Health Committee on March 7th, 2023

[The Chair, Liberal, Sean Casey]

Welcome to meeting number 55 of the House of Commons Standing Committee on Health. Today we meet for an hour and a half with witnesses on our study of children's health. We also have scheduled the last 30 minutes, roughly, of today's meeting to do some committee business in camera, so that we can plan for the coming weeks.

Today's meeting is taking place in a hybrid format pursuant to the House order of June 23, 2022. I have a few comments for the benefit of witnesses and members. For those of you—

[Luc Thériault, Bloc, Montcalm, QC]

Mr. Chair, the sound needs to be adjusted. I can barely hear the interpreter and my microphone volume is turned up pretty high. If there's a change, I risk getting a shock. Also, unless people are having trouble hearing your voice, you are drowning out the interpreter.

So, I'd like us to adjust that before we start.

Thank you.

[The Chair, Liberal, Sean Casey]

Thank you, Mr. Thériault.

We're currently adjusting and hopefully this will be resolved soon.

All right. I think perhaps I will reread the opening until you tell me that everything is okay, and then we'll pick up where we left off.

This is meeting number 55 of the House of Commons Standing Committee on Health. Today we're meeting for an hour and a half with witnesses in our study of children's health, and then for approximately 30 minutes at the end of today's meeting, we will be in camera so that we can plan for the coming weeks. Today's meeting is taking place in a hybrid format pursuant to the House order of June 23, 2022.

I will now make a few comments that have become all too familiar for the members but maybe less so for our witnesses.

We have a couple of witnesses on Zoom today. For interpretation, you have the choice at the bottom of your screen of either floor, English or French audio.

Dr. McLaughlin, you can use the earpiece and just pick the desired channel.

Screenshots and taking photos of your screen are not permitted. The proceedings today will be broadcast on the House of Commons website.

As required by our routine motion, I want to inform the committee that all connection tests were done before the meeting and everything is fine in that regard.

I would now like to welcome the witnesses who are with us this afternoon.

We have Dr. Andrew Lynk, chair of the department of pediatrics in the faculty of medicine, Dalhousie University. Dr. Lynk is also the chief of pediatrics at the IWK Health Centre and past president of the Canadian Paediatric Society and Pediatric Chairs of Canada. He is joining us via video conference.

We have with us in the room Dr. Tom McLaughlin, pediatrician and clinical assistant professor at the University of British Columbia. Joining us by video conference is Professor Bruce Verchere, from the departments of surgery and pathology and laboratory medicine at the University of British Columbia.

Thank you all for taking the time to be with us today. Each of you has up to five minutes for an opening statement. We're going to begin with Dr. Lynk.

Welcome to the committee, Dr. Lynk. You now have the floor.

[Dr. Andrew Lynk, Chair, Department of Pediatrics, Faculty of Medicine, Dalhousie University, As an Individual]

Thank you, Mr. Chair, and thank you for having me today to present. I'm speaking on the traditional and unceded lands of the Mi'kmaq. We are all treaty people, and I know my department of pediatrics and the Canadian Paediatric Society and the Pediatric Chairs of Canada are all committed to advancing the truth and reconciliation recommendations as they apply to children and families and health—

[Luc Thériault, Bloc, Montcalm, QC]

Excuse me, Mr. Chair.

I'm really sorry, but the sound is far too loud in the room for me to follow. I can't hear the interpreter at all, despite the adjustments that were made. The sound is too loud in the room.

[The Chair, Liberal, Sean Casey]

Thank you, Mr. Thériault.

I apologize. The two-week break may have caused new problems. It will be taken care of right away.

Dr. Lynk, we're going to get you to restart. I'll reset the clock. If it comes across too loud in the room, we'll adjust as we go.

Please, go from the top, if you would. Thank you.

[Dr. Andrew Lynk]

Thank you, Mr. Chair.

I want to thank the committee for inviting me to present today.

I am speaking on the traditional, unceded lands of the Mi'kmaq here in Halifax, Nova Scotia. We are all treaty people. My department of pediatrics, the Canadian Paediatric Society and the Pediatric Chairs of Canada are all committed to advancing the truth and reconciliation recommendations as they apply to families, children, youth and health.

I'm going to build my remarks on some of the September 27 presentations that were made to this committee by my colleagues from the Canadian Paediatric Society, Children's Healthcare Canada and the Quebec association of pediatricians.

In addition to my current pediatric leadership roles, I spent 26 years practising as a community pediatrician on beautiful Cape Breton Island in Nova Scotia. I had the privilege to serve thousands of children from across the island—from first nations communities to children with cancer, children with autism and those who were born prematurely and with dozens of other conditions. I also served the one in four children and families living in poverty there.

I'll always remember a family who came to me. They were a young couple who were probably in their early twenties. Both had lived lives of adversity and were down on their luck. They had a new baby girl. They brought the baby into my office. After I had examined the baby and we had talked a bit, I asked them what they wanted for their daughter. They said they wanted a better life for her than they'd had. I said, “Okay.”

I've thought about that ever since, both as an individual practitioner and as a leader. Obviously I have to rely on my political colleagues to make some of the big system changes to make that happen.

It's quite clear that political discussions around health and health care always get mired in and confused over jurisdictional issues between the provinces and the federal government. It will probably always be like that—at least for the next little while.

I thought to myself, if I'm going to present today and if I were a member of Parliament or a senator, what would be the top 10 things I would focus on if I wanted to try to advance the well-being and health of children and youth here in the country? That's what I'm going to give you. It's my top 10 things in pretty quick succession. You've heard some of them before.

First, when it comes to mental health transfers, commit at least 25% of that, representative of the child and youth population, to services for child and youth health. We still have too many families, children and youth who cannot access mental health services in a timely fashion.

The second thing is one you've heard from the Canadian Medical Association. I think there's movement on it, but it's really crucial. The federal government should take the lead on a national health human resources centre. They could collate provincial data on the number, distribution and mix of nurses, physicians, mental health workers and allied health workers like lab techs and respiratory technicians, as well as the ages of those health workers and the number of trainees in various programs. This data would allow for planning to meet the increasing demands of the Canadian population now and in the years to come. We're dropping the ball on this one. We're flying blind. It reminds me of what happened to PPE during the early phases of the pandemic, when the provinces and the feds thought everybody had it under control and we didn't coordinate. This is a major issue.

Number three is that I would support the establishment of a national school meals transfer program, so that all children in schools receive a healthy breakfast and/or lunch without stigma. Currently, about one in five children—this is higher for newcomers and indigenous children—live in relative poverty. This would be a major benefit. Senator Rosemary Moodie and her colleagues have been trying to advance this. We would back this.

Number four is a really important one. In 2016, the federal government and Parliament passed the Canada child benefit. In Nova Scotia, in the year prior to that 2016 date, about one in five children lived in absolute poverty—below the market basket measure. In 2019, which is the last year I have data for, that's gone down to just one in nine. One in nine is still too many, but it has been almost cut in half. It's most likely because of the Canada child benefit. That needs to be indexed to real inflation.

When you look at the UNICEF rankings, right now Canada ranks 26 out of 38 rich OECD countries, with the worst child poverty rates. There's no vaccine for poverty. Doctors can't do this. We need everyone to work on this.

In the United Nations Convention on the Rights of the Child, which Canada passed in 1991, article 27 states that all children should be entitled to adequate standards of living.

Are we really living up to that if one in nine children are still living below the absolute poverty line?

There's another quick point from the Hospital for Sick Children, which was recently published in The Globe and Mail. They're talking about the complexity of the tax forms for poor families and families entitled to apply for some of these measures. They're saying they're difficult for some to navigate and asking if we can make them simpler.

Number five, I would encourage you to pass Bill C-252, which puts restrictions on the advertising of foods high in sugar, fat and salt to children under the age of 13.

Quebec has the lowest incidence of child obesity and children who are overweight in the country, and they have such regulations in law. We know that one in three children in Canada is obese or overweight. They have a higher risk of going on, as young adults and even as teenagers, to have problems with heart disease, liver disease and diabetes—and these are significant problems.

Number six, establish a child-friendly national pharmacare program. One in six families in this country find it difficult and struggle to pay for their children's prescriptions. Also, fund a national, evidence-based and pediatric-sensitive formulary that all practitioners in Canada can use.

Number seven, prioritize housing, water, health and educational opportunities for all indigenous children and youth. I have seen some of the benefits of this among our first nations communities in Cape Breton. When given the opportunity, these children and youth thrive, blossom and make huge contributions to our society.

Number eight, Canada should continue to take a leadership—

[The Chair, Liberal, Sean Casey]

Dr. Lynk, can I get you to wrap up? There will be lots of time for questions. Perhaps you could make some concluding comments.

Thanks.

[Dr. Andrew Lynk]

Sure.

The final point, I would say, Mr. Chair, is that we should establish a national child and youth commissioner in Canada to keep an eye on this issue. We have commissioners for grain and the environment and sports integrity. This is really essential. It's been tried before. Senator Moodie is still leading this initiative to include indigenous leadership in communities as well.

That concludes my remarks.

Thank you.

[The Chair, Liberal, Sean Casey]

You will get lots of chances to build on them through the rounds of questions.

Thank you very much.

Dr. McLaughlin, welcome to the committee. You have the floor.

[Dr. Tom McLaughlin, Pediatrician and Clinical Assistant Professor, University of British Columbia, As an Individual]

Thank you, Mr. Chair, for giving me the honour of being able to speak to the committee.

I'm Tom McLaughlin, a clinical assistant professor at the University of British Columbia and a pediatrician on the in-patient unit at BC Children's Hospital, where I take care of some of the sickest and most complex patients in the province. I also lead the Canadian Paediatric Society's work on pharmacare. I do research and policy work on children's drug insurance, pediatric formulations and, more recently, drug shortages.

Clinically, I have the best job in the world. Much like Dr. Lynk, I get to help children survive illness, thrive and develop into wonderful people. A big part of how I do that is by prescribing medications. Drugs allow us to easily treat once-lethal infections, help kids focus at school and, more recently, allow children with rare and devastating illnesses to live and lead better lives.

Despite this promise, Canadians struggle to access the drugs they need. Canada is the only rich country with universal health care that is without universal drug insurance. Almost one in 10 Canadians can't afford prescriptions, and almost a million reduce heat or food to afford drugs. This impacts all Canadians, but children face additional barriers to accessing drugs. This is because Canadian policies, largely federal policies, governing the development, approval and reimbursement of drugs, are largely designed for adults.

First, children have unique drug needs. Young children often need liquid medications. However, many drugs are available only as tablets. This means that drugs are compounded, where the tab is crushed and mixed with something by either a pharmacist or a caregiver. Almost 75% of prescriptions for kids are compounded. It might seem trivial to be talking about mixing pills with applesauce on the kitchen table, but this causes real harm. An eight-year-old boy in Mississauga named Andrew died a few years ago from a compounding error, and many more have been harmed by dosing errors.

Another unique issue in pediatrics is the prevalence of high-cost drugs for rare diseases. Despite the name, rare diseases are not rare in children. Almost one in 10 Canadians has a rare disease, and 70% are children.

A second issue is our regulatory system. Health Canada doesn't currently require manufacturers to apply for a pediatric indication or use for a drug, even when studies exist to support pediatric prescribing or where that use is expected—for example, for a new antibiotic. As a result, over 80% of pediatric prescriptions are off-label, meaning they're used in a way that hasn't been rigorously analyzed and approved by Health Canada.

Many drugs that are available in other countries aren't available in Canada at all, requiring importing drugs through our special access program. Even when drugs are approved, they aren't necessarily covered by provincial or federal public drug insurance programs that poorer families rely on.

Currently, federal agencies, or INESSS in Quebec, perform an economic analysis on new drugs, comparing their costs and impact on mortality and quality of life. For many pediatric conditions, this data just doesn't exist, so this analysis is very limited. It also doesn't capture the economic impacts outside the health care system. Treating a child with ADHD medications may help them stay at school and not require expensive educational supports, or it might allow a caregiver to go back to work. The harms of compounding aren't captured in reimbursement recommendations either.

All of this means that public drug formularies, the lists of covered drugs, can and do exclude important evidence-based drugs for kids. As a result, drug coverage for low-income families who depend on public formularies is extremely variable. In my own unpublished research, I show that children with common conditions like asthma and ADHD may have 100% of their drugs covered or 0%, up to $1,500 a year, depending on which province they live in.

What can the federal government do? The Canada pharmacare act, planned by the end of 2023, promises to ensure universal, comprehensive and affordable drug access for all Canadians, but it should also address the unique barriers that Canadian kids face accessing needed medications.

We should modernize Canada's regulatory system to reduce the need for off-label prescribing and increase access to safe liquid formulations. We should implement a pediatric rule, as many countries have done, whereby manufacturers must proactively develop pediatric studies and apply for a pediatric approval when they apply for an adult one.

We should support the national strategy for high-cost drugs for rare diseases with investment proportional to the disease burden in kids and support the pipeline for innovative treatments. I suspect my colleague, Dr. Verchere, will discuss this.

We should develop a pan-Canadian evidence-informed pediatric drug formulary, as Dr. Lynk said, that appropriately values liquid medications and the economic impacts of pediatric drugs outside the health care system. We should also work with provinces to ensure that a child's postal code doesn't impact their coverage for drugs.

Children deserve the same access to safe, effective and available drugs that adults have in Canada. I hope this committee can help us get there.

Thank you.

[The Chair, Liberal, Sean Casey]

Thank you, Dr. McLaughlin.

Next, from the University of British Columbia's departments of surgery, pathology and laboratory medicine, by video conference, we have Dr. Verchere.

You have the floor, Dr. Verchere. Welcome to the committee.

[Dr. Bruce Verchere, Professor, Departments of Surgery, Pathology and Laboratory Medicine, University of British Columbia, As an Individual]

Thank you, Mr. Chair.

I'm joining you all from Vancouver, where I live and work on the traditional and ancestral and unceded territories of the Musqueam, Squamish and Tsleil-Waututh peoples.

I'm a diabetes researcher at the BC Children's Hospital Research Institute, and I'm grateful for government support of my research through the Canadian Institutes of Health Research and through national networks such as the Stem Cell Network.

I'd like to talk to you today about diabetes in children, its huge health burden and impact, the importance of research, and the opportunities we have in Canada as world leaders in diabetes research and care.

Diabetes is currently an incurable disease and a tremendous burden on children and families. Children who develop diabetes have it for the rest of their lives. They face a lifetime of checking blood sugar and administering insulin every day, all the while living with fear, in danger of low blood sugars. It's a life-changing disease.

There are two main forms of diabetes, type 1, in which the insulin-producing cells of the pancreas are destroyed by the immune system, and type 2, a metabolic disease associated with obesity and aging.

While one can develop diabetes at any age, children are particularly impacted by type 1 diabetes, as it is most commonly diagnosed in children and youth. Canada has one of the highest incidences of type 1 diabetes in the world, and it is increasing in prevalence each year. Type 2 diabetes impacts more than three million Canadians and is normally seen in adults but is disturbingly being found increasingly in youth, associated with the rising prevalence of obesity. This form of the disease disproportionately affects children of certain ethnic groups, including those of South Asian descent and, in particular, indigenous youth.

Because children live with diabetes for many years, they are at a high risk for complications later in life, including kidney failure and blindness. Children with diabetes are also at an increased risk for mental health problems, including depression and anxiety. The burden to children, families and our health care system is immense.

I have two main messages for you today about addressing the burden of diabetes in children and the important role government can play. The first relates to the implementation and funding for the national framework for diabetes, and the second is regarding continued support of diabetes research in Canada.

First, speaking to Canada's national diabetes framework, which was tabled just a few months ago, this is a huge milestone for all people living with diabetes in Canada, including children. I want to acknowledge the vision and effort of those who brought this to reality, including the health minister; MP Sonia Sidhu, chair of the all-party diabetes caucus; and many stakeholders who've contributed.

The diabetes framework provides a strategy for government to improve health outcomes for people with diabetes, including children, through several mechanisms. These include ensuring Canadians have access to resources for disease management, increasing awareness of diabetes for all Canadians, and funding and promoting innovative and impactful research.

The national framework for diabetes has the potential to have a tremendous impact on the lives of thousands of Canadian children living with diabetes and their families. I urge the government to ensure the resources are provided to fund and implement this framework, beginning in 2023.

Second, I'd like to talk briefly about the importance and promise of diabetes research to children's health. Canada has a truly remarkable research history in diabetes, and Canadian diabetes research has never been stronger. In 2021 we celebrated the 100th anniversary of the discovery of insulin by Banting and Best, a miraculous Canadian medical research discovery that allowed children with type 1 diabetes to live long lives. However, even Sir Frederick Banting said that insulin is not a cure for type 1 diabetes. More research is still needed.

Since the discovery of insulin, Canadian diabetes researchers have made pioneering contributions, including the development of drugs in common use today for type 2 diabetes and weight loss, and pancreatic islet cell transplantation in type 1 diabetes and, more recently, in stem cell-based therapies for type 1 diabetes. It's important to point out that the development of these therapies builds upon foundational research and took many years to move from discovery in the lab to the clinic. Tri-council funding, particularly from the CIHR, ensures that the best diabetes research is funded across the country, that the diabetes research capacity in Canada remains strong and that new discoveries will find their way into new therapies.

In 2021 the Canadian government invested in diabetes research through the CIHR Institute of Nutrition, Metabolism and Diabetes, in partnership with other funders, including the JDRF, the Stem Cell Network and Diabetes Canada. This investment has had a transformative impact on diabetes research in Canada, including the creation of national teams and networks working together to understand the cause of diabetes and find new treatment. I feel it's important that the momentum built by these national diabetes research teams not be lost. I urge government to continue to support diabetes research through these special initiatives and innovative partnerships, while maintaining and increasing tri-council funding for foundational research.

To conclude, diabetes is a life-changing disease and a tremendous health burden, affecting thousands of Canadian children. I encourage the government, through implementation and funding of the national diabetes framework and increased research funding, to continue to address this significant child health issue.

Thank you for allowing me to speak to you this morning. I'm happy to answer any questions.

[The Chair, Liberal, Sean Casey]

Thank you, Professor Verchere.

We're going to begin with rounds of questions now, starting with the Conservatives.

Dr. Ellis, you have six minutes.

[Stephen Ellis, Conservative, Cumberland—Colchester, NS]

Thank you, Mr. Chair, and thank you to the witnesses for being here today. As you all may or may not know, we are coming close to the end of our study on children's health, which has become very large. As all of you pointed out, it's a very complex topic in terms of the breadth and the actual number of things that can influence children's health.

That being said, Dr. Lynk, I will start with you. You talked a bit about mental health transfers and 25% of those transfers coming to children. Certainly, that's been part of our difficulty. The Canada mental health transfer has not been forthcoming from this government, even though it was promised, but that's a whole other kettle of fish.

Maybe you could talk a little about resiliency in kids. Is there an opportunity to speak about that in the education system, as well?

[Dr. Andrew Lynk]

That's a great point. Mental health care is just one aspect of mental health. Obviously, families that are functioning well, schools that are functioning well and all those support systems are really important, too.

UNICEF does a very good job. If you look at its 2020 report card for Canada, it had about 125 different indicators. It measures some of these specific things around resiliency and how kids are doing.

I was a little shocked, actually. A quarter of children are feeling sadness or hopelessness for long periods of time. Only 50% rate their lives high on life satisfaction. A quarter of kids go to bed or school hungry, at least sometimes, because of a lack of household food. They go through all those different things.

If I'm understanding your question correctly, Dr. Ellis, resilience is more than just health care. It's the family income. It's how well you're supported at school. It's how well communities function. It's about the environment. It's complicated, obviously, but that's why I think I was trying to make sure that at least if there were mental health transfers going to the provinces, the provinces should be accountable to make sure some of that money is dedicated to child and youth services.

With that—and I agree with the current government that provinces are responsible—if they are going to get the money, I think they need to publish outcomes, wait times and how people are doing. That's a wonderful opportunity for us to compare between provinces, because provinces that are doing better—and most provinces are doing better in something than other provinces, for various reasons.... It's an opportunity to learn, to share and to scale things up.

I don't know if I have answered your question completely, but you're right. Resilience is important, and it's supported in many different ways.

[Stephen Ellis, Conservative, Cumberland—Colchester, NS]

Thank you very much, Dr. Lynk.

Dr. McLaughlin, you talked a bit about children's medications. I will just focus on that. There's another topic I will come back to later, perhaps, if we have time.

Certainly, when we look at the drugshortagescanada.ca website, we still understand that children's Tylenol and children's ibuprofen are short, and in my estimation every first-line antibiotic that would be given orally to children is also in short supply in Canada.

I am wondering if you have any comment on that. Have we seen this before? Do you have any ideas around the solutions?

[Dr. Tom McLaughlin]

That's an excellent question.

You're right. Drug shortages actually have existed beyond this current round of, specifically, pediatric shortages. That relates to some of those regulatory issues that I talked about.

There are many drugs that are not widely available in Canada, so through our special access program we essentially import and do one-off importation for them as a routine matter. That is something that continues to this day. It has been worsened by the pandemic in terms of common children's medications like Tylenol, Advil and antibiotics. I think it gets back to, in large part, what I was talking about related to regulation.

We struggle with having child-friendly drugs available in Canada, because our regulatory system doesn't push manufacturers or support manufacturers to make those drugs widely available, so there's more we can do on that through incentives and through mandates to be applying for needed drugs.

[Stephen Ellis, Conservative, Cumberland—Colchester, NS]

Thank you very much for that.

You talked a bit about rare diseases. Certainly, we know very clearly that Canada is the only G7 country that doesn't have a rare disease strategy, and this disproportionately affects children. It's probably not exactly in your area of expertise, but I'm wondering if you might have some comments around rare diseases, the definition and how you think Canada might proceed with a rare disease strategy.

[Dr. Tom McLaughlin]

The reality is that pediatrics, as a field, has evolved. We have largely become a specialty of rare diseases. At my hospital, I work on the in-patient ward. It's the tertiary referral centre for the province of British Columbia. A quarter to a third of all patient beds are filled by a child with a rare disease. Clinically, this is in every pediatrician's wheelhouse.

I agree. We need to have dedicated leadership. This is a role for the federal government. It can serve an important coordinating role, as it does in many other areas of health care. We should have a national strategy for rare diseases. It should involve work on our drug development pipeline, our regulatory system, and how best to support and reimburse these medications, which can be very expensive.

I certainly agree that we need a national strategy.

[The Chair, Liberal, Sean Casey]

Thank you, Dr. McLaughlin and Dr. Ellis.

Next, we have Mr. van Koeverden for six minutes.

[Adam van Koeverden, Liberal, Milton, ON]

Thank you very much, Mr. Chair, and thank you, witnesses, for coming today. Your testimony has been very interesting and valuable in producing a report to Parliament on children's health.

Dr. Lynk, you started listing your top 10 priorities, but I think you got through only eight. Before I ask a question, I'll provide you with an opportunity to list the other two. I was taking notes, and my list is incomplete.

[Dr. Andrew Lynk]

I appreciate that. I submitted my notes.

I'm sorry. Perhaps I should have cut it down to eight, but here are the other two.

Firstly, we need to continue to take a leadership role in global climate change and greenhouse gases. If we don't address that one, the next generations are going to be in big trouble, I think.

Secondly, I'll go back to what Dr. Verchere was talking about: Invest in health research. Keep an eye on those budgets. Canada has a stellar record of contributing globally to new innovation and knowledge that are actionable and make a difference to people's lives. Through our CIHR, there's a specific institute of human development, child and youth health. Make sure we are well funded, so we can support some of the world's best scientific minds and mentor the next generation of clinicians and scientists.

I remember being in Cape Breton, when I started as a medical student, and looking after kids with cancer who died or had a high chance of dying. By the time I started practising and was well into my first decade, these kids sometimes had 90% survival rates. That's all because of clinical trials and research. It's the same for diabetes, to Dr. Verchere's point. We have all these new tools because people are doing research to help us, as a health care community.

Those were the other two things I wanted to mention.

[Adam van Koeverden, Liberal, Milton, ON]

Thanks. It's a very good list. There are a couple that stand out for me. There is current work on this committee around marketing to kids and a national child and youth commissioner—that one is spoken about on this committee, around health care for children, as well. Thank you for underscoring them. I would say it's a very comprehensive list of suggestions.

I'll move on to Dr. McLaughlin.

We talked a bit about rare diseases. February 28 was the rare disease advocacy day. I used to participate with a friend named Simon Ibell, who started something called “Be Fair 2 Rare”, and it got me thinking more about rare disease advocacy prior to becoming a politician. I think I've mentioned him before on this committee. He was a special guy who left us too soon. His work was exceptional.

I'm wondering to what degree you've been engaged in Canada's rare disease strategy and rare disease drug strategy. I spoke at the CORD conference last year. I had an opportunity to hear from advocates directly. They mentioned that work is ongoing, but it's complicated, obviously, because it's so far-reaching.

Do you have any comments on Canada's future rare disease strategy, as it pertains to drugs or treatment?

[Dr. Tom McLaughlin]

That's an excellent question.

The future of pediatric drugs is going to be something we all have to grapple with. It's in the name. These are expensive drugs for rare diseases.

By and large, these are drugs for a very small population. It is relatively difficult to get high-quality evidence in the way we used to do it, through randomized controlled trials and that sort of thing. We often have imperfect evidence as part of our development strategy, and that also impacts reimbursement. Should we be paying $100,000, $1 million or $2 million for medication for a child with a significant rare disease?

It's important to balance lots of different things. The magnitude of the disease matters. If it is a child whose life is threatened, I think it makes sense to have more focused and specific end points that may be about that child. It might be that we cover a drug and then we monitor that child's symptoms. We need to develop the evidence infrastructure on how to follow evidence for a very small population.

That also impacts reimbursement decisions, again. Our current systems, CADTH and INESSS, account for patient perspectives and for the magnitude of diseases. That needs to be further fleshed out.

At a federal level and at a governmental level, there are lots of dollars involved. What amount of funding should we be supporting this with? I would say this is something that should be commensurate with the disease burden.

All those things should be part of a rare disease strategy. I would encourage everyone to look up CORD and other national advocacy organizations that have very fleshed-out positions on this.