Health committee  It's still available. It was never not available.

Health committee  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 consid

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

Health committee  The practitioner has the option to submit to the special access program for Cystagon. It is still approved today. It was never not approved. But a practitioner has to present a request that explains the medical rationale for why the available therapy is not right for that patient

Health committee  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

Health committee  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—

Health committee  —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 th

Health committee  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 t

Health committee  A regulatory framework exists. We can't compel sponsors to come in and apply for market authorization. We can certainly encourage them. We can explain the process, facilitate, and provide incentives. However, ultimately it's a company's decision whether they market a product in C

Health committee  I have one thing to add. Our regulations do not give authority to compel a sponsor to come to Canada. We cannot compel a company to market a product in Canada, so we work to encourage them to come. We work to provide incentives at the level of clinical trials. We work to ensure

Health committee  The short answer is yes. The special access program has been in operation for a long time and, as I mentioned earlier, it requires a dialogue with physicians, so the physician has to determine that specific therapy is the best choice for their patient. They submit a request; it

Health committee  Certainly there's a trend in development of drugs to personalize medicine. Genomics and other markers can be used to hopefully identify subpopulations of patients with a disease where a therapy might be most beneficial, have the least number of side effects and cause the least nu

Health committee  I support what Cathy said. With orphan diseases—rare diseases—one of the challenges in studying drugs is the size of the population you're studying. Globally there may be fewer than 1,000 patients suffering from it. Normally, in clinical trials that approve drugs for market auth

Health committee  The special access program is meant to be accessed by practitioners who have authority in provincial jurisdictions to prescribe unapproved medication. It's a process that begins with a physician making a decision that the unapproved therapy is the best choice for the patient in f

Health committee  I have previously.