BY SEAN TREMBATH, THE STARPHOENIX
Photograph by: Gord Waldner , The StarPhoenix
Saskatchewan’s NDP Opposition is criticizing the provincial government for rejecting a Saskatoon family’s request to fund drug treatment for three children suffering from a rare developmental disease.
“We think the government needs to immediately reverse this decision and get these children the drugs they need,” said NDP health critic Danielle Chartier, MLA for Saskatoon Riversdale.
Muhammad Akhter has three children — Muhammad, 12, Khadija, 10, and Sara, eight — who suffer from Morquio syndrome or mucopolysaccaridosis IVA (MPS). The disease affects many aspects of development. The children have bowed legs, fragile bones, respiratory and heart problems and diminished sight and hearing.
“The symptoms are affecting almost every part of the body,” Akhter said.
Health Canada approved the drug Vimizim, or elosulfase alfa, as a treatment for MPS in 2014. It was also approved by the U.S Food and Drug Administration that year. Vimizim can cost up to $460,000 a year per patient.
Health Canada’s trials found the drug to be safe and effective for people aged five to 65. In particular, a test that measured how far patients could walk over six minutes saw a statistically significant improvement in those who had been given the drug.
“In conclusion, the efficacy of Vimizim (2 mg/kg/week) has been adequately demonstrated,” Health Canada’s report said.
Speaking to media on Monday about the decision to refuse coverage for the drug, Health Minister Dustin Duncan referred to a different study — a common drug review done by the Canadian Agency for Drugs and Technologies in Health (CADTH), a non-profit established by the federal and provincial governments in 1989.
The CADTH’s study looked at Health Canada’s testing and reached a different conclusion. It acknowledged the improvement in walking distance, but said “the clinical relevance of this finding is uncertain.”
When looking at the trials as a whole, the CADTH said there was not enough evidence to say the drug is effective.
“Treatment with elosulfase alfa has not been shown to improve other clinical end points, including reducing pain, fatigue, disease progression, or the need for surgical intervention,” the CADTH report said.
Duncan said the children were also denied approval because an out-of-province specialist questions the drug’s effectiveness in children older that five.
On Thursday, Chartier noted that Saskatchewan is already paying for another child — a girl under five — to receive the treatment.
The Isaac Foundation, a national advocacy group for people with enzyme disorders, released a statement in support of Vimizim treatment on Thursday. It said 29 people in Canada are receiving the treatment, 27 of whom are older than five.
“Every one of these patients is seeing improvements,” the statement said.
Chartier also provided a fact sheet from BioMarin Pharmaceutical Inc., the company that sells Vimizim, that states the drug is safe and effective for people five years and older.
Duncan said Monday that the government would review the denial and seek another professional opinion on the drug. Chartier said the government should reverse the denial immediately.
“These children every day experience issues that get worse. The time to make the decision to change is right now,” she said.