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
For further information:
Communications for RQMO
For information : RQMO