young and Dr. Tinmouth for their testimony today.
Colleagues, this meeting concludes the witness testimony that we had on the work plan for this bill. The next meeting where we will have this bill on the agenda will be the clause-by-clause consideration of the bill. This meeting is planned for Wednesday, October 22. However, it remains to be confirmed, as the committee may be referred other matters which we may need to prioritize.
A couple of reminders regarding clause-by-clause meetings: Members who wish to propose amendments during clause-by-clause consideration of the bill should reach out to the Office of the Law Clerk and Parliamentary Counsel to ensure amendments are drafted in the proper format and in both official languages. Those consultations should begin as soon as possible to allow sufficient time for the drafting of amendments.
After clause-by-clause consideration, the committee may wish to append observations to the report. Observations should be short and in both official languages. The Library of Parliament analysts may provide you or your staff with support in preparing draft observations for the committee’s consideration.
(The committee adjourned.)