Revision - Decision Fails Patients with Transfusion-Dependent Anemia
TORONTO, June 1 /CNW/ - Patients requiring regular blood
transfusions are challenging a recent decision by the Committee to
Evaluate Drugs (CED) that severely limits access to the new iron
removal (chelation) therapy, Exjade.
"The decision reflects a total lack of understanding of the importance of Exjade to patients with transfusion-dependent anemias," said Riyad Elbard, president of the Thalassemia Foundation.
These patients depend on blood transfusions to survive, but ironically, the blood leaves excess iron that can damage the liver and heart and lead to early death unless it is removed, or chelated, from the body. Patients at risk are those with rare blood disorders and bone marrow failure, such as thalassemia, sickle cell disease, Fanconi's anemia, aplastic anemia and myelodysplasia.
Exjade is the first once-a-day oral medication approved in Canada that binds to the iron to remove it from the body. Before Exjade, the only treatment for iron overload was deferoxamine, an infusion drug that required patients to be connected to a pump for 8 to 12 hours per day five to seven days a week. The treatment has many side effects and the process is so onerous that many patients, especially young adults, do not comply sufficiently.
Perhaps a dozen deaths among this patient population over the past few years are directly attributable to the complications of iron overload.
"Many of our members will eventually suffer from transfusion-related iron overload. Without iron chelation therapy these members will suffer organ damage and die from iron overload. Exjade represents a complete breakthrough in the area of iron chelation. It has the potential to improve the quality of life of our members as it places less of a burden on the patient allowing them the freedom to take a once-a-day pill," said Dottie Nicholas, president of the Sickle Cell Association of Ontario.
Also at risk are typically older patients with myelodysplasia who require blood transfusions. Iron chelation is standard of care, but many cannot tolerate the toxicities of deferoxamine or the infusion regimen.
"Unfortunately, the Ontario decision denies patients with myelodysplasia access to Exjade, so only those fortunate enough to have a private drug plan have this treatment option," said Dr. Richard Wells, Chair of the Medical-Scientific Advisory Committee, Aplastic Anemia and Myelodysplasia Association of Canada.
Patients believe it is critical that decision makers understand the impact of the decision to severely limit access to Exjade as a treatment alternative for transfusion-related iron overload.
Lack of Patient Voice
"When new therapies cost more than old therapies, we appreciate that drug plan decisions must be guided by scientific and economic assessment to assure the best use of public healthcare funds. However, they must also take into consideration the patient impact," said Durhane Wong-Rieger, president of the Anemia Institute.
Unfortunately, patients have limited input into health policy decisions. Patients requiring blood transfusions were long ignored by the healthcare system until tragedy struck in the form of HIV and Hepatitis C. Many lives would have been saved if the authorities had listened to the patients, who were among the first to realize something was terribly wrong. It took thousands of lives and an inquiry to change the blood system and to begin to include the patient voice at the decision-making table.
Now, Canada's blood supply is among the safest in the world. But patients are still at risk, unless they receive appropriate therapy to manage the iron overload due to transfusions.
Call for Access
The patient and the physician community call upon the Ontario government and the drug manufacturer, Novartis Pharmaceuticals Canada, to work together in the best interest of patients to assure appropriate access to iron chelation therapy. We urge a speedy decision to assure that Exjade is available as a treatment option for all patients at risk for transfusion-dependent iron overload.
/For further information: or to request an interview please
Jennifer Gordon, Carolyn Santillan, Edelman, Phone: (416) 979-1120 ext. 345,
351, Jennifer.firstname.lastname@example.org, Carolyn.email@example.com/
Posted: June 2007
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