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
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
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:
For further information: or to request an interview please contact
Jennifer Gordon, Carolyn Santillan, Edelman, Phone: (416) 979-1120 ext. 345,
351, Jennifer.firstname.lastname@example.org, Carolyn.email@example.com