Evidence of meeting #118 for Health in the 42nd Parliament, 1st Session. (The original version is on Parliament’s site, as are the minutes.) The winning word was diseases.

A recording is available from Parliament.

On the agenda

MPs speaking

Also speaking

Michael Brudno  Professor and Scientific Director, Centre for Computational Medicine, Hospital for Sick Children, As an Individual
Ian Stedman  Osgoode Hall Law School, York University, As an Individual
Alex MacKenzie  Clinician Scientist, Children's Hospital of Eastern Ontario (CHEO)
Joel Lexchin  Professor Emeritus, School of Health Policy and Management, York University, As an Individual
Stacey Silverberg  Stakeholder Engagement Manager, Government Affairs and Market Access, Janssen Inc. Pharmaceutical Companies of Johnson & Johnson
Raj Grewal  Brampton East, Lib.
Jacqueline Dobson  Government Affairs and Policy Manager, Government Affairs and Market Access, Janssen Inc. Pharmaceutical Companies of Johnson & Johnson

October 25th, 2018 / 8:30 a.m.

Liberal

The Chair Liberal Bill Casey

Welcome, everybody, to the 118th meeting of the Standing Committee on Health.

Today we're going to continue our study on rare diseases and disorders. We have four groups to make opening statements. We welcome you all.

Today, speaking as individuals, we have Dr. Michael Brudno, Professor and Scientific Director, Centre for Computational Medicine, Hospital for Sick Children; together with Ian Stedman, Osgoode Hall Law School, York University. From the Children's Hospital of Eastern Ontario, we have Dr. Alex MacKenzie, Clinician Scientist. By video conference, speaking as an individual, we have from Toronto Dr. Joel Lexchin, Professor Emeritus, School of Health Policy and Management, York University. From Janssen Inc., a Pharmaceutical Company of Johnson and Johnson, we have Stacey Silverberg, Stakeholder Engagement Manager, Government Affairs and Market Access; and Jacqueline Dobson, Government Affairs and Policy Manager, Government Affairs and Market Access.

We'll open in that order and ask Dr. Brudno to begin.

8:30 a.m.

Dr. Michael Brudno Professor and Scientific Director, Centre for Computational Medicine, Hospital for Sick Children, As an Individual

Good morning, and thank you for the opportunity to speak to you today about genetic testing and its role in enabling access to therapies for Canadians affected by rare disease.

I'm a professor at the University of Toronto's Department of Computer Science and the director of the Centre for Computational Medicine at the Hospital for Sick Children. My team and I work to develop computer software and algorithms to assist rare disease clinicians and patients, and we have been involved in many Canadian and international rare disease genomics efforts.

With me today is Ian Stedman. He's a rare disease patient and parent who went through a 32-year-long diagnostic odyssey to get an answer for him and for his family, and he will talk about the cost of being undiagnosed and the value that the diagnosis brought to him.

To begin, I would like to talk about the importance of genetic diagnosis for rare diseases. Our genome contains 20,000 genes, and 80% of rare diseases are caused by changes or mutations in one or a few of these. We now have technology that can decipher the whole genome, identify disease-causing mutations and differentiate between the greater than 7,000 diseases for as little as $1,000.

As you heard from Marc LePage, the President and CEO of Genome Canada, on October 16, countries around the world are embracing this technology. The U.K. government plans to decode the genomes of five million patients as part of a national initiative that builds upon the completed “100,000 Genomes” study. The United States just announced a $1.5-billion program through the NIH called “All of Us”, which will combine the genomes of one million Americans with data on their health, environment and lifestyle to identify optimal treatments.

These programs exist because precision medicine will lead to significant cost savings through early intervention and treatment. This is especially true for rare diseases, where patients undergo a significant diagnostic odyssey. Recent studies demonstrate that early diagnosis of rare disease patients through genome-wide testing leads to cost savings of as much as $8,000 per patient per year, in a health care system very similar to Canada's. The reason for these savings is that patients without a diagnosis do not save the health system money by going untreated. They cost the system money because they are the cause of unnecessary visits, investigations and interventions.

Ian can illustrate this with his story.

8:30 a.m.

Ian Stedman Osgoode Hall Law School, York University, As an Individual

Good morning and thank you.

As Professor Brudno said, I went 32 years without a diagnosis. Throughout the time I was sick, I had arthritis, headaches, full body rash, fevers and a generally low sense of self-worth, to be honest. I went to my doctor's office—my family doctor—about 200 times before I was 18 years old. I went to see over 30 specialists, had countless ER visits and tons of tests. The answer was always, “We don't know. Try these drugs and we'll see if we can treat your symptoms and let you live.”

Everything changed in 2012 when my daughter was born, because she was born sick as well. We ended up at SickKids and we got genetic testing. It turns out that we have a one-in-a-million disease called Muckle-Wells syndrome. It's caused by a single gene mutation.

That allowed us to get access to medicine, and the medicine, like a light switch, turned off all the symptoms overnight. There has been no looking back—32 years of struggle disappeared like it never happened—so I get to live the rest of my life disease-free. My daughter was only one then. She's six now. The only way she knows she's sick is that a nurse comes to our house every eight weeks and gives her a needle. Otherwise, it's smooth sailing.

Diagnostics, genetics, it's amazing, life changing. It's sad to think that we're the lucky ones now. Hopefully, that isn't the case going forward.

Thank you.

8:30 a.m.

Professor and Scientific Director, Centre for Computational Medicine, Hospital for Sick Children, As an Individual

Dr. Michael Brudno

Ian is a great example of why genetic testing is so important and why we need to enable access to genetic testing.

Since receiving treatment, Ian has become one of Canada's scholars in the area of parliamentary ethics law and has published extensively on the topic. He also holds a fellowship on artificial intelligence, ethics and law and is researching how artificial intelligence solutions can be implemented and regulated in our society. I would submit that the cost of his testing and treatment pales in comparison to his contributions to Canadian society. He and countless patients like him are why genetic testing is so important, and why it's important to help eliminate barriers to access to treatment.

Genetic diagnosis can open the path towards therapy, as it did for Ian; however, the diagnosis must be timely, as one can miss the optimal treatment window. Ian, for example, had a one-third chance of dying from amyloidosis by the time he was 35 if he did not receive treatment.

In many other diseases, the window for treatment is even smaller. As a result, many hospitals in the U.S. are now experimenting with rapid genomic testing, with a result within 48 hours for patients in neonatal intensive care units. This also has been shown to actually reduce overall costs because of a reduction in the myriad of tests that would otherwise follow.

I want to now turn to the issue of the costs of these tests and drugs. While the sticker shock of the price of genetic testing and the many rare disease therapies is understandable, it must be compared to the costs on the health system for patients who are not diagnosed and who do not receive proper treatment.

Dr. MacKenzie will speak more about the costs of treatment, but these costs do not grow linearly with the number of patients. Because so much of the cost of a drug is sunk research costs, the more patients identified brings down the per-patient costs, while the benefits multiply proportionately.

The key to this is identifying and aggregating information for all rare disease patients. Canada is a small country in terms of population, and people with a rare disease are spread all across it. Our efforts on precision medicine in rare diseases, including early genetic testing, have to be national in scope.

In summary, I would like to make three recommendations to the committee.

First, when considering the costs of treatments and interventions, we always think about the aggregate costs and also the aggregate benefits at the economic, societal and personal levels.

Next, genetic testing for people with rare and/or undiagnosed diseases needs to be given a priority in any strategy that is aimed at removing barriers to access to treatment. This testing has been proven to reduce overall costs and can open the door to therapies, as it did for Ian.

Finally, because so few people have any individual rare disease and higher numbers significantly reduce costs, a national strategy to address access to rare disease drugs in particular and precision medicine in rare diseases more broadly needs to be national in scope.

Thank you. We'll be happy to take your questions.

8:35 a.m.

Liberal

The Chair Liberal Bill Casey

That was six minutes and 54 seconds.

Thank you for that personal testimony. That means a lot to us. It helps us to get a grasp on this.

Now we'll go to Dr. Alex MacKenzie.

8:35 a.m.

Dr. Alex MacKenzie Clinician Scientist, Children's Hospital of Eastern Ontario (CHEO)

That's a wonderful job, guys. That's a hard act to follow.

I'm a clinician scientist at CHEO. I work on rare diseases. In deference to the Dutch, I'm going to keep it pretty brief and open it up for more questions.

I would like to run the numbers. This is probably old news to the committee but I'll give you a numerical overview.

The number of rare disorders is 7,000, as Michael said. That number may well grow with time but that's our estimate right now. Roughly one million Canadians are affected. One in 12 is bandied about. We think that is an overestimate. It's closer to 2% to 3%. Nonetheless roughly one million Canadians are affected.

Over 50% are children. Under 50%—less than a half— have a diagnosis now. When I look at the in-patients on my ward at CHEO, I see that roughly one-third have rare diseases. These diseases are responsible for one-third of deaths in the first year of life, and one-third of children with rare diseases will die before they reach the age of five. That's to give you a sense of the impact of rare diseases.

The proportion of rare diseases for which there is no therapy is 94%. We do not have a therapy for most of these disorders.

Perhaps the most telling statistic is that when you look at the proportion of general years of life lost for rare diseases, it's around 4.6%. That's years of life lost in Canadian society. For infectious diseases, the number is around 1.4% or 1.6%. For diabetes, it's only 2.6%. It's really dramatic. I think the reason for this is that it takes life early on, so that carries a disproportionate impact.

As Michael said, we live in revolutionary times right now with economic DNA sequencing. The first responders to this are genomes. Everybody in this room will be in our electronic medical health record within a decade—no question. It's going to impact how we're perceived at risk of disease, prevention and therapeutic options. Whether you call it genomic, precision or personalized medicine, it's a revolution.

The children with rare diseases are really the first responders to this revolution. I would say parenthetically that getting this right is not just good for children with rare diseases; it's good for all of us.

We have in the guise of Michael, whose Matchmaker Exchange platform has been adopted by Johns Hopkins in Baltimore, Cambridge University and the National Institutes of Health in Washington, an absolute global leader in rare disease diagnosis. He's too modest to say so.

With Kym Boycott, my colleague leading Care4Rare at the children's hospital, we have a pan-Canadian consortium where we've diagnosed rare diseases at an unprecedented rate—almost 200 new rare disease genes identified. There's no other developed country that has really done a national, full-on assault like that. I'm not sure if it's the submersible Canadian ego, our desire to work in committees, but we really are number one as far as going coast to coast to coast and addressing this problem.

As we heard so eloquently from Ian, a diagnosis really is a form of therapy—absolutely critical. When Mark LePage talked to you about their Genome Canada initiative to do 30,000 rare disease genomes, that's a tremendous project with huge potential. It's a bit like Dickens: the best of times and the worst of times. It's incredibly exciting what's going on as far as diagnosis is concerned, but the lack of therapies and also the cost of therapies must be mentioned.

My laboratory works on repurposing drugs. We take clinically approved drugs and look at indications for rare diseases. In working over the last five years, we've identified five potential therapies for diseases involving epilepsy, aortic aneurysms, nerve degeneration and muscular dystrophy. These drugs cost as low as a dollar a day, sometimes less than that.

Dr. Vicky Siu, a colleague in London, is giving histidine to the old Amish individuals with a seizure disorder. Clara van Karnebeek, at UBC, has started a web page to show doctors how to use dietary modulation for rare diseases. There are other ways of addressing rare diseases than new drugs.

But obviously new drugs will be needed and therein lies the rub. What can Canada collectively do about the problem of the cost of these drugs?

Mike laid out very well the economic argument for looking at them perhaps differently from other drugs.

I would just say a few things. I think we need to show a united front. Right now, Canada is alone in the OECD and the developed world in having a balkanized provincial approach. This is shown in how, after Switzerland and the U.S., we pay the third most in the world for these drugs. We have the pCPA, but we really need a stronger policy lever in this regard. We need to start prescribing biosimilars and generics more than we do now. These are under-prescribed.

The U.S. introduced legislation about 15 years ago to accelerate biosimilars. These are drugs that are the biologicals, the antibodies, etc., that aren't exactly the FDA-approved drugs, but are the generic form. This legislation in the States has made a difference.

We need to look at things such as managed access programs, where the companies generate data as we go along with the therapy so we can assess who should get their drugs, based on hard evidence, and who should actually stop the medication. It is one of the most difficult things to face—at what time do you actually stop a medication when it's not being effective?

For my part, I think we may need to rethink how we go after rare diseases. Right now, it's academics like me working in labs collaborating with big pharma and biotech companies, but I think we need the almost Thomas Edison or Henry Ford plant approach to rare diseases in a generalized fashion, where you have open access, much like what Aled Edwards talked about. We need a transparent factory approach where you're making gene vectors, which go to specific tissues that you can put different genes into, novel ways of isolating proteins in an industrial fashion, a really systematic approach to repurposing drugs such as what we did, the idea of a universal donor cell where you can actually generate cells that do not induce immune responses, such as those that Jonathan Pitre, the butterfly boy, succumbed to just last year.

There are a number of thoughtful systematic approaches one can use to tackle rare disease therapy generation.

8:40 a.m.

Liberal

The Chair Liberal Bill Casey

I have to ask you to wind up, there.

8:40 a.m.

Clinician Scientist, Children's Hospital of Eastern Ontario (CHEO)

Dr. Alex MacKenzie

Yes, and that's it. I just want to say that—

8:40 a.m.

Liberal

The Chair Liberal Bill Casey

You wound up; that's perfect.

8:40 a.m.

Clinician Scientist, Children's Hospital of Eastern Ontario (CHEO)

Dr. Alex MacKenzie

—basically, it will sort itself out one way or the other, but I think we need to tackle this problem.

8:40 a.m.

Liberal

The Chair Liberal Bill Casey

Thank you very much.

Now we go by video conference to Dr. Lexchin.

Also, I should point out that some of this will no doubt be in French, so we have interpretation services available. You will need them eventually.

Okay.

8:40 a.m.

Dr. Joel Lexchin Professor Emeritus, School of Health Policy and Management, York University, As an Individual

Thank you very much for the opportunity to appear. I'm giving this presentation not just on my behalf but on behalf of the other 10 people who signed the written brief.

This presentation is going to concentrate on drugs for rare diseases, and I'm just going to go through the recommendations that we have.

First of all, if there is a policy around rare diseases, it should not be based on the assumption that drugs are not commercially viable just because there are small numbers of people who have the disease. In order to prove that our research and development into drugs for rare diseases is not commercially viable, companies should have to divulge their R and D costs and show that these costs are not commercially acceptable, given the number of people in Canada that the company expects to treat with the medicine and the price it plans to charge.

Second, based on U.S. figures for the number of drugs for rare diseases that have been approved recently—and recently in the past half-dozen years or so, 37% of drugs in the U.S. have been labelled as drugs for rare disorders—there may not actually be any need for Canada to provide monetary incentives for research into rare diseases. However, it may be appropriate for Health Canada to set up a distinct regulatory path for these drugs. As an alternative to providing companies with monetary incentives, the Canadian government should consider investing more money into research into these diseases.

Third, if the overall prescribing rate for an orphan drug exceeds the number of people with the rare disease, then the drug should lose its orphan status.

Fourth, when more than one disease is caused by the same pathophysiological mechanism, and therefore could be treated but with the same drug, then each disease should not be considered separately when deciding whether a drug should receive orphan drug status.

Fifth, Health Canada needs to guard against the acceptance of biomarker subsets of disease and limit the use of the orphan drug designation to situations where the drugs are truly distinct. For example, if the same drug treats more than one genetic abnormality that causes the same disease—so we have a drug for cystic fibrosis that's being used to treat cystic fibrosis caused by two different genetic mutations—then each cause of the disease should not be treated as a separate rare disease.

Sixth, while small numbers of people with rare diseases place limitations on the design of clinical trials, Health Canada should demand the highest degree of rigour possible in these trials. In addition, post-market clinical trials should be a requirement when the evidence regarding either clinical benefit or safety is unclear, and Health Canada should monitor and publicly report on the progress of these post-market trials.

Next—and this is a point that's already been made—the assumption that one in 12 people have a rare disease in Canada is unreliable and should not be used to form Canadian policy about drugs for rare diseases. Any definition of a rare disease should not just take into account how frequently it occurs, but also needs to incorporate the element of severity. In other words, it should occur infrequently and also be severely debilitating.

Finally, any recommendation that the House of Commons standing committee makes based on the testimony of groups representing patients with a rare disease should take into account conflicts of interest that these groups may have with companies that would benefit from any policy that's developed.

Thank you.

8:45 a.m.

Liberal

The Chair Liberal Bill Casey

Thank you very much. I appreciate your concise comments.

Now we'll go to Janssen Inc., Pharmaceutical Companies of Johnson & Johnson, for seven minutes.

8:45 a.m.

Stacey Silverberg Stakeholder Engagement Manager, Government Affairs and Market Access, Janssen Inc. Pharmaceutical Companies of Johnson & Johnson

Thank you.

Honourable members, I would like to thank the committee for inviting me here today to speak with you regarding your study into barriers to access to treatment and drugs for Canadians affected by rare disorders. As I begin, I would like to share a bit of background as to why I've been afforded this particular opportunity to address all of you today.

I work for Janssen, which is part of the Johnson & Johnson family. I have worked with them for four years; however, I do come to you today with 25 years' experience in the innovative pharmaceutical industry. While I've had a variety of roles through my long and rewarding career in this sector, I'm so incredibly grateful for the time spent meeting patients, working with patients and being part of their journeys, especially when I've been able to personally witness the impact we have had, and will continue to have, on their overall survival, their quality of life and, yes, even in some cases, their cure.

Over my tenure in this industry, I have been able to observe patients with schizophrenia reintegrate into society and live more productive lives than they or their carers ever could have imagined. I've stood witness to cancer patients being put on a new treatment that has literally breathed new life into them. In fact, cancer can now be considered in some cases a chronic disease versus a death sentence, once again because of access to innovation and because of this innovation. These are just a couple of examples of some types of patients I've had the privilege to work with over the last many years.

Today, I would like to ask all of you to ensure that we don't do anything to unintentionally put barriers in the way of these patients, and truly look for ways to additionally facilitate their ability to access medications as one part of their treatment plan. I would also ask that you look to afford the same type of access to innovation for patients living with rare diseases, a very vulnerable population.

I will share with you a brief overview of Janssen's work in providing access to innovative medicines, specifically focusing on the rare disease population. I will speak to the need for an orphan drug regulatory framework, which should be designed to enable development and availability of medicines for rare disorders. Finally, I will provide an overview of concerns we have with two ongoing government initiatives that we believe may limit access to new innovative drugs for rare diseases.

In Canada, as you may be aware, we don't have a formal rare disease framework or strategy, but it is important to note the “one in 12” number that's been noted today. As well, that number comes from the Canadian Organization for Rare Disorders. One in 12 Canadians lives with a rare disorder. It is incumbent on us, as Canadians, to help this at-risk population.

Back in June, 2017, Johnson & Johnson acquired Actelion, a leading innovator in the rare disease pulmonary arterial hypertension, or PAH population. PAH is a rare disease that I will address today. For further details, in the interest of time, please refer to the written documentation we submitted to you earlier.

PAH is a progressive disease that can strike at any time and does not discriminate. It is a challenging disease to diagnose, and often these patients languish in the system. The smallest everyday tasks that you and I take for granted seem to a PAH patient to be like climbing Mount Everest. As mentioned, it's a progressive disease and it can lead to fatal consequences. If left untreated, these patients live an average of two to three years from the time of diagnosis.

Although there is no cure for PAH, there are treatment options, and due to the innovative medicines approved in Canada, Canadians can live longer and healthier lives. These treatments have meant that now 50% of patients survive five years or more from time of diagnosis, but access to these medicines is limited, often with criteria to meet and delays in approval. Some are not even reimbursed by public payers. These are crucial medicines for those who need them and delays to access can be devastating.

A strategy for rare diseases would help address these issues, better defining what treatments should be made available to patients, to ensure there is funding available to pay for them.

There are barriers to access that arise because of uncoordinated and uneven approaches across the country to both the regulation and reimbursement of these medicines. To address this, Janssen encourages the federal government to implement an orphan drug regulatory framework that would offer additional support to encourage the development and availability of orphan drugs in Canada. Recent government efforts, including the orphan drug regulatory approach, are very encouraging, but this framework would further solidify the government's approach to accessing drugs for rare diseases.

Additionally, given there are uneven and unequal funding approaches to drugs for rare diseases across the country, Janssen encourages the federal government to fund a separate drug program specifically for rare diseases. This could be done in parallel with the ongoing work related to national pharmacare, and would ensure that funding would be available when Canadians need these medically necessary medicines.

In the same vein, we are concerned that two ongoing initiatives of the federal government could further exacerbate these issues of access: the proposed reforms to the Patented Medicines Prices Review Board, PMPRB, and the implementation of national pharmacare. We recommend for both initiatives that the federal government closely consider the potential implications to ensure that these do not have unintended negative consequences.

Regarding the PMPRB changes, we are concerned that the new regulations, as they stand, could decrease access to new innovative drugs, including drugs for rare diseases. The updated regulations may result in Canada not being an early-launch country for drugs, which would slow access to innovative medicines that Canadians need. It has been seen that fewer medicines are launched in countries with prices at the median of the new proposed comparator countries, including medicines that target rare diseases.

We encourage the federal government, as it develops a national pharmacare policy, to consider potential implications for Canadians with rare diseases. Coverage for new innovative medicines is essential to ensure the best care for rare diseases, but moving to a single-payer public plan may impede this. Accessing new medicines is time-sensitive for patients with rare diseases. It is vitally important that the existing public and private mix of drug plans be maintained to ensure that the latest medicines are available for Canadians.

Finally, both changes could result in fewer clinical trials in Canada. For those with rare diseases, and especially life-threatening illnesses, clinical trials are the earliest means to getting innovation and hope to patients. In Canada we value health. In Canada we value safety. Let us ensure that these proposed policy changes do not put at risk some of the core values that define this wonderful country.

This study you have so boldly undertaken can have a substantial impact on three million Canadians. As you continue these deliberations, I urge committee members to take a close look at concerns raised by me and others regarding the PMPRB reforms and national pharmacare to ensure that the implementation of these programs does not have unintended negative consequences that could limit access to new innovative medicines for rare diseases.

I would like to thank the committee for providing Janssen with the opportunity to speak today. It has truly been an honour.

8:55 a.m.

Liberal

The Chair Liberal Bill Casey

Thank you very much.

Thank you, all, for your opening remarks.

Now we'll go to our seven-minute questioning round, starting with Mr. Grewal.

8:55 a.m.

Raj Grewal Brampton East, Lib.

Thank you, Mr. Chair.

Thank you to the witnesses for coming here today. I'll be splitting my time with the honourable member to my left.

Dr. MacKenzie, you put the number at around one million, and everybody else, including the documents I have, puts it at about 2.8 million Canadians suffering from rare diseases.

Can you elaborate on why your estimate would be 1.8 million?

8:55 a.m.

Clinician Scientist, Children's Hospital of Eastern Ontario (CHEO)

Dr. Alex MacKenzie

That's roughly 2% to 3% up. It's just based upon our personal observation at the CHEO genetics clinic and extrapolations therefrom.

If one drills down in the literature—and perhaps Joel might have a comment, because both he and I are saying that one in 12 is perhaps a bit inflated. That in no way is to undermine the severity or seriousness of rare diseases. I just think we need to be as careful as we can about the numbers.

If you look at Australian, Belgian and Italian studies, in which they have done this well, it comes in at roughly that benchmark. Given the diaspora that makes us up, there is no reason to anticipate, from a genetic point of view, that it would be any different from the 2% to 3%.

I think there may be a bit more data. I tried to drill down on the one in 12 from the CORD web page, but I am unable to find the source of those numbers.

I'm not sure if Joel—

8:55 a.m.

Brampton East, Lib.

Raj Grewal

I think if you want something that everybody on the panel can agree with, identifying the number of Canadians suffering from rare diseases is probably the number one priority in order to address the problem.

8:55 a.m.

Clinician Scientist, Children's Hospital of Eastern Ontario (CHEO)

Dr. Alex MacKenzie

I would agree, and I think if we go ahead with the Genome Canada project to do the 30,000 rare disease genomes, that will be a step in the right direction, as would be other general genomic sequencing projects.

8:55 a.m.

Brampton East, Lib.

Raj Grewal

Thank you.

Ms. Silverberg, thank you for your testimony. You work for Johnson & Johnson, which is a large pharmaceutical.

You mentioned that in Canada we value health and we want to ensure a healthy population. I think the job of any government is to ensure the success of its people.

Isn't it a bit of a conundrum for Johnson & Johnson that you're developing pharmaceuticals and your number one objective is for shareholder value? That comes in contradiction with developing drugs that would be helping people with rare diseases, because some of those drugs may not be profitable in the market but would do good in terms of the social licence associated with them.

Could you comment on Johnson & Johnson's role as an important stakeholder in developing a solution to this problem?

8:55 a.m.

Stakeholder Engagement Manager, Government Affairs and Market Access, Janssen Inc. Pharmaceutical Companies of Johnson & Johnson

Stacey Silverberg

Johnson & Johnson is actually bound by our credo. It really helps us with every single decision that we make. Our credo puts patients, as well as doctors and nurses and mothers and fathers, at the core of everything we do. That's our guidepost for everything that we look to do. Ultimately we are responsible to shareholders as well, but there's a huge opportunity to have a mix, and I do believe very strongly that we do that. We are a for-profit organization, undoubtedly, but we are patient care first and foremost.

I have been privy to some recent meetings where we know things will not be as commercially viable, in some of the terminology used today, but again, going back to the credo, it's the right thing for us to do. If we have new innovation to bring to patients, we need to continue to work in that vein and put patients at the centre of every decision we make as an organization.

9 a.m.

Brampton East, Lib.

Raj Grewal

Thank you.

Doug.

9 a.m.

Liberal

Doug Eyolfson Liberal Charleswood—St. James—Assiniboia—Headingley, MB

Thank you very much, and thank you all for your presentations.

Ms. Silverberg, something that Dr. MacKenzie said echoes what we found repeatedly during our pharmacare study, that Canada pays the third highest cost for medications in the world. You're concerned that changes to the PMPRB could decrease access to innovative drugs. How is it that the rest of the world is paying so much less for drugs, but putting our country more in line with the rest of the world would cause this decrease in innovation?

9 a.m.

Stakeholder Engagement Manager, Government Affairs and Market Access, Janssen Inc. Pharmaceutical Companies of Johnson & Johnson

Stacey Silverberg

It is a difficult issue to understand, and admittedly it took all of us in the community who are not health economists a while. I don't profess to be a health economist. My background is certainly coming up through the pharmaceutical organization.

What we understood going forward was that, when you look at the current basket of countries, Canada has a favourable position whereby we get early access because of the pricing and the comparative countries being used. With the new proposed changes, that does put us in a situation where a lot of lower-cost countries are put now in the comparative basket, compared to where we currently are with the seven countries being referenced. Because of that, what it actually means for patients, and all future patients in Canada, is that, with a less favourable pricing regime, this will potentially affect the opportunity to stay an early-launch drug country.

Secondly, it may not foster an environment where global companies will look to Canada for clinical trials. That is the reality of being 2% of a global picture, that Canada unfortunately may be at risk for not bringing that innovation, starting at its earliest point, here, which is the earliest point for people such as Ian and his daughter to have access to new treatments that may help them.

Thirdly—

9 a.m.

Liberal

Doug Eyolfson Liberal Charleswood—St. James—Assiniboia—Headingley, MB

Sorry to cut you off, but I have limited time.

For the common diseases such as diabetes, insulin is a century-old drug and the price hasn't gone down in literally decades. It's still quite inflated. The prices of life-saving medications such as EpiPens are actually skyrocketing, despite the fact that it's a fairly basic drug.

Are you saying, in order to foster the development of medications for rare diseases, we have to keep common drug prices artificially high?