Health committee  Mr. Chair, members of the committee, thank you very much for having me speak to you about how we've been directly affected by life with the rare disease cystinosis. I would also like to personally thank MP Ben Lobb for caring about our ongoing issue since the first day that our

Health committee  Speaking in general about the States, which is the closest to us and we're the most involved with them, I know that Horizon pharmaceuticals owns the patent for the new drug Procysbi. It has a seven year...and now they've added another three-year extension. Basically, for the next

Health committee  That's correct.

Health committee  We are just under $15,000 per year.

Health committee  Under the inherited metabolics disease program Olivia's Cystagon is covered. With the new approval, Procysbi is as well. Our access to Cystagon is through SAP right now. Currently, we have to apply every three months for Olivia to stay on this treatment as it's flawless for her.

Health committee  Yes. It's roughly calculated from the price that we had to search tirelessly for. That is what Olivia's dosage would come to.

Health committee  She is probably at 50 pills a day to maintain her health. I want to add, if I may backtrack—sorry—to what the issue is here as we talk about pharmaceutical companies coming here, and as Mr. McKinnon said. You asked about the cost. The pharmaceutical companies are two steps ahead

Health committee  I'm grateful that we're here today. I know that having patients come here and speak is relatively new to this kind of situation. I guess I really never knew what advocacy meant until I started in the role of doing it. It takes time away from my children, but I'm grateful to be he

Health committee  For our daughter, we're just under $15,000 a year.

Health committee  Her dose would switch to potentially $300,000, because it's a cost per pill.

Health committee  That's correct.

Health committee  This is where it comes down to the bureaucracy of business and Health Canada. It was basically because the new drug was approved. SAP is there for special access when there are no drugs currently available in the country to take. Procysbi was approved in July 2017, and we went t

Health committee  Okay, yes.

Health committee  Prior to all the changes, I didn't even know they had to apply every six months; honestly, I thought it was once a year. I would call the pharmacy when we were headed down to the clinic and I would just say that Olivia needs a refill on her prescription and there were no question

Health committee  In Canada, there are between 75 and 100 patients.