Health committee  Yes, I do believe we need to move forward. They might not be perfect, but they're progress and that's what we need. Mr. Davies, I'd like to answer your question, but I just want to say one thing. Ms. Rempel Garner, I appreciate your calling my testimony today “compelling”. As

Health committee  With Olivia it's genetic. She also lives with chronic kidney disease, and.... Go ahead, Mr. Chair.

Health committee  Okay. Olivia lives with chronic kidney disease. Chronic kidney disease is a lifelong circumstance until it leads to a kidney transplant. So on top of cystinosis, which is what the high-cost drugs treat, she has a lot of treatments for renal issues that are not covered. Even thi

Health committee  This is something we always think about as a family. One, we're very fortunate that my husband does have a great benefits package. When Cystadrops came onto the market, the insurance company lay in the weeds to see if the government was going to cover it before the company made t

Health committee  Yes.

Health committee  Yes, and I'm going to speak only to our rare disease because it is the only disease I represent. We are very fortunate that a cure for cystinosis is on the horizon. In 2019, a clinical trial opened that uses gene and stem cell therapy, which, again, will hopefully result in a cu

Health committee  Yes. The active ingredient, cysteamine, is in all four of the products that I previously talked about. With cysteamine, the hardest part of almost any treatment, if you ask any patient, is the side effects. The side effects of cysteamine are from all drugs. When they took Cystag

Health committee  The drug is based on weight, I just have to point out. There is the two-year-old who takes Procysbi for $56,000 a year versus Olivia who takes Cystagon at $18,000 a year. If Olivia were on Procysbi, she would have to take a higher dose, which would result in more money. The comp

Health committee  Mr. Chair and members of the committee, thank you for the opportunity to appear before you to share my personal views about the Patented Medicine Prices Review Board and my personal experiences as a rare disease advocate. My name is Erin Little. I am the president and co-founder

Health committee  For us, for cystinosis, the research is being done in the States. We have doctors up here doing some research, new research, but everything has been funded directly by patients, because our rare disease population gets no public funding. I can't think of it off the top of my hea

Health committee  That's interesting. We don't take pharma money to run any part of our organization. We host a golf tournament to fund research, and for our tournament in 2017, Horizon pharmaceuticals was a sponsor of ours. This was prior to the issue and what I have learned over the past year.

Health committee  On the Liv-A-Little website?

Health committee  I'm not even aware that it must read that way. Thank you.

Health committee  Yes. Horizon Pharma purchased what was Raptor Pharmaceutical. It was Raptor Pharmaceutical within the U.S. with the Cystinosis Research Foundation, which I'm a part of. The family organization directly funded Raptor to bring Procysbi to the market, and Raptor then sold that piec

Health committee  It's interesting because what we're finding, actually, when we do start to research rare diseases is that it is unfolding and is involved in helping other diseases. There's a drug library that is progressing. Let's say that they take Cystagon and they see if it can help other d