Muhammad Akhter spent 12 hours trying to figure out how to tell his wife some terrible news.
The couple’s three children have been denied treatment coverage from the province for their rare genetic disease, but Health Minister Dustin Duncan said he will seek a second opinion.
Muhammad Abdullah, 12, Khadija Amir, 10 and Sara Amir, 8 have an enzyme-related disease called Morquio A Syndrome (also called MPS IV type A). Their bodies lack an enzyme in their blood that breaks down cellular waste in the body. Without treatment it can lead to multiple diseases and premature death. The children have already experienced growth problems and stiff joints. Two of the them already use wheelchairs.
There is no cure, but treatment with a synthetic enzyme called Vimizim could slow down the disease and potentially extend the life of the children. The treatment would cost hundreds of thousands of dollars a year for each child.
“I was not expecting to be denied from the (common drug review’s) side because this is the last resort. This is the last hope,” Akhter said. “It’s a feeling, it’s a pain from the inside. It’s pretty hard.”
The Ministry of Health sought consultation from experts outside the province and provided information from the family and their physicians. Health Minister Dustin Duncan said they were told the drug wouldn’t be as effective on the children because they were older than five years.
“The longer it takes for this drug to be administered, the disease progresses to a point where it just becomes a question of whether or not there is efficacy to administering this drug,” Duncan said. “You would like to be able to say yes to everybody, but you want to ensure that for the sustainability of the system that you’re using the health care dollars as wisely as possible.”
Morquio A Syndrome only affects an estimated 100 people in Canada right now, according to the group Morquio in Canada.
This is the last hope.
The Isaac Foundation said another Saskatchewan child has already received funding for the treatment, and Duncan said he only knew of one child being approved for therapy in recent memory.
The family, with the support of the foundation, met with the minister Monday to plead him to reconsider.
Duncan said because another child was approved for the drug before and because he feels there is debate in the medical community about the effectiveness of the drug at certain ages, he would like to review the siblings’ case.
“I’m going to ask the ministry to consult further with some other out-of-province experts to give me a second opinion on this,” Duncan said. “There’s not a lot of options for these patients so we want to make sure we give a full look before we close the door.”
Akhter said the family is now waiting anxiously, adding they aren’t sure what they will do if the second answer is still no.
“It’s like night and night; no shining,” he said.