EDMONTON – The Alberta government has approved funding to treat a three-year-old St. Albert girl suffering from a rare enzyme deficiency.
According to The Isaac Foundation – a group that supports families of those suffering from Maroteaux–Lamy Syndrome (MPS) – funding for Aleena Sadownyk was approved on Monday.
In an email to Global News on Monday, Andrew McFayden of the Isaac Foundation simply writes: “Treatment for Aleena has been approved!”
Alberta Health Minister Fred Horne confirmed to Global News on Monday that the province will fund the treatment for young Aleena Sadownyk.
“I’m pleased that we have been able to extend coverage for this drug,” he said. “This is a drug that is not licensed for sale in Canada, and we have a program in Alberta that allows for us to consider clinical evidence, and other factors as well, and in some cases we are able to provide coverage. So, a positive outcome in this case. From here, medical experts will work directly with the family.
Aleena was recently diagnosed with MPS, which means she lacks the enzyme needed to break down waste in her body, so it builds up on organs, bones, and muscles.
“She’s a little girl,” says her father Dane Sadownyk. “She didn’t ask for this, she didn’t deserve it, she should be given every opportunity like any other child to have the best shot, the best chance at life.”
The disease will cut her life short if she doesn’t get treatment.
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“Her heart valve is affected, her liver and spleen are enlarged, and of course the deposits on the bones,” says her mother Laura Sadownyk. “It will result eventually in death.”
However, the drug Aleena needs – Naglazyme – isn’t approved in Canada, so her family has been appealing to the province to provide funding for the costly treatment.
On Monday, Global News was able to reach the Sadownyk family by phone after they had been told by the Isaac Foundation that funding for the treatment had been approved.
“We’re so relieved,” says Laura.
“We probably found out, I don’t know, maybe ten minutes ago,” she explained around 9:30 a.m. Monday. “We’re speechless, and Dane’s in tears, and I’m still in shock here. I can’t believe it. I’m so happy, and so grateful for everyone who’s helped us out.”
“It means she’ll be able to do what she wants to do in life, and have every opportunity like anyone else,” she explains. “It means the symptoms will stop taking over her little body… it’s not a cure but it will definitely stop any more debilitation from happening.”
“I’m hoping that any parents in the future don’t have to go through this,” she adds.
Naglazyme treatment costs about $300,000 for a year for someone Aleena’s age.
In a news release sent Monday, the Isaac Foundations says “Alberta Health has made a decision to fund a life-sustaining treatment required by a St. Albert, Alberta toddler.” It explains that, “prior to Alberta’s approval, treatment was already being funded in BC, Saskatchewan, Ontario, and Quebec.”
A spokesperson with the Alberta government says the province spends “$13 million per year on rare disease program drugs and $3.5 million has been allocated for Short Term Exceptional Drug Therapy program this year. This doesn’t include treatments for rare cancers.”
Health Canada has sent Global News the following statement on the matter:
In Canada, the federal government through Health Canada is responsible for evaluating drugs for their safety, efficacy and quality. A drug may not be legally sold unless approval is received from Health Canada.
Pharmaceuticals are a shared responsibility between federal, provincial, and territorial governments. The provinces and territories are responsible for purchasing, managing, and delivering their supply of drugs for their clients and jurisdictions. The Government of Canada purchases drugs for populations it is responsible for, such as aboriginal Canadians and members of the Canadian Forces. Most publically funded drug plans in Canada are paid for by the provinces that make their own determinations regarding which drugs will be funded.
On October 4, 2012, the Federal Government announced that it is developing a new Orphan Drug Framework that will facilitate the generation of knowledge about an orphan drug and its use in treating a rare disease. This is intended to reduce uncertainties about the benefits and harms associated with a drug and better inform the decision making at all levels including the decisions made to fund a drug or not.
It is important to note, that we recognize the unique circumstances of rare disease and that is why we have the Special Access Program. The Special Access Program exists to give patients with serious or life-threatening illnesses access to medicine not available in Canada. Health Canada works quickly to review requests under its Special Access Program (SAP) to ensure the proposed drug is safe. When Health Canada receives such a request, the vast majority are responded to within 48-hours.
The drug Naglazyme, has been authorized for nine patients through SAP.
The PC MLA for St. Albert, Stephen Khan, has been urging Alberta Health to move quickly in making a “positive decision” for Aleena and her family.
In a message posted on his Facebook page Tuesday, August 6 Khan writes:
“As many of you have recently learned, a St. Albert family is seeking assistance from Alberta Health Services (AHS) to cover medication treatment for their three year old daughter’s rare medical condition. Aleena Sadownyk is an adorable three year old girl, who suffers from a potentially fatal and very rare enzyme deficiency, known as MPS VI.
“MPS VI has proven to be treatable with a drug called Naglazyme. This drug, unfortunately, has not been approved for use in Canada. The Sadownyk family is currently seeking funding for the treatment from AHS, through the Province of Alberta’s Short Term Exceptional Drug Therapy Program.
“As the MLA for St. Albert, I fully endorse the Sadownyk’s request for funding support from AHS. My office and I have been advocating and working with the Minister of Health’s office on this issue since early July, when we first learned of the Sadownyk family’s situation.
“While I respect there is a process to follow for the Short Term Exceptional Drug Therapy Program review, I urge AHS to move quickly in rendering a positive decision for the Sadownyk family. Every day is an eternity for this young family, all the while knowing that a treatment for this debilitating disease is at hand. Currently four other provinces — Quebec, Ontario, Saskatchewan and British Columbia — have all made exceptions for families in need of this specific treatment.
“We remain hopeful that we will have a successful resolution for Aleena and her family. In the meantime, our thoughts and prayers go out to Aleena, her family, friends and supporters.”
On Friday, August 2, Health Minister Fred Horne sent a statement to Global News:
“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.”
Wildrose Health Critic Heather Forsyth has written a letter to the minister asking him to fund Aleena’s treatment.
“Every day this child waits her condition deteriorates. We need to help her,” says Forsyth.
New Democrat health critic David Eggen also sent a letter to Minister Fred Horne requesting that he immediately approve funding for Sadownyk. He’s also calling on the PC government to list the necessary treatment for future patients.
“This is another sad and stark example of the PCs’ approach to health care—they prefer to diminish and de-list services instead of strengthening and expanding them,” said Eggen. “As a result, this young girl and her family have been forced into a crisis constructed from PC neglect.”
On Monday, Horne said he would like there to be a greater discussion about how treatment coverage for rare diseases could be handled by governments in Canada.
“There is a need for a discussion on a pan-Canadian basis about how we make these drugs available and about how we do so in a way that’s affordable and based on good evidence. I’ve been calling for some time for a discussion for a national catastrophic drug program for Canadians. It could include elements that would deal with rare and orphan diseases like we’ve heard about in this case.”
“The number of Canadians that are affected by these diseases is very, very small, but the cost for some of the drugs is very large.”
With files from Vassy Kapelos, Global News
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