Health Minister Should Fund Life-Saving Drug for Alberta Teen with Very Rare Disease: Wildrose

HIGH RIVER, AB: A drug treatment plan recommended by an Alberta teenager’s doctor as a way to improve her quality of life, and potentially extend her life, should be considered for public funding under Alberta Health, the Wildrose Official Opposition said.

A 17-year old teenager from High River was diagnosed with C3 glomerulopathy at 10 years of age. The young lady’s condition has gradually worsened over the past six years. C3 glomerulopathy is very rare, with an occurrence rate of less than two persons per million. C3 glomerulopathy causes chronic inflammation of the filters in the kidneys leading to kidney failure.  Kidney failure is best treated by a kidney transplant but patients with C3 glomerulopathy have recurrence of the disease in their transplanted kidney. Treating the disease to prevent kidney failure is the optimal management for C3 glomerulopathy

Her doctor, Julian Midgley, Paediatric Nephrologist and Associate Professor at the University of Calgary has recommended the use of a drug named Soliris as the best treatment for her disease. Soliris blocks the terminal portion of the complement cascade that is activated in C3 glomerulopathy. The total anticipated cost of the treatment is over $700,000 a year. Soliris has not been approved for use in Canada for this disease – but it is approved for other uses. The maker of Soliris has chosen not to pursue Health Canada approval for Soliris as a treatment for C3 glomerulopathy. Approving a drug for a new purpose is expensive and there would be fewer than fifty patients in Canada that might benefit from the treatment.

Paying for Soliris is a problem. The family’s private insurer has declined to cover the cost as the medication is not licensed by Health Canada for C3 glomerulopathy. Alberta Health’s Short Term Exceptional Drug Therapy Program has also denied the request for funding. Alberta Health is refusing to fund the use of Soliris because it is not currently approved for this use in Canada. They have stated that they want statistical evidence, other than case reports, that Soliris is effective –something that would be very difficult to do with an extremely rare disease, and which would also need to be funded.

Wildrose Highwood MLA Wayne Anderson said the NDP government should recognize the special circumstances in the case of a very rare disease and look to use their mandate to publicly finance the prescription.

“This drug could make a world of difference in the life of this girl, but unless the provincial government is willing to cover the expense, it will be too costly for any family,” Anderson said. “With the family having already received a no from their insurance provider, the federal government, and the Short Term Exceptional Drug Therapy Program, they are running out of options. It is my sincere hope the Minister will recognize the special circumstance in this case and approve funding for the required drug to begin treatment.”

“While the cost of the treatment option is very high, it pales in comparison to the ability for this young lady to live out her life free from health complications that will result from her condition continuing to deteriorate,” Wildrose Shadow Health Minister Drew Barnes said. “Health Minister Sarah Hoffman should carefully consider this request, and recognize that our Short Term Exceptional Drug Therapy Program is not properly set up to deal with very rare diseases and needs to be reviewed and improved.”