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.
NDP
Don Davies NDP Vancouver Kingsway, BC
Dr. MacKenzie, you mentioned, and I'm sorry if I got this wrong, a 30,000 rare disease genomes project.
Clinician Scientist, Children's Hospital of Eastern Ontario (CHEO)
Yes.
Clinician Scientist, Children's Hospital of Eastern Ontario (CHEO)
How can they support it? Marc LePage is here as an observer, and perhaps he could elaborate later on.
Fundamentally—not to go into some inside baseball talk here—it's in the budget this go-round, I think, for part of the global Genome Canada ask. That's how it's going to be remunerated, and I just think it's overdue. As Michael said, there are five million U.K. genomes being done right now. We're asking for 30,000. We're international leaders in this realm, and I just think the return on investment will be phenomenal.
Sorry; that's a little subjective.
NDP
Don Davies NDP Vancouver Kingsway, BC
Thank you.
Ms. Silverberg, I'll give the last question to you. If I heard your testimony correctly, you suggested that there's a relationship between early launch and the impact of pricing. I think you suggested that Canadians may risk having early launch of drugs if we reduce prices. I wrote down your words, and you said that a “less favourable pricing” environment may result in reduced access.
If, as a drug company, you're putting patients first, why would that be?
Stakeholder Engagement Manager, Government Affairs and Market Access, Janssen Inc. Pharmaceutical Companies of Johnson & Johnson
The reality is that we do put patients first. We will do everything to try to make things available, and they will become available. The timing of such a decision may potentially be affected, though, because we are a global organization, and there will be countries that will have an opportunity, again, as patients, to have it earlier.
We are worried about our Canadians. They will eventually potentially get access, hopefully in most cases, but there will be a risk of not launching here, because ultimately decisions have to be made as to what's best for other patients in other jurisdictions. We never want to put patients at risk.
The reality is that those who are lower than the OECD median.... In fact, PMPRB creates their own reports, so the data is actually there. New countries, such as the Netherlands, are being entered into the comparator basket. New Zealand is not in the comparator basket, but only 16% of drugs are being launched there because of an unfavourable pricing structure. In Spain 21% of drugs are being launched.
Right now, with the current environment, 50% of drugs in Canada are actually being launched here because of the prices that are afforded within this—
Liberal
Sonia Sidhu Liberal Brampton South, ON
Thank you, Chair.
Thanks to all of you for being here.
My questions are for Dr. MacKenzie and Dr. Brudno.
You said that for neonatal testing, 50% of kids died before the age of five. How can we get to a solution for genetic testing in terms of neonatal testing?
Clinician Scientist, Children's Hospital of Eastern Ontario (CHEO)
Michael, I'll tackle it first.
Basically, I think clinically introduced DNA sequencing will identify these individuals before they manifest their symptoms. That's the short answer. We have newborn screening in our institution where we look at 150,000 babies a year. For Ontario we study 40 diseases. With new exome sequencing, in the brave new world we'll be able do for it hundreds and thousands of disorders, to identify those pre-symptomatically.
Professor and Scientific Director, Centre for Computational Medicine, Hospital for Sick Children, As an Individual
To follow on from that, I would slightly disagree with my colleague in that I think pre-symptomatic testing using genomics is still a little bit further away. However, the ability to have very rapid testing once the symptoms onset can help improve outcomes significantly.
In the U.S., a 48-hour genome is becoming a new norm in neonatal intensive care units. When you have a baby who's extremely sick, they will bring a genome. They will do a test. They will try to identify the right therapy within 48 hours. That's instead of trying option one, option two, or option three, during which time the baby is getting progressively worse and during which time there is a deterioration that may not be reparable once the right treatment is identified. With certain seizure disorders, a baby's brain will basically fry before the medicine has the ability to kick in. You need to find the right medicine and you need to find it quickly.
These very rapid testing regimes, which the precision medicine initiatives like the ones suggested are trying to bring to fruit, are the right approach.
Liberal
Sonia Sidhu Liberal Brampton South, ON
Thank you.
There's a lot of potential for artificial intelligence to help people living with rare diseases and disorders.
What are the major advantages of artificial intelligence? Can you explain that? Ian, can you explain your thoughts on that too?
Professor and Scientific Director, Centre for Computational Medicine, Hospital for Sick Children, As an Individual
Artificial intelligence is another way of saying “really advanced computing”.
There are advanced computational tools that can help individuals in all phases of our lives. With your Siri assistant or your Google Assistant, you can do things by talking that you previously would have had to spend a lot longer time typing or entering on the phone.
It's the same in the case of rare diseases. These artificial intelligence methods can help patients undergoing therapies who need help with day-to-day tasks, which they can get artificial intelligence to do for them.
At the same time, artificial intelligence also forms the core of how we analyze these genomes. Having large numbers of genomes allows us to learn from these, to identify what changes in the genomes correspond to what clinical outcomes, and to identify why with two individuals with exactly the same genetic mutation, one may be playing soccer and the other is on a ventilator and unable to walk.
Having more and more patients, more and more data, are critical for artificial intelligence solutions, to learn the difference between those two patients and to be able to predict, for new patients, the most likely trajectory and the best intervention.
Clinician Scientist, Children's Hospital of Eastern Ontario (CHEO)
We also use it for therapeutic configurations for rare diseases as well. We identify potential therapies without actually going to the benchtop and test tubes. We're just doing a sort of computational analysis.
Liberal
Sonia Sidhu Liberal Brampton South, ON
Thank you.
Ms. Silverberg, with regard to PH or pulmonary hypertension, is it considered a rare disease?
Stakeholder Engagement Manager, Government Affairs and Market Access, Janssen Inc. Pharmaceutical Companies of Johnson & Johnson
Yes, it actually is defined as a rare disease.
Jacqueline Dobson Government Affairs and Policy Manager, Government Affairs and Market Access, Janssen Inc. Pharmaceutical Companies of Johnson & Johnson
For the record, it's pulmonary arterial hypertension, which is otherwise known as PAH, for short.
Stakeholder Engagement Manager, Government Affairs and Market Access, Janssen Inc. Pharmaceutical Companies of Johnson & Johnson
This is the rarest form of pulmonary—
Liberal
Sonia Sidhu Liberal Brampton South, ON
Do you think that when people are enrolled with clinical trials that big trials are more effective?
Sometimes all the population cannot participate in clinical trials. How do you measure the efficacy for the trials?
October 25th, 2018 / 9:20 a.m.
Stakeholder Engagement Manager, Government Affairs and Market Access, Janssen Inc. Pharmaceutical Companies of Johnson & Johnson
That would not be my area of expertise, but we'd be happy to follow up for you.
Clinician Scientist, Children's Hospital of Eastern Ontario (CHEO)
Big trials aren't possible. You need to go to even one trial sometimes because it is so rare. You just need to be very fastidious about the biomarkers and that the questions you're asking are doable.
It's a new way of doing clinical trials, no question.
Government Affairs and Policy Manager, Government Affairs and Market Access, Janssen Inc. Pharmaceutical Companies of Johnson & Johnson
Clinical trials are not just one phase. They occur in three phases.
As I'm sure you can imagine, progressing to each phase becomes more and more difficult, even before it can get going through our pricing regulations, as well as through our commercialization bodies through the provinces to be listed.
Liberal
Sonia Sidhu Liberal Brampton South, ON
Thank you.
The committee heard from Dr. Craig Campbell, from his department of pediatrics and neurology at the Children's Hospital at the London Health Sciences Centre, that registries are essential to capturing data on the health benefits and risks of new drugs on rare diseases.
Can you describe what additional steps Health Canada could take to support the surveillance of the safety and efficacy of a drug that could be more effective?
Dr. MacKenzie.
Clinician Scientist, Children's Hospital of Eastern Ontario (CHEO)
Craig Campbell, an esteemed colleague, has a Canadian neuromuscular disease registry that is run out of Calgary. It is a very impressive disease registry, where you can track safety and do clinical trials. It's a perfect model for how things should be done in a perfect world.
I think as we go ahead with the companies and new drugs being introduced, an important part is setting up these networks and capturing the data as we go forward, ensuring efficacy and ensuring safety. I know the that the CNDR is in conversation with Biogen on spinal muscular atrophy, for example.
I think that's an absolutely important aspect that we need to do, ultimately not to sort of perseverate with the Genome Canada project, but the introduction of these rare disease genomes is going to be a good platform from which to move on to registries as well.
