Rare and orphan diseases, forgotten by the government - The RQMO demands that Health Minister Réjean Hébert implement a regulatory framework for rare and orphan diseases

MONTREAL, June 10, 2013 /CNW Telbec/ - Following publication on June 3 of the drug list by the l'Institut national d'excellence en santé et en services sociaux (INESSS), le Regroupement québécois des maladies orphelines (RQMO) has expressed its disappointment at the failure to include four orphan drugs on the list of drugs reimbursed by RAMQ.

A long-awaited promise
According to Ouellette, the director of the RQMO, the rationale for refusals contained in the recommendations of the INESSS show that the assessment methods designed for common diseases for which the natural history or indicators are well known, cannot be applied to rare diseases. In her letter, Ms. Ouellette recalls the promises made by the health ministry in 2007 to develop a regulatory framework for rare and orphan diseases applicable to the INESSS drug evaluations. The new Framework would ensure that evaluations are adapted to the particular conditions of drugs that are conceived for people with rare diseases and the difficulty of conducting clinical trials on small populations.

The RQMO also believes that unfairness in the assessments exists not only in terms of standard evaluation methods for the therapeutic value, but also regarding the criteria to access treatment for common illnesses.  For example, often the patient's illness must be quite advanced, leaving little room to prevent complications.

Mme Ouellette condemns the injustice which persists in access to medication for people with rare diseases. One such example, in the June 3 decision by INESSS not to include several drugs approved by Health Canada in the updated list of medications covered by the RAMQ drugs is harming individuals living with rare diseases, namely cystic fibrosis, idiopathic pulmonary fibrosis, Gaucher disease and phenylketonuria. Drug treatments which could improve quality of life, even save lives. These are just a few drugs on a long list of refusals, several of which are the first and only treatments of certain rare illnesses.

An innovative policy
"The Government of Québec must consider a global and innovative policy for purchasing rare disease drugs," Ouellette says. "We must follow the example of countries which address the issue of the overall cost of drugs for rare diseases, not case-by-case. We have to consider how these diseases affect ethical and societal aspects of the lives of people who suffer from these conditions. The portion of the population that suffers from rare diseases is also entitled to benefit from a fair distribution of resources."

About RQMO and rare and orphan diseases
A rare disease is defined as one that affects less than one in 2000 patients. More than 7,000 rare diseases have been identified worldwide. An estimated 5% of the Quebec population may be affected by either of these diseases. The Quebec Coalition of orphan diseases (RQMO) is a non-profit organization whose mission is to provide information and support to individuals and families struggling with a rare and orphan disease, as well as raise awareness of rare diseases.

SOURCE: Regroupement québécois des maladies orphelines

Source :
Sarah Cope
Communications for RQMO
[email protected]
514-585-6223

For information : RQMO
http://rqmo.org/acceuil.html