Quebec PNH Patient Association extremely disappointed by INESSS' position on only life-saving treatment

MONTREAL, June 3, 2011 /CNW/ - The Association québécoise de l'HPN (paroxysmal nocturnal hemoglobinuria) is both surprised and dismayed by yesterday's recommendation by the Institut national d'excellence en santé et services sociaux (INESSS) to not include Soliris on Quebec's public drug insurance plan drugs list. The President of the Association québécoise de l'HPN, Barry Katsof, cannot understand INESSS' position given the numerous studies and clinical cases clearly demonstrating that this drug is saving the lives of people suffering from this ultra-rare blood disease.

"In terms of efficiency, Soliris has been proven not only to save the lives of PNH patients, but also to allow those who receive the treatment to lead active and productive lives, and return to having a normal life expectancy," said Katsof. "As for the budgetary impact, it is negligible given the rarity of the disease and the very small number of patients who may require access to Soliris through the public drug insurance plan."

According to Dr. David Roy, director of the research laboratory in ethics and aging of the Research Center of the University Geriatrics Institute of Montreal, INESSS' recommendation on Soliris has created a fundamental inequity among PNH patients. "The direct consequence of this recommendation is that it established an inequity between patients suffering from PNH, because those who do not have the financial and social resources cannot have access to this life saving treatment."

Dr. Roy added that it would have been reasonable and equitable for the government to fund the drug, as 26 other industrialized countries already do, according to three criteria. "Recent studies prove Soliris' efficacy (Kelly et al.) in curbing high morbidity and premature mortality, and only a very small number of Quebecers patients are asking government for access to this treatment."

This funding refusal from INESSS has serious implications for patients with PNH, as they now face the uncertainty around their future access to treatment. Moreover, this refusal creates additional barriers in the process to access Soliris, which already requires considerable efforts for patients given the provisions under public drug insurance.

To date, less than twenty patients are suffering from PNH in Quebec. Unfortunately, a small number of patients are still waiting for access to Soliris. Some have been luckier than others, like Gisèle Sauriol who has been living with the disease for 15 years. "Four months ago, with the help of the Association and the RAMQ, my insurance company gave me access to Soliris, a treatment that allows me to fight the disease and live a normal life. The effect of the drug was immediate and I quickly saw positive results. My only wish is for all patients suffering from PNH in this province to have access to this life-saving medication that gave me back a healthy quality of life," declared Ms. Sauriol.

Soliris, available since 2009 in Canada, is the first and only treatment for PNH that allows a patient's life expectancy to return to that of a healthy person1. PNH is a very rare, progressive and fatal acquired disease that affects the blood and major organs. It develops without warning, usually when patients are in the prime of their lives. It is characterized by haemolysis (the destruction of red blood cells) which can lead to life-threatening blood clots, kidney disease, pulmonary hypertension and other catastrophic consequences. Without treatment, approximately one‐third of PNH patients do not survive more than five years and about half die within 10 years from the time of diagnosis.

About the Association québécoise de l'HPN

The Association québécoise de l'HPN is a not-for-profit Canadian organization formed in 2009. The mission of the organization is to connect Canadians affected by Paroxysmal Nocturnal Haemoglobinuria and advocate best possible care for patients. The organization also provides support to caregivers, and works to increase awareness and understanding of PNH.

1 Kelly RJ, Hill A, Arnold LM, Brooksbank GL, Richards SJ, Cullen M, Mitchell LD, Cohen DR, Gregory WM, Hillmen P.  Long term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival. Blood. Prepublished April 1, 2011; DOI 10.1182/blood-2011-02-333997.

SOURCE Association québécoise d'hémoglobinurie paroxystique nocturne

For further information:

Andréan Gagné
Cohn & Wolfe | Montreal
514-845-2257, ext. 4224

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Association québécoise d'hémoglobinurie paroxystique nocturne

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