This could be a Chance for Life for Canadians with rare disorders

    TORONTO, Sept. 2 /CNW/ - When Canada's health ministers sit down next
week in Quebec City, they may make history by introducing Canada's first
coordinated strategy for treating Canadians living with rare disorders. The
stakes are high. Canadians suffer and die from these deadly illnesses while
knowing that treatments finally exist, but are out of reach. This may be about
to change. The Canadian Organization for Rare Disorders (CORD) has learned
that "drugs for rare disorders" will be on the agenda when the federal,
provincial, and territorial health ministers meet on September 4-5 in Quebec
    "This could be a turning point in Canada's treatment for patients with
rare disorders," said Durhane Wong-Rieger, president of CORD. "In 2005,
patients and families camped for two days outside the meeting room of the
ministers of health and, at the end, the ministers promised not only to treat
the patients who were present but also to develop a plan for drugs for rare
disorders. Today, three years later, there is still no plan."
    Internationally, a rare disorder is defined as a condition that affects
fewer than 1 in 2,000 persons. More than half are diseases that affect
children and many are severe and life-threatening. Some of the more well known
are cystic fibrosis, muscular dystrophy, pulmonary hypertension, and
thalassemia. Others, like Pompe disease, hypophosphatasia and paroxysmal
nocturnal hemoglobinuria (PNH), affect fewer than 25 Canadians.
    Canada is one of the very few developed countries without an "orphan
drug" strategy. As a result, new therapies are typically not accessible to
Canadian patients until, two to five years after these innovative, life-saving
treatments are available in the rest of world. And this is if they are lucky,
as sometimes, these treatments never become available.
    CORD has put forward a practical proposal based on a year of consultation
with Canadian and international experts and is hopeful it will be adopted by
health ministers. The proposed Chance for Life Strategy combines "best
practices" from around the world. According to Dr. Wong-Rieger, "The strategy
meets the needs of all stakeholders and is a way forward from this impasse
that is costing the lives of vulnerable Canadians."
    The Chance for Life Strategy proposes that once a new drug for a rare
disorder has been approved by Health Canada, an advisory committee will
develop treatment guidelines based on international standards. Patients who
meet the treatment criteria will have immediate access. Those who achieve
clinical "benchmarks" indicating the drug is effective will stay on therapy;
those who do not, will agree to stop treatment. Patients are entered into a
registry where they are monitored for clinical outcomes and adverse reactions.
These data will contribute to an international database to evaluate how well
the drug works and the long-term effects.
    Because these patient populations can be very small, it is important that
knowledge is shared internationally. By the time these drugs are available in
Canada, the international community of experts has most often developed
treatment guidelines. Canada needs to be treating according to international
standards, both to share data, and to ensure that our patients are being
treated comparably.
    CORD and patient groups such as the Canadian Association of Pompe are
once again calling upon health ministers to endorse the Chance for Life
Strategy immediately to ensure access to therapies that have been approved by
Health Canada for rare disorders, but are not universally funded across
Canada. For example, the drug Myozyme is used for the treatment of Pompe
disease, an ultra-rare genetic disorder with an estimated 20 patients in
Canada. It is being funded in Alberta for one patient on an exceptional basis,
but not in any other province or territory. Elaprase, a therapy approved by
Health Canada for Hunter Syndrome (MPS II), is available to patients in
British Columbia and Alberta, but not to the six patients in Ontario. This is
despite television coverage of the patients' plights, front-page news stories,
signed petitions, and even a YouTube video from Canadian basketball icon,
Steve Nash, calling for treatment.
    "To date, almost no treatment for a rare disorder has been funded in
Canada without strident advocacy," said Ed Koning, president of the Canadian
Fabry Association and vice-president of CORD. Ed and his wife were among those
who brought placards and petitions with hundreds of signatures to the
ministers' meeting three years ago.
    Kirsten Harkins, executive director of the Canadian Society for
Mucopolysaccharide and Related Disorders was there with her young son, who
suffers from MPS-I. She said, "It's heartbreaking when you know this miracle
drug can halt the crippling effects of this disease and give your child a
chance at a normal life, but it's not funded in Canada."
    This past May, Members of Parliament voted almost unanimously in support
of a private member's motion calling for treatments for rare disorders. By
implementing the Chance for Life Strategy, Canadian ministers of health could
move Canadians with rare disorders from being among the worst treated in the
developed world to among the very best.

    About CORD

    The Canadian Organization for Rare Disorders is Canada's national network
for organizations representing all those with rare disorders. CORD provides a
strong common voice to advocate for health policy and a healthcare system that
works for all those with rare disorders. CORD, in partnership with member
associations, works with governments, researchers, clinicians and industry to
promote research, diagnosis, treatment and services for all rare disorders in
    To find out more about CORD, visit

For further information:

For further information: Alia Hassan, Cohn & Wolfe, (647) 259-3305,; Angela Covato, CORD, (416) 969-7431,

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Canadian Organization for Rare Disorders

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