Health Committee on Oct. 30th, 2018

[Catherine Parker]

I would just add that we are working very aggressively with the list of drugs that are on special access, especially for the rare diseases, to bring those into some kind of authorized state.

In working with the companies, it's a matter of would it take to get them to file for approval of a product in Canada. If they have filed to another jurisdiction and they have the medication approved there, we will use the assessment reports of those regulators, the decision that regulator made, to help us get those products off SAP and into a marketed state, so that the patients, families and physicians don't have to go through the special access program.

[Ron McKinnon, Liberal, Coquitlam—Port Coquitlam, BC]

Thank you.

I'll go now to Ms. Sidhu.

[Sonia Sidhu, Liberal, Brampton South, ON]

Thank you, Chair.

Thank you, all, for being here.

In rare diseases sometimes there's a lack of scientific evidence. What type of evidence does the department consider in its regulatory review?

We heard before from a panel that said Canada needs a drug review policy. Can you comment on that?

[Catherine Parker]

I can certainly comment on the fact that we treat every drug for a rare disease uniquely. We meet with the manufacturer of that drug and we go over what kind of data they have available, what kind of data they are capable of getting, what kind of data may have been generated elsewhere in the world. We negotiate on a case-by-case basis what will be the requirements.

Every drug is different, and even in the rare disease area, you could be dealing with a drug for two or three patients versus one drug for 100 or 200 patients. It may be a very rare disease in Canada, but not as rare in other parts of the world, so there may be data from other types of trials.

I can't say this more earnestly: We treat every, every drug case by case. We agree on and design an approach to that drug with the manufacturer that suits the needs of that patient community and the data they are capable of obtaining. We use conditional approvals. We use priority review. We use reports from other regulators. We use literature information. We take all the information....

We like to refer to it as the totality of the data, but it is unique to each product.

I don't know if Dr. Stewart wants to add to that.

[The Chair, Liberal, Bill Casey]

Actually, the time is up for that question.

Ms. Gladu.

[Marilyn Gladu, Conservative, Sarnia—Lambton, ON]

Thank you, Chair.

Thank you, witnesses. It's good to see you again.

It's clear from the testimony we've been hearing that when it comes to rare diseases, people are in the situation where they need the medication and they will die without it. It also seems true that the better experiences they've had are when they're involved in clinical trials and they're able to get access to the drugs through that.

I'm interested to hear what you think about the right-to-try legislation that has been introduced in the U.S. Basically, when you have people in this situation, it's a very, let's say, efficient fast lane of drug approval for giving clinical trial approvals and letting people try things that may save their lives.

What do you think about that?

[Dr. John Patrick Stewart]

Access to clinical trials is often when we're doing investigational testing because there are certain requirements on the sponsor to have a well-designed protocol, that the risks are mitigated to the degree possible, that patients are informed and that you have REB approval. In the development of drugs, we encourage access to be through a well-designed clinical trial.

Having said that, there are challenges when patients with rare diseases may be distributed randomly or very widely in small numbers across the country. We work with sponsors to encourage access to those individual patients or specific patients. Failing that, there are other options, like open label trials or compassionate access programs, where individual patients, under the design of a protocol, can get access to a drug that may not actually be in the larger trial that's ongoing.

When trials finish, then there's also a concern about ongoing access. Again, there are opportunities, if the sponsor is prepared to continue to provide access, so that patients that are responding to the product can continue to get access through an open label extension or compassionate access. We encourage that until such time as it's market authorized.

Under the right to try, right to try can mean different things. It can also mean that a patient who wants access to investigational therapy where there may be varied or no evidence around its efficacy wants access to that drug. When we did the SAP renewal, the thinking around the special access program, we did a consultation back in December of last year and January this year. It was one of the questions we asked. There were health professionals, health care system workers, patient support groups and associations. By and large, there was very little support for the right to try.

Some of the reasons we heard were typically about, when you're talking about right to try you're talking about a serious or life-threatening condition, and almost unanimously, you require a health practitioner to be involved in that care. The special access program allows that to happen. A health practitioner can evaluate an individual patient, look at the products available, look at what evidence there may be, credible or not, to support the use and come forward with an application. It's also something in jurisdictions elsewhere that there's not a lot of support for. In fact, most manufacturers have commented that they don't want their investigational products necessarily being accessed in that way. They would rather that it be in its early development or a properly designed trial, where you can control for variables, and the evidence is usable to move forward with the support of market authorization.

Cathy, do you have any other—

[Marilyn Gladu, Conservative, Sarnia—Lambton, ON]

That's very good.

We heard testimony that, when the PMPRB takes a really long time to come to price certainty, there's a fear that will discourage people from bringing their drugs to our country. We've probably talked about that before.

Have there been any reconsiderations to the modernization that's happening with PMPRB?

[Karen Reynolds]

As I think I mentioned last time, the department published proposed regulations to modernize the PMPRB in the Canada Gazette, part I almost a year ago, in December 2017. The proposal hasn't been finalized. We continue to consider the results of that consultation and engage with stakeholders on key issues related to the proposal.

[Marilyn Gladu, Conservative, Sarnia—Lambton, ON]

As well, we heard that, once people come to the end of their clinical trial, there is a gap of time before they receive approval. There was a recommendation from one of the witnesses that if the trial was completed with no negative consequence, it should have instantaneous approval so they can continue to take the drug.

Are you aware of this situation and can it be fixed?

[Catherine Parker]

I think this is what Dr. Stewart was talking about.

Yes, absolutely, there is the opportunity for patients to continue on treatment after the clinical trial. That's through an extension protocol or an open label type of situation.

However, it is all dependent on the manufacturer being willing to continue to provide. That's the reality. We do require that they file a protocol as to how the patients would be treated, but we do a very efficient and timely review of that. Much of this timing and this gap is influenced by the manufacturer and what they are willing to do.

[Marilyn Gladu, Conservative, Sarnia—Lambton, ON]

Thank you.

[The Chair, Liberal, Bill Casey]

Mr. Davies.

[Don Davies, NDP, Vancouver Kingsway, BC]

I don't know if you heard any of the testimony of the patients who were just here, but one of the mothers of a daughter who has a chronic rare disease testified that Health Canada makes her reapply every three months for the drug that her daughter needs to save her life. Why would that be?

[Dr. John Patrick Stewart]

I would assume you're talking about the special access program and applying for it.

As for the three months, I can't speak to specific requests as that's confidential, but typically a special access program request, if it is for a chronic illness or a longer-term use, would be approved for six months, and then it would need to be renewed again. The thinking, as I mentioned earlier, is that these are unapproved therapies and the regulations require the requesting practitioner to report on the use of the product. So, it's there.

In our special access renewal, we are looking at this from a client service perspective on an ongoing basis or a situation where it is a well-established therapy and the reason it's not on the market is that the manufacturer hasn't come to the market in Canada. The product may be approved in other jurisdictions. We will extend that to a longer period of nine months....

[Don Davies, NDP, Vancouver Kingsway, BC]

My understanding from the testimony we received was that it used to be every six months, but Health Canada has now made them apply every three months.

[Dr. John Patrick Stewart]

I think that might have been in the context that—I'm thinking it probably happened—as I mentioned earlier, when a product.... I'll speak specifically about a situation with Cystagon and Procysbi, which were two products—

[Don Davies, NDP, Vancouver Kingsway, BC]

Those were the products.

[Dr. John Patrick Stewart]

—for use with cystinosis through the special access program.

One of the products, an extended use product, Procysbi, applied for market authorization and received market authorization in June 2017. At that point we were still getting requests for both of the products through the special access program, knowing that it typically takes a manufacturer about three months to get their labelling in order and get it on the market. Knowing that in three months there would be an approved marketed therapy for this condition, it made sense that both requests for Cystagon and Procysbi were reduced to three months.

Our assumption was that most, if not all, patients would transition to the approved therapy. In fact, in the spring when this product was being reviewed and being announced as coming on the market, there was a lot of support from treating physicians who were involved in this disease group as well as some of the patients who were out advocating that it was great that Procysbi was coming to market. Our anticipation was that, in three months, there wouldn't be a need for as many requests, if any, through the special access program; hence, we reduced it to the time period we thought would be required for a product to be accessed.

[Don Davies, NDP, Vancouver Kingsway, BC]

Try to help me understand this, too.

The testimony we received was that their daughter was responding very well to Cystagon. I understand it was under the special access program. The cost was $15,000 per year. Their family was compelled to transition to Procysbi, which costs $300,000 per year, and the only difference—it's the exact same molecule—is the coating that affects the time release of the medication.

To lay people sitting here, that sounds absurd and ridiculous. Why would we have a policy that drastically increases the cost of the medication for no real difference in therapeutic value and, in fact, maybe, from this family's experience, a diminution in therapeutic value? Help me understand that.

[Dr. John Patrick Stewart]

Sure. I think it's important to point out that the situation of Procysbi and Cystagon was rare. It's very unusual that you would have two products coming through the special access program for one rare disease or that you have two products with a very different price.

The consideration under the regulations for the special access program is that we verify whether it's a serious and life-threatening condition. Have the products that are available on the market been considered, tried and failed or considered and not available or not suitable? Is there evidence on its use, safety and efficacy?

When Procysbi came on the market—it's the same molecule; one was extended release and one was immediate release—there was no clear medical reason at the time to say that Procysbi wouldn't be a suitable alternative. The special access program does not consider cost in its review. If you're looking for the reason why a product may be unavailable, cost is not considered. In fact, we're often not aware of what the cost is.

When Procysbi came on the market, the program had no idea at all what the price was going to be. It's not a conversation we're involved with. It's not part of the statutory purpose in the Food and Drugs Act that the special access program consider cost. In fact, their concern would be, if we went in that direction, that it would have an impact on market authorizations in general. It would introduce an unpredictability in the country in the sense that, if an innovator company wants to market a product and goes through the costs of doing research and development, the cost of marketing and the cost post-market, and there is a possibility that the special access program will provide access to a cheaper product that has not gone through extensive safety, quality and efficacy, you might destabilize whether innovator companies will come to Canada, because they have no guarantee of a secure market. It's not something that we—

[Don Davies, NDP, Vancouver Kingsway, BC]

But coming from the reverse end, if we're setting policy as a government and we're saying to patients that we're going to force them to take a drug that doesn't work as well, that costs way more than the drug they want to take and it's the same molecule—and it's paid for by the taxpayers—can you not see that Canadians would have some real concerns about how this program is being managed if that's the result?

[Dr. John Patrick Stewart]

You bring forward some very important considerations, some of which are beyond the mandate of the special access program.

[Don Davies, NDP, Vancouver Kingsway, BC]

I'd like to move to pricing.

Dr. Joel Lexchin testified to the committee. He said:

...the drug companies will not open up their books to reveal their R and D costs for new medications. There's a figure of $2.6 billion that's bandied around about being the cost of getting a new drug to market. That kind of figure is based on confidential data that won't be released. If drug companies want to prove that they need to charge these significant amounts of money that they do for new drugs, then they should prove to Canadians, to insurers, that those prices are actually justified, but so far they haven't.

To what degree are a drug company's production costs revealed to the government and considered in PMPRB reviews on excessive pricing? Do they open up their books and reveal their true cost of production to you?

[Karen Reynolds]

Your question is in relation to the mandate and the work of the Patented Medicine Prices Review Board. Unfortunately, I'm not in a position to comment on what information is made available to the board when making their price determinations.

As you may know, Mr. Davies, they sit at arm's length to the department. They're quasi-judicial. We don't have a view into the information that they receive. Officials from the board would be better placed to respond to your question.