3 August, 2013 mcfadyena

St Albert toddler has rare disease

Edmonton, AB, Canada / 630 CHED

August 02, 2013 02:51 pm
Some last minute negotiations began Friday between Alberta Health and an organization known as the Isaac Foundation, to get dire help for the family of a St Albert toddler.
Aleena Sadownyk has an extremely rare condition that has left the three year old with an enzyme deficiency, that’s breaking down her little body.

They are seeking treatment with a drug called Naglazyme.

“Alberta Health gave me a call to reiterate the fact that they’re working on this file and to tell us that they’re hopeful to have an expedited review and will render a decision soon,” said Andrew McFayden of the Isaac Foundation who has gone to bat for the family.
The Sadownyk family has been asking about the province’s Short Term Exceptional Drug Therapy program after originally turning them down through the Alberta Rare Diseases Funding Program.

“What that means I don’t know. I asked about timelines and bench marks and that sort of thing so that the family could make sure they were close to hearing the fate of their daughter buy I haven’t been able to get much of an update as far as time line.”
McFayden says the treatment has worked on seven other instances in Canada, including his own son.

Late Friday afternoon, Health Minister Fred Horne’s office issued a statement.
“My heart goes out to the Sadownyk family. I know they want to do everything they can to help their daughter. The family has contacted my office recently and were immediately put in touch with appropriate officials who could assist in supporting the family in making a funding request through appropriate programs.

As things stand, Naglazyme has not yet received general market approval through Health Canada. Because it doesn’t have federal approval, the options for funding this drug are different than for some other drugs. Albertans who need access to high-cost drug therapies not already covered by provincial programs may explore funding options through the Short-term Exceptional Drug Therapy Program. That program relies on medical experts to assess whether a specific drug will be safe and effective for a specific patient. It’s done on a case-by-case basis, so it can take a patient’s needs and particular health condition into account.

“As a politician, I don’t get directly involved in assessing which drugs would be funded through that process — and appropriately leave that assessment to medical experts. Department officials will continue to support the application process, a clinical review is underway now and I understand we expect a response soon.”

McFayden says time is of the essence.

“It’s tough that we’re going into a long weekend and to be honest with you, if I thought we could wait, we would have put this out on Tuesday, but I think the sooner a decision gets rendered the better, because even after a decision is rendered if it is a positive decision, and there’s no guarantee whatsoever the province is even going to say yes, then we’d have to ensure that the hospital staff will be trained.”

Officials from the drug supplier would come in from California to train the staff at the Stollery.

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