THE STANDING SENATE COMMITTEE ON SOCIAL AFFAIRS, SCIENCE AND TECHNOLOGY
EVIDENCE
OTTAWA, Wednesday, October 1, 2025
The Standing Senate Committee on Social Affairs, Science and Technology met this day at 4:15 p.m. [ET] to Bill S-201, An Act respecting a national framework on sickle cell disease.
Senator Rosemary Moodie (Chair) in the chair.
[English]
The Chair: Welcome to this meeting of the Standing Senate Committee on Social Affairs, Science and Technology. My name is Rosemary Moodie, a senator from Ontario and the chair of this committee.
Before we begin, I’d like to go around the table and have senators introduce themselves.
Senator Osler: Senator Flordeliz (Gigi) Osler, representing Manitoba.
Senator McPhedran: Marilou McPhedran, representing Manitoba.
Senator Senior: Paulette Senior, representing Ontario.
Senator Greenwood: Margo Greenwood from British Columbia.
Senator Arnold: Dawn Arnold from New Brunswick.
Senator Burey: Sharon Burey, senator for Ontario.
[Translation]
Senator Petitclerc: Chantal Petitclerc from Quebec.
[English]
Senator Bernard: Wanda Thomas Bernard, senator from Mi’kmaw territory, Nova Scotia.
[Translation]
Senator Gerba: Amina Gerba from Quebec. I am not a member of the committee.
[English]
Senator Surette: Allister Surette, Nova Scotia.
Senator Muggli: Tracy Muggli, Treaty 6 territory, Saskatchewan.
The Chair: Thank you, senators.
Colleagues, we will hear from all the witnesses on the meeting notice today; however, I will ask that we carve out 10 minutes at the end of the meeting for a quick in camera session.
Today, we are starting our study on Bill S-201, An Act respecting a national framework on sickle cell disease. Joining us today for the first panel, we welcome the Honourable Dr. Mégie, former senator, who introduced the bill in the Senate in May, and the Honourable Senator Ince, who is now the sponsor of the bill.
Thank you for joining us today. You will each have five minutes for your opening statements, followed by questions from committee members. Senator Ince, the floor is yours.
Hon. Senator Tony Ince, sponsor of the bill: Thank you, Madam Chair and members of the Standing Senate Committee on Social Affairs, Science and Technology, for your consideration, and a very special thanks to the witnesses who have joined us today. You are all very busy people. We genuinely appreciate the time you have dedicated to Bill S-201, An Act respecting a national framework on sickle cell disease.
This framework will change lives and save lives.
Because I have a close family member with sickle cell disease, this framework is deeply personal. I am here because a senator retired. By asking me to become the sponsor of this bill, that senator bestowed upon me a great confidence for which I am deeply grateful.
I will conclude my remarks now so that, without further delay, the next speaker, the original sponsor of this bill, the Honourable Dr. Marie-Françoise Mégie, may have the floor.
[Translation]
Hon. Marie-Françoise Mégie, former senator, as an individual: Thank you, Madam Chair.
Honourable senators, good afternoon. I thank Senator Ince for agreeing to sponsor our bill. I thank the members of the committee for giving me the opportunity to speak today to Bill S-201.
Long before my arrival in the Senate, I was deeply moved by the stories shared with me by friends who, in their fight against sickle-cell disease, recounted the intense pain endured by their loved ones and the lack of understanding they often faced in hospitals. This disease has a major impact, not only on those who suffer directly from it, but also on their families and caregivers, who bear this heavy burden every day.
These powerful testimonies point to an urgent need for action. That is how the idea was born to introduce legislation on sickle-cell disease — Bill S-280 — during the 44th Parliament. Others had thought of this before me, such as the Member of Parliament for Etobicoke North in the House of Commons.
As I explained in my speech in the House, this genetic blood disorder significantly reduces patients’ life expectancy, causes severe chronic suffering, and even leads to disability. For many, without proper care, it is a true death sentence. It is estimated that about 6,000 people live with sickle-cell disease in Canada, and that number is expected to rise.
At present, many people living in remote regions do not have access to specialized sickle-cell care teams. Given that the prevalence of this disease is higher among Black and racialized populations, additional barriers — including socio-economic challenges, systemic discrimination, and structural racism — make care even more difficult to access. This is highlighted in the Public Health Agency of Canada’s 2020 report Social Determinants and Inequities in Health for Black Canadians: A Snapshot.
To remain within my five minutes, I will focus specifically on the content of the national framework.
This framework proposes nine actions. Each action calls on designated entities to act in partnership with the federal government and in collaboration with the provinces. Here are the requested actions.
Regulatory bodies for physicians, nurses and other health professions should incorporate sickle-cell disease into training curricula — for both learners and practising members.
The federal government should provide ongoing support to the Canadian sickle-cell disease registry to ensure its operation, administration and long-term sustainability.
Federal regulatory bodies should develop harmonized national guidelines to inform health policies. The Canadian Hemoglobinopathy Association, CanHaem, has developed one, but it is not widely disseminated.
The federal government, together with the provinces, should ensure equitable access to universal newborn screening for sickle-cell disease across Canada.
Associations and community organizations should develop public education initiatives to reduce stigma against those living with the disease.
The federal government, in partnership with the provinces and through Canadian Blood Services and Héma-Québec, should ensure a diverse blood supply in Canada’s blood banks.
The federal government should explore the feasibility of a tax credit for individuals living with sickle-cell disease and for their caregivers.
The federal government should examine the possibility of including specific criteria to make sickle-cell disease eligible for existing disability benefits.
Finally, the federal government should study the integration of essential treatments for sickle-cell disease into public drug insurance plans.
During my time in the Senate, I devoted myself to raising public awareness about sickle-cell disease at various events, including the annual breakfast on June 19, National Sickle Cell Awareness Day, which many of you know about.
Honourable colleagues, my greatest wish is that sickle-cell disease be widely known and that it benefit from improved care at all levels of intervention: prevention, diagnosis, treatment — including blood transfusions — and patient support.
I am available to answer your questions.
Thank you very much.
[English]
The Chair: Thank you, former Senator Mégie. We will now proceed to questions from committee members. For this panel, senators will have four minutes each for their questions, and that includes the answer.
Senator Osler: Thank you both for being here. Dr. Mégie, it is especially nice to have you back.
I have two questions related to scope and timeline. My first question regards the fact that the bill legislates that the federal Minister of Health must develop a national framework on sickle cell disease and that framework must set national standards for the diagnosis and treatment of sickle cell disease. Is it the bill’s intent to have the federal Department of Health set standards for diagnosing and treating medical conditions, and if so, who will assess if the standards are being met or not?
Regarding the second question, the timeline to table the national framework is one year. It’s a fantastic framework but quite complex and with many parts, including education and training measures, standards, a national registry, neonatal screening, drug plan analysis and tax credit analysis. Is one year realistic?
[Translation]
Dr. Mégie: Thank you for the question, Senator Osler.
For the first question, who will ensure that these measures are taken? You know that it is not the government that will develop everything related to training. There are regulatory bodies, such as CanHaem, that have started to do this and have produced a good document. However, when I mentioned it to my colleagues in the medical profession, no one was aware of it. It is important to work with the professional bodies that are involved in this. The government is not directly involved. However, it needs to be aware that this will be part of the framework.
Next, you’re saying it’s complex, right? Will we be able to do it in a year, is that right?
[English]
Senator Osler: It’s a very ambitious timeline for the framework with all the components to be tabled. Are you able to comment on that ambitious 12-month timeline?
[Translation]
Dr. Mégie: Thank you for saying that it is ambitious. It is a shared task. Part of it will involve bodies such as the Canadian Medical Association and the Royal College of Physicians and Surgeons of Canada. All of these people will be called upon.
On the other hand, the sickle-cell anemia associations of Canada and Quebec will be responsible for anything related to the population. They have already begun doing that work. They have documents on the internet about what to say to teachers when children are discriminated against at school and how to get organized. A lot has already been done, but it needs to be consolidated.
A number of partners are involved in this. When it comes to the registry, specialists are involved. There is a group that has responsibilities, but those responsibilities are shared among several partners. That’s what I said in my presentation. It can’t be separated, but as I said before, some partners will be working with the federal government. For the registry, for example, the federal government will be the leader, but we will need all the provinces and territories to consult their ethics committees and groups to do this.
[English]
Senator Hay: Nice to see you again. It was only briefly that we worked together — just minutes that I saw you in the Red Chamber.
I have two questions. They might be interrelated. First, have you received — or do you anticipate — any pushback, which could actually be systemic discrimination, regarding the proposed tax credits or inclusion in disability benefits? If so, how do you suggest we address that?
Second, how do we ensure Bill S-201 addresses the historical under-representation of sickle cell disease in Canadian health policy, which could also be systemic discrimination, and how do we build accountability into the policy to ensure that discrimination does not happen?
[Translation]
Dr. Mégie: Thank you for the question.
I have been working with sickle-cell anemia associations for many years. I have never heard of any opposition. On the contrary, everyone is expecting something to happen. They dream of the day when it will become law. Unless you have heard of opposition somewhere, I have not heard of any opposition so far. On the contrary, everyone is behind the bill.
Could you repeat your second question?
[English]
Senator Hay: Because of the under-representation of sickle cell in health policy, I worry about it perhaps being agreed upon, adopted, legislated and then pushed to the side. How do we build accountability into the policy so that it isn’t so under-represented?
[Translation]
Dr. Mégie: They are already neglected. Sickle-cell disease is already neglected. That is one of the reasons that motivated me to move forward with this bill.
All the researchers and specialists tell me that when they submit a proposal for research, it is not even considered. There is no data or registry. We don’t know how many people are affected in Canada. The figure of 6,000 is an estimate. There is no proof of that yet. We need a registry. That is why the bill calls for one. We need a data registry. Otherwise, research funding applications will never be accepted.
Senator Hay: Thank you.
[English]
Senator McPhedran: I want to add my warm welcome to Dr. Mégie, and I’m happy to have Senator Ince with us, as well.
I want to try to better understand how you believe this framework will influence treatments. It seems that some of the biggest barriers include rare blood types. Do you feel that, in the framework, this will make a difference in increasing access for rare blood types?
[Translation]
Dr. Mégie: It would make a difference for what?
[English]
Senator McPhedran: Do you envision the framework as making things better for patients who must access rare blood types?
[Translation]
Dr. Mégie: The difference is already there. The framework seeks to address this by asking Canadian Blood Services and Héma-Québec to ensure that they have as many blood donors as possible from all regions and ethnic backgrounds affected by this disease. As you know, apart from the normal groups, which are A, B and O and which everyone knows about, multiple antigens also exist that may stem from different ethnic groups. These antigens have already been set aside. If people are aware and informed, they’ll know that they need to donate blood to ensure the maximum possible supply. This responsibility will be shared by major institutions such as Canadian Blood Services and Héma-Québec.
[English]
Senator McPhedran: Acknowledging the responsibility, do you feel that this framework will either encourage or perhaps push those agencies to find more of the needed rare blood types?
[Translation]
Dr. Mégie: That’s our goal. We want to push them to obtain what the framework requires.
[English]
Senator Burey: Honourable Dr. Mégie and Senator Ince, thank you very much. It is a pleasure seeing you here and having the bill come to our committee.
Dr. Mégie, I have a few questions based on the psychological and financial impacts of sickle cell on families and patients — and also something about having a diverse enough blood supply for our diverse populations in Canada.
I noticed that, as part of the framework, you propose that the Minister of Health ensure that sickle cell disease is included among the eligibility criteria for disability benefits. I commend this initiative. In my career as a pediatrician, I had to complete many forms for other conditions but, frankly, never for a patient with sickle cell disease because they are often denied right off the bat. Can you imagine that, especially given that it is a lifelong, life-threatening condition with quality-of-life impacts similar to cancer — and, like with diabetes, there is no chronic disease tax payment?
In your view, what financial and psychosocial impacts could having the tax and financial credits available have on people living with sickle cell disease?
[Translation]
Dr. Mégie: Thank you for the excellent question. They aren’t eligible for anything.
As you know, some young adults with sickle-cell disease are making the effort to work or attend school. Unfortunately, they don’t have any financial support. They may be absent for two or three months because of their illness, but they won’t have any income. We want this factor taken into account. I noticed this factor when looking at the standard criteria on the completed forms. They don’t meet the criteria. For this reason, we’re asking that efforts be made to identify specific criteria for them, similar to the accommodations for type 1 diabetes. Since 2020, they have introduced criteria to ensure that people with type 2 diabetes can benefit from these subsidies. We’re asking for the same thing. We want criteria pertaining to sickle-cell anemia. These criteria can be identified, given the disability that this condition creates. That’s what we want.
Senator Burey: Thank you. I have another question.
[English]
During the sickle cell disease breakfast event in June 2025 — which was just tremendous — we learned that blood reserves from donors of African and Caribbean descent are extremely low in Canada. According to several researchers, this shortage is largely linked to the national policy that prohibits individuals who have previously contracted malaria or gone to malaria-infested areas from donating blood. In the United States, France and the United Kingdom, people who have contracted malaria are subject to a temporary deferral of three years.
In Canada, a strict blood donation restriction applies: That means you can never donate. Individuals who have travelled to affected regions are restricted, and if they have contracted malaria, they are subject to a permanent restriction.
In your opinion, why is this happening in Canada? Why are we not up to date with France, the United Kingdom and the OECD countries? Why is this happening in Canada?
[Translation]
Dr. Mégie: I really don’t know why. We’ll have government officials with us. We need to ask them about the criteria used to justify their refusal to follow the lead of the United States and the United Kingdom, because they could have done so.
Unfortunately, as you know, people of African origin head back to their countries for vacations. Their blood is badly needed, but they aren’t allowed to donate. Maybe a government official could address this next time.
Senator Petitclerc: It’s good to see you again, Senator Mégie. It’s too awkward.
Thank you for taking over, Senator Ince.
I want to dig a bit deeper into these eligibility criteria. I’m keen to hear about them. Senator Burey and I touched on the eligibility criteria for the current disability benefits. I want to focus on the section referring to the need for this framework to include an analysis of the possibility of introducing a specific tax credit for people with sickle-cell disease and for caregivers. Does this mean that, based on your findings, the current disability tax credit isn’t inclusive? Are the criteria not working? Does it have to do with the time frames? Why does this need to be part of the framework?
Dr. Mégie: This is important. Caregivers need financial support, and young people also need an income. This doesn’t work in day-to-day life.
I tried to find for you the 2018 social affairs committee report entitled Breaking Down Barriers. It provided a critical analysis of the disability tax credit and the registered disability savings plan. The report found that the criteria were too strict for people with sickle-cell disease to be eligible.
However, there’s a nuance. They say “serious illness” and “long-term disability.” Sickle-cell disease can result in a long-term disability, but it fluctuates. It can involve a long period of disability and invalidity and multiple hospitalizations. Afterwards, with the right treatment, including blood transfusions and red blood cell replacement, people can return to work and school. Six months later, they relapse. When they fill out disability documents, it’s important to understand that the people will never fully recover. For this reason, experts could study this issue and include a specific criterion for it. There would be no need to change the entire form. A criterion should be included to make it easier for doctors to fill out forms to apply for subsidies.
Senator Petitclerc: I gather that this would involve including criteria that take into account a certain type of remission and some changes?
Dr. Mégie: Exactly. They did this for type 1 diabetes. They included criteria that fit with certain aspects of the disease’s progression. They could do the same thing here. They did this in 2020. It’s a recent development.
Senator Petitclerc: Thank you. That helps me a great deal.
[English]
Senator Bernard: Welcome back, Senator Mégie, and thank you to both of you for being here. A lot of my questions have been asked and answered, so I will go in a different direction than I originally planned.
For my first question, I wanted to come back to the whole question of access to blood donors. Senator Burey highlighted some of the barriers, but I’m wondering if there are internal barriers within the Black Canadian community. Are there barriers within the community, and if so, what are they and how might the framework address them?
My second question is this: There are some people who would argue that framework legislation is not very impactful, so how would this be different? How would this be impactful to the community that you’ve identified?
[Translation]
Dr. Mégie: Thank you for your question.
First, there are barriers to blood donation in some communities, especially generational barriers. Older people, and even people in my generation, lived with the idea that AIDS was spread by certain Black communities. They passed this prejudice on to their children. They now think to themselves, “I’ll never give blood because people say that, since I’m Black, I’ll infect others with AIDS.” The children may have internalized this trauma because they were born here. They keep thinking, “I won’t donate blood.” Education and awareness are needed. It’s a vicious circle. These components are necessary to clear up the misunderstandings.
Other people don’t want to give blood for religious reasons. That’s another barrier. It always comes down to education and awareness.
Does the complexity of the framework make any impact? Perhaps I’ve worked on it too much to notice its complexity. I don’t find it complex, because each group has a role to play. We’re talking to every organization that trains people, nurses, doctors and others so that they can do this part of the job. They must set national standards that all professionals need to follow.
The associations are working on educating the public. The specialists are in charge of the registry. Everyone has started planting their seeds and is beginning to work on them. At some point, all they need to do is collect them. Provincial data in particular can be used to create national data. That may be where the difficulties lie. It’s necessary to overcome all these challenges and work on them. It will be a long process, but it must be done.
Did that answer your question?
[English]
Senator Bernard: Thank you.
Senator Muggli: The Honourable Marie-Françoise Mégie, nice to see you again.
My question is about the national standards and registry. Is there another health condition that has a registry and national standards in Canada that you would consider an example of best practice? Also, maybe there is another country that is doing this the best. If so, who would that be?
[Translation]
Dr. Mégie: We have models from other countries. Here in Ottawa, a registry is being set up with Dr. Pakhale. You’ll be meeting with her tomorrow. She’ll give you all the details. The registry is in its early stages. She’s trying to connect with fellow experts in other provinces in order to make adjustments. Otherwise, we won’t be able to move forward. The research won’t get very far, and we want the research to proceed.
[English]
Senator Muggli: Is there another health condition or disease for which a registry exists that is successful?
[Translation]
Dr. Mégie: I didn’t quite understand the question.
[English]
Senator Muggli: For example, is there a national registry for a different condition, like diabetes and so on, that could be a model?
[Translation]
Dr. Mégie: Yes. This is important because we shouldn’t reinvent the wheel. Nevertheless, we can draw inspiration from best practices and adapt them to our current efforts and to the reality of the disease in question. Even though a number of genetic blood disorders are involved, they don’t all have the same treatment. They can’t all be lumped together. That said, we can draw inspiration from existing practices. The private sector and associations will be doing a number of things. However, we also want government support for research.
[English]
Senator Muggli: Do you have an example?
[Translation]
Dr. Mégie: For example, Cystic Fibrosis Canada has worked hard to develop a registry that contains 40 years of data. We spoke with them. One of their representatives said that he’s ready to meet with us to provide inspiration whenever we want.
[English]
Senator Muggli: Is there another country we should look to for expertise?
[Translation]
Dr. Mégie: Yes. Our experts in Ottawa may tell you that we need to look at the practices in France and Great Britain. These places are way ahead of us given the high proportion of the population affected by sickle-cell disease. These two countries are way ahead of us, particularly in terms of numbers and record keeping. They can certainly provide inspiration.
Senator Surette: Good afternoon. My question concerns education, training and research. Can you talk a bit about the impact on universities and community colleges in terms of training in medicine and other health care fields? Tell us how the framework will affect colleges, universities and training, and describe the current research and education landscape. How will this framework improve the situation?
Dr. Mégie: As discussed, education and training must include higher entities that work with various faculties, such as medical and nursing schools. We’ve already met with a number of them, and we’ll be meeting with others soon to raise awareness.
Medical textbooks don’t devote much space to sickle-cell disease. The chapter on genetic blood disorders is quite long, but seems to be of little interest. Students study these chapters to pass their exams. We want them to know what to do with an affected patient.
In large specialized centres, patients receive good care from teams that specialize in sickle-cell disease. However, when patients go to rural areas or regions where the disease isn’t commonly discussed, they’re seen as having anxious parents. In addition, a young woman who comes in with a multitude of needle marks from multiple blood tests is viewed as a drug addict and is isolated before receiving care, even if she’s in agony. All these stigmas exist. Health care professionals must know what to do.
Some people don’t even know the name of the disease. A person from Africa knows the term “drepanocytosis”. If they arrive at the hospital and mention this disease, they’re asked what it is. This aspect is hugely important.
Senator Surette: Does the framework also cover awareness, guidance and commitment? What will be the financial impact? Does this mean that the government is committed to working with institutions and organizations to make progress on this issue?
Dr. Mégie: On a financial level, I don’t think so. It’s a matter of training and telling specialists, when they give their courses, to remember this disease and to talk about it. As for other entities that organize conferences and that don’t train the next generation at university, when I approached them, I asked them to include one or two workshops on sickle-cell disease in each of their conferences. Some learners are connected to universities, but members of these associations also learn through continuing education while on the job. We must reach out to all these entities to raise awareness so that, one day, everyone will finally be informed about the disease.
Senator Surette: Thank you.
[English]
Senator Senior: Thank you to my colleague for being here and to Senator Ince for picking up the baton.
I want to follow up on a couple of questions that were asked earlier. One is with regard to the importance of a framework and the research and how that leads to action. I’m particularly thinking about, for example, the importance of having the data that will inform what we do next. Is the framework really about the education, awareness building and data gathering? Would the next step then be the implementation? That’s my first question.
My second question arises from that, because I’m thinking of other health issues, particularly in the Black and racialized communities, where specific kinds of education had to be done in order to impact change. I’m wondering about that particular piece of education — whether it’s going into a barber shop, hair salons, churches and so on — and whether the framework allows for that kind of education to be done. If so, who would do it?
[Translation]
Dr. Mégie: I’ll answer your second question, since I appreciate this aspect and I’ve seen it in Montreal’s Black community with regard to HIV/AIDS. A person created a committee whose members went to hair salons and other places where people gather to share personal information. The committee members get noticed. With the manager’s permission, they provide training.
The associations also ask parents to do the following. When their child is singled out, they should meet with the teacher and explain that, when the other children in the class say that their child never comes to school, the teacher should tell the children that their child has been hospitalized three or four times. The education must include not only information drawn from literature, pamphlets and the internet, but also information gathered in the field. You made a valid point. I find that intriguing. This could also be done.
You also asked how research can lead to data collection. Dr. Pakhale started on her own in her little clinic at the Ottawa Hospital before it even opened up to large-scale research. She started with patients who came to her clinic. She recorded them one by one when she wanted to start the registry in Ontario. It has now grown as a result of the contacts established and the expansion of the research facility. It’s possible to start something similar in another province. All you need is a specialist. Without good data collection, the money won’t be spent. The money won’t come from the government, but rather from private companies working on research and innovative drugs. However, if no research is carried out in this area, these efforts will be wasted.
That’s why we need to seize this momentum in order to work hard on this issue. The momentum in question stems from the fact that new drugs and gene therapy are on the horizon. The companies working on this issue would also like the support of the bill to move forward.
[English]
Senator Greenwood: It is very nice to see you, Dr. Mégie.
I want to build on something that a few of the senators have mentioned. A lot of questions I wanted to ask were already taken. That’s good; I’ll be brief.
I was wondering whether, in the content of the national framework, there could be the inclusion of a knowledge translation strategy. I say that because sometimes we have to socialize the idea of what sickle cell disease is. Sometimes, families themselves don’t know. We have talked a lot about caregivers and their training, but what about the people who are being affected by this? Sometimes, you don’t know those things.
How do we socialize the idea that, first, it exists, and second, it’s very important to address it — and that we address it through the caregivers, families and communities?
There is a place for creating that awareness. People use the word “awareness,” but it is awareness beyond professional caregivers in the health care system; it is the awareness of communities and families. Once we socialize that notion, the importance of that no longer remains a question; it needs to be addressed.
Regarding all of these pieces, I’m in total agreement. I have sat in on a number of these pieces in my past life, developing national standards and all those sorts of good things. But there is a place for a knowledge translation strategy that is broader than just targeted training for caregivers — so I wondered if you had thought about that. Certainly, you are thinking about the broader community, so I add this to your thoughts.
[Translation]
Dr. Mégie: We first thought of health care professionals, specifically given the danger involved. When people come to the emergency room, children have died because the professional in question had no clue what to do. It’s important to work on this. However, families are doing an outstanding job. Certain groups are referred to as “warriors” because every time they meet someone, they talk about it. Despite our best efforts, some parents still believe that the subject is taboo. They say, “I won’t tell anyone that I have sickle-cell disease because of my children,” but the children already have it. Do you understand?
Other families help and support them and create discussion and support groups. These families designed the documents to hand out to teachers. As soon as you type “sickle-cell disease” into a search engine, you’ll see all the information. Some young people even explain their disease to the general public through podcasts or internet videos. These areas warrant further attention and continued progress.
[English]
The Chair: We have three senators who want to ask questions. You have two minutes each. If you have a longer question, we may have to ask for a response in writing.
Senator Bernard: I’ll just repeat the other question I had asked.
The comment I made was that there are several people who would argue that framework bills are not very impactful or effective for a number of reasons. My question to you is this: What makes you think this one would be impactful? What is the difference with this framework bill?
[Translation]
Dr. Mégie: I think that it will be different, given that no single person or institution is responsible for everything. If one institution or person were responsible for nine actions, I wouldn’t even write it down, because I would find that inconceivable. Given that each group has a role to play, it should work.
[English]
Senator Burey: Earlier, a question was asked by Senator Hay around accountability. Where is the accountability factor, with so many people having responsibilities for different components?
[Translation]
Dr. Mégie: We must get organized. We’ve already asked that, after one year, the government submit a report on progress made. After five years, we’ll be able to say what has and hasn’t been accomplished and why. We hope that this will work for the strategy.
[English]
The Chair: My question is very quick. It’s about the increasing trend around the care that’s delivered to children with rare diseases — to gather them up in centres that have a higher level of expertise to deal with these cases. That’s one trend.
The other trend is that we know the delivery of care is under the jurisdiction of the provinces. With that in mind, how will this framework help the problem that you’re talking about, which is in the community, at the first point of contact? How is a patient actually going to be cared for? How are you going to overcome the retreating level of care from these front-line areas? The provinces are making the decisions about who does what and where.
How does this framework help those problems?
[Translation]
Dr. Mégie: Normally, for financial reasons, when rare diseases, serious illnesses or major surgeries require a significant amount of money, not all hospitals provide this care. Instead, specific hospitals and facilities are designated to provide this care. However, if all professionals know about the issue, they can refer patients to the facility in their province. Even if the professionals don’t personally provide the care, they can know what to do in an emergency and then refer the patient to the specialized facility for treatment. Of course, it isn’t possible to have plenty of facilities for sickle-cell disease.
[English]
Senator Senior: You mentioned that everyone supports this, but does that include governments and drug companies? Who are the people supporting this, beyond the community that really needs it? Who are the people with decision-making powers who actually support this?
[Translation]
Dr. Mégie: Right now, we have the support of the people behind the Quebec and Canadian associations. However, we can’t say that we have government support. We’re waiting for the government to take up the issue so that it can ultimately provide support, first in the Senate and then in the House of Commons. At that point, we’ll know when and how the government will provide support. For now, the public needs this. Families struggling with the disease, their children and the people around them, even if they aren’t affected, see and witness the plight of these individuals. They strongly support it.
[English]
Senator Hay: As I look at the framework with the nine actions, I think of the framework as the overarching strategy, and some of these actions require many strategies that could support some of the others.
Let me explain. When you’re talking about measures for supporting public awareness, I think that will require a full communication plan and an awareness strategy. It will require funding and accountability, measuring whether there was an increase in awareness. Then it tags into the national research network and registry and national standards. This is because on the other end, the point of contact requires trust. So you need promotion.
Have you thought about how to build trust within the framework? I ask because data collection, requirements and trust are not a natural given for government organizations or government-sanctioned frameworks.
[Translation]
Dr. Mégie: I didn’t quite catch that. However, I can say that it’s a cycle. If we don’t have data, we can’t move forward with research. Research provides more information so that we know what to do. That’s why the communication strategy will play an important role. The registry must include both the disease and its background, for example, because the registry must be quite comprehensive. This creates an opportunity, but things can become tricky if a link is missing and we don’t have all the required information to submit to a research funding agency. One depends on the other. The communication strategy is vital. All our colleagues coming to speak will focus on this aspect to ensure progress with the framework. It’s a good suggestion.
[English]
Senator Hay: I have a quick follow-up question with regard to stigma and trust. There must be a strategy around overcoming stigma and fostering trust to have people come forward, as you said. Some parents don’t even acknowledge that their kids have sickle cell. I’m spending too much time on this; I support your bill.
[Translation]
Dr. Mégie: The stigma stems from the fact that health care professionals don’t know about the disease. You come in with a bunch of holes and a bunch of needle marks? You’re a drug addict. You come in too often? You don’t go to school enough? You’re lazy. That’s what patients face. I think that, if the professionals were well informed, they would know that these marks aren’t from drugs, but from different blood transfusions.
[English]
The Chair: This brings us to the end of the first panel. I would like to thank Senator Ince and former Senator Mégie for their testimony today.
For the next panel, I would like to welcome the following witnesses, joining us in person from Health Canada: Daniel MacDonald, Director General, Office of Pharmaceuticals Management Strategies, Health Policy Branch; and Dr. Co Pham, Executive Director, Centre for Vaccines, Clinical Trials, and Biostatistics, Health Products and Food Branch.
Thank you for joining us today, gentlemen. We will begin with opening remarks from Mr. MacDonald. You will each have five minutes for your opening statements, followed by questions.
[Translation]
Daniel MacDonald, Director General, Office of Pharmaceuticals Management Strategies, Health Policy Branch, Health Canada: Thank you.
We’re here today to support your study of this bill by providing information on the Government of Canada’s current measures in the area of sickle-cell disease, and rare diseases more generally.
I’ll speak to activities in the health policy, regulatory and research spheres.
From a health policy perspective, sickle-cell disease is considered — by various definitions — a rare disease. Estimates indicate that thousands of other diseases are also considered rare diseases, collectively affecting approximately 1 in 12 people across Canada.
Given this broad scope, the government is tackling a number of challenges common to all rare diseases as part of a single strategy called the National Strategy for Drugs for Rare Diseases, launched in 2023.
Examples of these challenges include:
[English]
There is the lack of timely and accurate diagnosis, known as the diagnostic odyssey, lack of treatment options and delayed or otherwise constrained access to emerging therapies and clinical trials — when they do exist — and difficulty with typical drug evaluation approaches, given small patient populations and uncertainty about treatment effects.
By definition, no strategy actions are tailored specifically to sickle cell disease, but several measures may have direct implications for patients and the sickle cell disease community.
For example, the strategy supports developing expert guidance on the types of conditions that could be tested and screened for in newborns across Canada; establishing registry standards and dedicated funding supporting registries in capacity-building efforts to improve the availability of critical data and evidence; and reaching agreements with all provinces and territories to support improvements in public drug plan coverage and reimbursement for rare disease drugs.
From a regulatory perspective, Health Canada reviews drugs submitted by manufacturers for authorization of sale in Canada. To date, the only specific treatment for sickle cell disease that has received approval in Canada is Casgevy; that was in 2024. It is currently being negotiated by the pan-Canadian Pharmaceutical Alliance, or pCPA, for coverage under public plans.
There are other treatments in Canada to help patients with symptom management, including red blood cell transfusions and drugs used off label. For instance, hydroxyurea has received market authorization in Canada for the management of certain cancer diagnoses. However, it is not authorized for management of sickle cell disease and so is used off label.
We also recognize that there are several emerging drugs to treat sickle cell disease at varying stages of research and development, though none have yet reached the point of being submitted to Health Canada by their manufacturers.
From a research perspective, the Canadian Institutes of Health Research, or CIHR, has invested $13.8 million into new sickle cell disease research over the past 10 years. This funded research ranges from biomedical studies to clinical trials, including new treatment avenues. Most of the work is based on project grants to researchers who, through the open or investigator-led granting process may choose to collaborate with charities, industry and other partners to strengthen their funding applications.
In the case of rare diseases, this collaboration often consists of a Canadian arm linked to international collaborations that are vital to achieve meaningful study sizes.
I want to close by acknowledging that while sickle cell disease is considered a rare disease, its prevalence is not equal across the population. We know that the disease disproportionately affects Black, African and Caribbean populations, and that these populations also face barriers to accessing some health services given existing policies regarding blood donation.
For this reason, the government is working with Canadian Blood Services and Héma-Québec to address donation barriers, strengthen the security of supply for at risk groups and improve donor recruitment. Thank you.
The Chair: Thank you, Mr. MacDonald. For this panel, senators will have four minutes for their questions, and that includes the answer. Please indicate to whom your question is directed — to a particular witness or both witnesses.
Senator Osler: Thank you to both witnesses for being here today. I believe both of you were in the room when I asked my question to Senator Mégie, and again, it is about the bill’s requirement to set out evidence-based national standards for the diagnosis and treatment of sickle cell disease. The first part of the question is this: Can you fill me in on how Health Canada, through this framework, would set standards and who would assess and monitor compliance?
The second part is on the neonatal screening, which is a provincial and territorial responsibility. How do you see the framework coordinating something that falls under provincial and territorial jurisdiction?
Mr. MacDonald: I’m happy to address that question in the context of our experience through the National Strategy for Drugs for Rare Diseases. Regarding the question on how Health Canada works on setting standards, we do all of this through two avenues. The first is working directly with provinces and territories. It is their jurisdiction. Health Canada’s role here is to convene and to offer support in the development of evidence and anything to support that conversation.
This leads to the second theme of the national strategy, which is the work that we undertake through Canada’s Drug Agency and the Canadian Institute for Health Information. The work happening there has to do with what we call the foundational work. In terms of standards that could be adopted by provinces and territories in exercising their jurisdiction, what might some best practices be? Canada’s Drug Agency, for example, was able to convene a newborn screening panel, bring together experts, develop some guidance and publish that.
That is there for provinces and territories to use. It is their jurisdiction as to whether and how they use it, but in this specific case, a report was published by Canada’s Drug Agency. It made recommendations for what you might list as indications to screen for in newborns. On that list were three subtypes of sickle cell disease, but the point is that it was a broad framework. It is published and available.
We then talk to provinces and territories. It is a piece of the information that is considered in that conversation, and through the agreements that have been signed with all provinces and territories about the implementation of the National Strategy for Drugs for Rare Diseases. We have a working group where we talk about screening and diagnostics and where we’re going to go with a portion of the funding that is identified for that. The role of advice produced by Canada’s Drug Agency will be part of that conversation regarding how provinces and territories choose to execute.
Senator Hay: Thank you for being here. As a side comment, $13 million over 10 years is pretty nominal for research, and did you say clinical trials too? You don’t have to answer that. It just didn’t seem like very much, which leads me to drill into the question of what internal capacity and funding Health Canada currently have to support the development of this national framework on sickle cell disease. How will it get prioritized at Health Canada?
Mr. MacDonald: To clarify, is the question in relation to research?
Senator Hay: The national framework. The other comment was just a side note that it didn’t seem like a lot, which is a barrier in itself.
Mr. MacDonald: I’ll answer the question by picking up on the comment first, because an element of the National Strategy for Drugs for Rare Diseases is enabling CIHR to conduct research — not in the specific domain of sickle cell research; the general approach of the strategy is broad, to undertake research that is anticipated to have benefits for as many rare diseases as it can be applied to, so I would point to $20 million over five years. The national strategy funding has been identified to support pediatric clinical trials and treatment networks through RareKids-CAN and established by the Maternal Infant Child and Youth Research Network. There is $2.3 million over four years, increasing clinical trials —
The Chair: I’m very sorry to interrupt you. We are having difficulty hearing you. Could you raise your volume just a bit?
Mr. MacDonald: I’ll move the microphone closer.
Senator Hay: The root of my question is this: How will the national framework get prioritized at Health Canada?
Mr. MacDonald: I’ll move to that. The intent of the national strategy, through the main tool of the dollar amount, is the bilateral agreements that have been signed. In the bilateral agreements, there are a number of what we call eligible expenditures that provinces and territories may use that funding to support.
In terms of looking through the bill as it’s proposed, a significant number of activities in those proposed areas would qualify as eligible expenditures if provinces and territories chose to devote expenditures that were in the area of sickle cell.
That is not to say that they will. It is a broad strategy that applies to activities across all rare diseases by design, but there is a section of the agreements that permits things involving communications, best practices and adoption, as well as what I described in my last answer as being the foundational work that we’re trying to put in place to enable that to be done.
It’s not just for the actual reimbursement of specific drugs, as listed in the agreement.
Senator McPhedran: Thank you for being here. It’s helpful to have your experience to help us respond to this. I have a question about hydroxyurea. What are the impacts and indicators for patients and prescribers for this drug being used off label for patients? Are there any efforts to regularize this as a treatment for this disease?
Dr. Pham: I’ll start.
As you know, hydroxyurea is a mainstay in the management of sickle cell disease. It has been around for a long time. In fact, it’s indicated for various uses. Its mechanism of action is an anti-metabolite. We recognize it as a chemotherapeutic agent as well.
The fact that it’s not indicated in Canada but is indicated officially in other jurisdictions doesn’t mean that health professionals can’t apply it to specific patient populations who may need it or utilize the known evidence base within the scientific literature for its application. As indicated, we know of it from long-standing usage. For example, I think the first pivotal trial that directed the United States Food and Drug Administration, or USFDA, in approving hydroxyurea with the official indication for sickle cell disease was back in the late 1990s. That information is applied here as well.
The reasons why we don’t have an official indication are various, and you may know about them. For an old drug that’s not so expensive regarding marketing and commercial applications, it might not be as interesting for sponsors and manufacturers in our economic environment here. That’s something that we can reflect on as well.
Coming back to its usage, health professionals can still decide to use it. Within our health care system here, health care professionals may utilize their professional judgment and exert their authorities and abilities to decide on off-label use. They have done so. Therefore, hydroxyurea is applied and used as a treatment for sickle cell disease in Canada.
Additionally, we know that there are other agents within the pharmaceutical realm that are used off label in Canada, and, again, they may be approved in other jurisdictions. There are probably two that you recognize. That is something we continue to try to look at in terms of where the options are for having marketed labels in Canada.
I’m not sure if you’re going to ask here, but I’ll extend a thought. This question around rare diseases for drugs is interesting, because we are probably at an inflection point where therapies are coming on board that we haven’t seen in this disease state. For probably 20 or 30 years, we have not really had more than what you have just noted here, but Mr. MacDonald has indicated here that Health Canada has approved Casgevy, a gene therapy. We know of another that the USFDA has also approved — another gene therapy — so there are two in the U.S.
We see on the horizon monoclonal antibodies, so there are biological treatments there. We also see small-molecule options that are coming online as well.
In my line of work as a regulator for clinical trials, I do not have a crystal ball, but I do have some insight in terms of what is coming. We see in late-stage clinical trials — stages 2 and 3 — some promising agents and, in early stages, more innovative biological agents coming as well.
We’re on the cusp of seeing more therapeutic options.
The Chair: Thank you, Dr. Pham. I’m going to interject a question to follow up on Senator McPhedran’s question. Many of us in this room don’t know that close to 80% of the drugs in the pediatric world are used in an off-label fashion. It is almost a standard of practice in this country. Where is the trusted jurisdiction program now, where we rely upon other countries we have identified as trusted jurisdictions to bring us their label information about drugs that we were going to adopt and use, so that practitioners like me don’t have to go back to the researchers and can actually use Europe’s and the U.S.’s knowledge bases with their labels?
Dr. Pham: Daniel, is there a policy side to this that you wanted to open with?
The Chair: We have been in discussions about this for a few years, right?
Mr. MacDonald: So the question is about reliance on foreign jurisdiction approvals in the drug approval process. It’s something that is not finished yet; it’s in progress.
Do you have anything?
Dr. Pham: There is a lot of development to be done. Your question opens up a lot of avenues, and improvements are necessary. Wearing my regulator’s hat here, I think Daniel is absolutely right that there are initiatives in progress to try to address exactly the scenario and situation you’re describing, in order to allow us to apply scientific knowledge, international decision-making in labelling and applications of standards of care in a way that clinicians can use to benefit the pediatric population.
The Chair: Thank you.
Senator Burey: Thank you so much for being here and for lending your expertise and digging into the weeds.
First, I want to salute Health Canada and the government for putting forward the National Strategy for Drugs for Rare Diseases, because I think it’s foundational, as you said, Mr. MacDonald, in laying the groundwork and setting the standards that this framework — I can only suppose — will build upon or align with.
I have two questions. The first is regarding a hydroxyurea issue and the fact that it’s still used off label. I’m a pediatrician, so you know what’s coming. The problem is that when you have to explain to parents and families that this drug is off label, it’s very hard for them to accept it. It can also be stigmatizing: “Something is wrong with me. Why am I doing that?” I’m sure that aspect has come out, but it is a very serious one, particularly in marginalized communities. That’s just a statement to take it back and reflect on how important it is for us to have an on-label designation.
I also really wanted to drill down on the research money that Senator Hay spoke about. Being researchers, you’re well aware of the research that’s going on looking at some of the clinical data — indicators of inequity in research and funding for various rare diseases. One article I’m looking at is one on sickle cell, which was just in The Lancet and released September 2025. You know this data already in terms of funding per capita, basically, of the rare disease.
Would you have that kind of research in Canada? For example, if you compared sickle cell to cystic fibrosis and hemophilia, would you have total research spending? What is the funding per registered patient? That kind of data digs deeper so that we can have an equitable distribution of research funding. I will ask you to answer that.
Mr. MacDonald: I’ll start with a clarification, perhaps, of what the research funding delivered through the national strategy is doing and — as I think your question is about primary research dollars — how that relates to actual research into specific indications and treatment. The research funded through the national strategy is more foundational, regarding that system trying to encourage access to clinical trials for all rare diseases, setting up a pediatric network for clinical trials — that sort of an emphasis. It regards how you can use the data that exists in the health system today to properly identify who we are talking about and what information we have about them to use in other broader applications of treatment and research.
So that’s sort of a step back. It’s about setting up the system and the environment. I think what you’re referencing is primary research — how much primary research is being done in cystic fibrosis versus another disease. I’m sorry, but on that I would have to defer to the Institutes of Health Research. They couldn’t be with us today, but we’d be happy to take that sort of a question back to them.
Senator Burey: Thank you.
Senator Bernard: Thank you both for being here today. I’m not sure which of you could answer the questions I have. I’m going to be very brief. I have two fundamental questions I want to ask. One is this: What are the key measures that you feel must be included in this framework? My second question is this: Is this doable within this one-year timeline? Is that one-year timeline realistic?
Mr. MacDonald: Again, I’ll answer the question in the spirit of the experience that we have had in the implementation of the national strategy we’re putting into place now. In terms of key measures, critical measures for us — the bilateral agreements are an expression of convening provinces and territories for whom this is the jurisdiction in a common direction. So the bilateral agreements are important as there is a cohesion here.
To think long into the future about what the money provided for the national strategy will be able to accomplish, in the bilateral agreements that were signed, there is a component about real-world evidence generation and its application to decision-making regarding future drugs for rare diseases that haven’t come out of the pipeline yet. It’s what we call developing the toolbox for evaluation and decision-making, which would have an impact on the drug approval system in Canada for drugs for rare diseases long into the future, for drugs that we don’t know of yet.
Those were critical parts for us. If you’re talking about drugs, it has to be related to patient care; that’s why there is a screening and diagnostic portion. There was also a recognition that we can do so much and act in so many areas, and so focus was definitely a key element of how the national strategy was developed.
It is ensuring that, for the pieces of the national strategy that you have, you have enough in there to make meaningful progress in all of the areas that you choose. That’s why there are four specific pillars and that’s the way that it’s built.
In terms of whether it is doable in one year — speaking about the national strategy — it is going to take a lot of time to have the partnerships, to have the necessary collaborations and to get answers to some difficult questions as to how, for example, evidence generation will be taken into account and decision-making be influenced. So from our perspective, it’s a longer time frame.
Senator Bernard: So how long? I think you have an answer but are maybe a little reluctant to tell us.
Mr. MacDonald: We started with the first three-year period in the agreements for a reason. We also started with a very explicit lessons-learned approach, meaning that the national strategy was put in place and there was almost a sense that we heard from those who were waiting for it, “Do something.” The authorities for the strategy do expire after three years, the end of the agreements, and then we will take stock of what we have learned collectively with provinces and territories. We have a stakeholder and patient connection through what we call an implementation advisory group that we will consult, regarding how the strategy went and what they suggest didn’t go so well in terms of what we stated as a government we were trying to achieve here. Did we achieve it? If not, what might be improved? The answer to your question is that this is a step-by-step thing. We don’t know how far out that thing is, so we are dealing with the first step, learnings the lessons and going from there.
Senator Senior: Thank you for being here. In light of the strategy and the progress that you’ve made and the focus on rare diseases, is a national framework as developed necessary to advance treatment and provide access to drug coverage for individuals affected by sickle cell disease now?
Mr. MacDonald: As a department, we’re implementing the national strategy, and our hope today was to be as clear as possible about what it is that the national strategy is intending to achieve, how it’s structured to try to do that and where there might be touch points specifically regarding sickle cell disease.
We offer that in the sense of input to your study as to this specific bill and the construct of another framework. However, in terms of a determination as to whether there is a necessity for something else, we’re focused on the national strategy and trying to make it as applicable as possible to as many rare diseases as possible.
Senator Senior: I’m trying to be a bit more pointed in this because I hear you saying you’re focused on the national strategy, which I appreciate, but I’m really asking specifically about sickle cell disease and access to critical drugs now. My concern is waiting on a framework in order to provide that access is costing lives. So that’s the basis of my question.
Mr. MacDonald: This is my direct response in terms of the application of the National Strategy for Drugs for Rare Diseases for sickle cell disease specifically: Once a drug has passed through the drug-approval process in Canada — which means it has passed the health technology assessment, or HTA, process, it has gone through the pCPA price negotiation and provinces are now at the point of being able to determine whether to put it on the public formulary — when it is a rare disease, the agreement allows them to use the funding from the Government of Canada to support the provision of new drugs for rare diseases. There is a set of specific ones. They can put it towards those or improve access to existing ones.
In terms of sickle cell disease, when we reference that Casgevy is currently under negotiation with the pan-Canadian Pharmaceutical Alliance, the importance of that is there will come a decision point where provinces and territories will be determining whether to put that on their formulary. If they were to do so, this is a drug that under the national strategy they would be able to allocate funding to.
The rest of the activities that we set out, all that foundational work in terms of registries for rare disease, I understand that you’re getting a presentation on your next panel from somebody who’s trying to put one together. The guidance that has been published from Canada’s Drug Agency is there to guide anyone trying to put together a rare disease registry, so that would be an asset for them to use. I just highlight those as a couple of connections.
Dr. Pham: I will add as well to your question, which I think is very important, recognizing that in Canada, Health Canada currently has approved products that are available for the medical management of those with sickle cell disease. So, although we did have the discussion and questions in regard to the ideal state of them being authorized with labelled indication, they are available.
So, as we look forward to innovative therapies that are coming online, I think that the program itself is what Daniel is talking about. Looking at the availability of analgesics or pain medications in cases where we need to manage patients with sickle cell disease, we have some of those options there. The two examples that Senator McPhedran had asked about earlier, those are pharmaceutical agents available as mainstays in Canada as well.
Senator Petitclerc: I have a follow-up question for clarity. This falls under rare diseases. I understand that. I assume that resources, funds and time are limited when it comes to rare diseases. Are the rare diseases competing with each other when it comes to funding? Maybe you’ve touched on this already, but if they are competing, how do we prioritize where we invest time, research and treatment? In the context of this proposed bill, would a framework somehow help provide an environment where this rare disease is treated as more of a priority?
I’m trying to understand the environment in which choices are made.
Mr. MacDonald: Thank you for the question. I will start by indicating my role Health Canada: I do not manage a drug plan.
Senator Petitclerc: Yes. I know that.
Mr. MacDonald: I just need to clarify that on behalf of all the people I work with who actually do, because what I’m going to say is almost speaking for them, and I want to be careful.
If I interpreted it correctly, your question is about whether sickle cell disease is in competition with other rare diseases for access to funding through the national strategy. I’m going to explain that it is not.
The way the agreements are structured, provinces and territories always remain responsible for their jurisdiction and any decisions about whether to list and reimburse for any drugs for rare diseases. But the agreements are designed to make it — in essence — easier for them to do so, because there is dedicated funding being provided through an agreement for them to be able to do so. They always make their decisions in the broader sense of the question, “Is there evidence that this is an effective drug?” It’s passed through all of the steps of the pharmaceutical approval process in Canada. When it gets to the end, the difficulty with rare diseases is that quite often, because of their nature and the evidence that comes with whether the hoped for and anticipated benefit is there, there can be some questions about that. The spirit of the national strategy is to make it easier to make those listing and reimbursement decisions. There is some funding there to help de-risk it, so to speak, while we collect real-world evidence that shows whether it is, in fact, what we had hoped it was.
In that dynamic, there is not a competition between rare diseases, no.
Senator Petitclerc: Thank you. Are you saying that this applies to research as well?
Mr. MacDonald: To return to a response I provided earlier, we’re looking at a step back in terms of the overall environment to enable research, clinical trials and so on for all rare diseases. So it’s a very broad rare disease, 1-in-12-Canadians type of approach. It does not, in the national strategy, have any bearing on the primary research that is undertaken by researchers regarding a specific disease and whether we have a treatment for it.
Senator Petitclerc: Okay, thank you.
The Chair: I want to ask you a question, if I can interject before we go into the second round. It’s my impression that we’re talking about two different things. We’re talking about a national strategy that sits in place to advance work around all rare diseases, is nonspecific and is actually centred around drugs — drug use and drug financing. What we have in front of us is a framework that is attempting to pull out a particular rare disease and speak to it in its full context — the envelope of care, the social implications, the financial support for patients, the medical care being delivered and so on.
If this framework were passed, how would Health Canada take on the new legislation that it faces? Because if I am correct, it bears no resemblance to the strategy; it’s outside the strategy, or seems to be.
Mr. MacDonald: You are correct in your characterization of the two things that we’re in essence talking about here. The National Strategy for Drugs for Rare Diseases approach was broad and wide specifically because we were addressing the problem of 1 in 12 Canadians having a rare disease, but there are thousands of them, whether it’s 7,000, 8,000 or 10,000. I’ve seen different numbers, but there are thousands. So with the funding that was available, the government attempted to create a framework that would address as many as possible by setting foundational elements that could be leveraged long into the future to benefit the treatment of many rare diseases.
The other thing that we were faced with was a statistic that we’ve heard many times: that of those thousands of rare diseases, only 5% have a drug therapy. To take your point, there are elements other than drugs that are important for care. That was one of the elements that was important to us. We had to focus to ensure that we were achieving something in the areas that we elected to address. We focused on drugs because they are so important to so many. It’s really the system by which drugs are seen, considered and approached and decisions are made that we’re trying to address.
To answer your question — if we had this legislation that was very specific to one indication, it would have a significant effect on how Health Canada manages this sort of rare-disease-wide approach. We would have to then have to accommodate; we would have a commitment in legislation where we need to do something for one of the thousands. We would have to ensure that we did that somehow. But I don’t have a definitive answer right now as to how that would happen.
Senator Burey: This is just following on that clarification. As I said at the outset, I salute Health Canada for producing the National Strategy for Drugs for Rare Diseases. My question is this: Is there anything in this bill that would hinder the rare disease strategy? My second is this: Has the case been made for the necessity of continuing the rare disease strategy? Is there anything that will hinder it?
Mr. MacDonald: Hinder it? No. We’re very cognizant at Health Canada that the national strategy already operates in a bigger world, that there are elements of care that the National Strategy for Drugs for Rare Diseases is not focused on and that we need to work with partners. Groups are coming together to address different parts. So we’re operating in a broader ecosystem as it is and recognize that. The framework, as I have seen it laid out, explicitly recognizes that there is a winder framework in which treatment for sickle cell disease operates. It’s just for one indication, not for trying to do everything. So would it hinder it? No. It just requires some adjustment.
Senator Burey: Second, would it help make the case for the necessity of a rare disease strategy? You just talked about time and funding constraints in three years; then you’re going to reevaluate. To me, this is something we need to and should look at continuing. In this framework, is there anything that would make the case for continuing it?
Mr. MacDonald: I think the case for the continuation of the existing National Strategy for Drugs for Rare Diseases is clear from the decision that put it in place initially. I think the existence of all of the bilateral agreements with the provinces and territories and the progress that’s being made in the work with them — focusing on real-world evidence decision-making and the funding being used to improve national consistency and access — are making the case for it.
Senator Burey: Thank you.
Senator Osler: Gentlemen, the nice thing about being on second round when you come to the Senate Committee on Social Affairs, Science and Technology is that all of the questions that I had for second round — the ones on duplications posed by Senator Senior, the ones on the one-year timeline posed by Senator Bernard and a clarification on a framework when we have an existing rare disease strategy — have been answered. So I just want to say thank you for being here.
Senator McPhedran: I have a very quick question. In your experience, are frameworks like this helpful for the work that you are charged with doing?
Mr. MacDonald: The national strategy is a framework. We are working with partners to figure out what the pieces of that broader framework will look like. Part of what we do at Health Canada is work with partners in provincial and territorial jurisdictions in the area of administration of drugs.
Yes, a framework is helpful because it provides direction. To my point that you need focus, you need to prioritize your activities, to sequence and so on, and these things are helped by having a clear vision from the outset of what you’re trying to achieve, as well as the design of the pieces you think are important to put together to achieve that end. So as a tool, we find it very useful.
The Chair: I will ask the last question.
How will Health Canada work with the provinces differently to how you’re working now? You already have evidence out there around neonatal screening. You already have information around the drugs — shared information from trusted jurisdictions. As you framed it earlier, it’s there and you offer it. How would you work differently now that you have a piece of legislation that says to organize all of this in one place and try to sell it to the provinces? That’s my read on it. What is yours?
Mr. MacDonald: When we deal in the national strategy, obviously when you’re trying to come from the level of a framework, you have to come down several levels to actually deliver something that makes an impact on Canadians. In the environment I’m describing, where we’re convening with provinces and territories and working with everybody, we have to talk about specific indications and drugs related to them at particular times.
There is already an element within the national strategy of coming down to specific indications. So there is a common set of drugs in the bilateral agreements that are tied to specific indications that are being investigated for applications and real-world evidence. If we had this legislation and the sickle cell framework in place, we would have to be cognizant that, as we are doing our work, we need to be clear about what is happening that is relevant to sickle cell disease.
That said, the overall approach of trying to do things that are going to benefit as many indications as possible would remain, so we would have to balance those two competing priorities.
The Chair: Thank you very much, Mr. MacDonald and Dr. Pham. I thank you both for your testimony today. I know we’ve been a difficult crowd. Senators, this brings us to the end of this panel.
Colleagues, we will continue in camera.
(The committee continued in camera.)