Violet Revet is a very sick little girl and her family is worried the province isn’t moving quickly enough to help her.
The three year old from Langenburg is one of only nine people in Canada diagnosed with
Mucopolysaccharidosis VI (MPS-VI). The ultra-rare disease has no cure and the longer Revet goes without treatment the shorter her life could be.
“Violet right now without treatment is definitely going to suffer and that’s really devastating for the family,” explained Andrew McFayden in an interview Wednesday from his home in Ontario.
McFayden knows all too well the impact of MPS-VI. His son Isaac was diagnosed with it in 2005. McFayden said the disease ravages the body if left untreated.
“It’s a very devastating condition. Essentially kids that are diagnosed with MPS lack an enzyme in their blood that breaks down all the complex sugars. Because those complex sugars that occur naturally in the body don’t break down they need to find a place to go and so they end up being stored in the bones, tissues, organs, and muscles and it leads to many progressive symptoms: heart and airway disease, progressive stiffening of the joints, corneal clouding, spinal cord compression. Essentially every muscle, tissue, and organ in the body is affected by this disease with the exception of the brain.”
McFayden was told Isaac would only have seven or eight years to live. That was before doctors realized a treatment was making the rounds in the UK and the US, a synthetic enzyme known as naglazyme that acts as a replacement in the body.
Isaac could get access to it under a special access program but the drug wasn’t covered in Ontario. It took McFayden eight months of lobbying before the provincial government there agreed to pay for it and he suspected the delay in Violet’s case is the same as the delay there.
“It’s (up to) a million dollars a year, so I understand that governments have to look closely at this type of thing. Usually what they say they have to look at is the safety of it but it’s already available in the United States, most of the UK…many, many other countries have this. The safety has been proven. Essentially it comes down to dollars and cents and (I believe) that’s why these provinces are delaying.”
Thanks to weekly treatments many of isaac’s symptoms were reversed. It’s given the boy a new lease on life as his family hopes that a cure could one day be found.
Asking for help
After her diagnosis Violet’s family made a request to Saskatchewan’s health ministry for funding so she can get the same treatments. After going more than six months without an answer they went to McFayden for help and he says time is very much of the essence.
“Early detection and early treatment of this conditions is paramount to the future success of children with MPS-VI,” he asserted.
In an effort to spur the government to action McFadyen and supporters of The Isaac Foundation, an advocacy and fund raising group he started to help others with the disease, have been lobbying Premier Brad Wall via the social networking web site Twitter.
On Wednesday the Premier responded, saying they had his attention and revealing that he had directed the Health Ministry to contact the Revet family director. But McFadyen says lip service doesn’t do a sick little girl any favours.
“In Canada we’re supposed to have equal and fair access to health care and right now Violet is not receiving that.”
Tracey Smith is the director of pharmaceutical services for the ministry. She said the delay it is not about the cost of the drug. She denied that any unnecessary delays have happened, insisting the government didn’t get the family’s funding request until July 9th.
“We received the request that day and we have been reviewing the request. We’re working as quickly as possible in order to make a decision and communicate that back to the family.”
She said every funding application goes through a similar review process but she stressed the ministry is looking at the fact that the treatment is already paid for in Ontario, Quebec, and B-C, among other things.
Smith said she’s hopeful a decision will be made in a few weeks but she won’t say what the likelihood of approval is.
McFayden is sorry to hear it; he said work needs to begin as soon as possible because pharmacists and hospital staff that will be working with Revet will need weeks of training before treatments can begin.
Edited by CJME’s Lisa Schick.