SASKATCHEWAN TO PARTICIPATE IN RARE DISEASE DRUG STUDY

Saskatchewan will participate in the Canadian Fabry Enzyme Replacement Therapy Research Consortium study to review the effectiveness of drug therapies for people with Fabry’s, a rare, genetic disease.

As part of this study, the federal and participating provincial governments, in conjunction with two drug manufacturers, are providing enzyme replacement therapy for Fabry patients who meet the criteria of the study.  Five Saskatchewan residents are expected to meet study criteria. 

“The issue of expensive drugs for rare diseases is a complex one that is being discussed among all provinces, territories and the federal government,” Health Minister Len Taylor said.  “Participating in studies like this furthers our understanding of rare diseases and the effectiveness of available drug treatments.”

In September 2004, as part of the 10-Year Plan to Strengthen Health Care, First Ministers directed federal, provincial and territorial health ministers to establish a ministerial task force to develop the National Pharmaceuticals Strategy (NPS).  Expensive drugs for rare diseases is one of the five priority areas of the NPS.  Work in this area is focusing on establishing a framework for aligning regulatory and reimbursement systems across the country to provide equitable access to proven drug therapies for rare diseases. 

Rare disease therapies are often prohibitively expensive and do not meet the common standards of evidence for provincial drug plan coverage, as few eligible participants rarely provide statistically significant results.

Fabry disease is caused by a deficiency of the enzyme needed to metabolize the body’s lipids, which then build up to harmful levels in the eyes, kidneys, autonomic nervous system, and cardiovascular system.  Those with Fabry’s are at an elevated risk of heart attack, stroke and/or kidney disease.

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For more information, contact:

Joan Petrie
Health
Regina
Phone: 306-787-4083