Calling for the manufacturer and drug insurance plans to ensure CF
patients receive equitable and affordable access to KALYDECO™
TORONTO, Dec. 3, 2012 /CNW/ - Health Canada has issued a Notice of
Compliance (NOC) for KALYDECO™, approving the drug for use in Canada.
This gives Canadian cystic fibrosis (CF) patients with the G551D
mutation (ages six and older) a chance to gain access to this new,
breakthrough treatment. Cystic Fibrosis Canada calls on Vertex
Pharmaceuticals Inc. and drug plan administrators to work together to
resolve the high price of KALYDECO™ so that CF patients can access this
drug in an equitable and affordable way.
"The approval of KALYDECO™ is just the first step in bringing this new,
breakthrough therapy to CF patients," said Ken Chan, Vice President,
Advocacy, Research and Healthcare. "As one of the most expensive drugs
in the world, CF patients simply cannot afford to pay for this
much-needed medicine. Cystic Fibrosis Canada is calling for a
compassionate approach from manufacturers and drug plan administrators
when it comes to making new, life-changing medicines accessible to CF
KALYDECO™ is the first therapy that targets the underlying cause of
cystic fibrosis. This drug helps to improve the function of the
defective protein, leading to better lung function, weight gain and
lower sweat chloride levels. For a CF patient with the specific G551D
mutation, access to KALYDECO™ could lead to a healthier, longer life.
Canada now joins the United States and the European Union in making
KALYDECO™ available for use for CF patients. The G551D mutation is
present in roughly 100 Canadians with cystic fibrosis.
"When the gene responsible for cystic fibrosis was discovered in Canada
back in 1989, one could only imagine the moment when that discovery
would lead to a drug like KALYDECO™, said Cystic Fibrosis Canada
President and CEO Maureen Adamson. "This is the beginning of good news
for Canadian CF patients, and I look forward to even better news when
this drug will be affordable and accessible to the patients who will
benefit from it." Cystic Fibrosis Canada helped fund the international
team that discovered the CF gene under the leadership of Dr. Lap-Chee
Tsui at the Hospital for Sick Children in Toronto.
Key opinion leaders throughout the Canadian CF clinical community also
strongly support giving patients access to this drug.
"KALYDECO™ is an incredible leap forward in CF therapy," said Dr. Larry
Lands, CF Clinic Director at Montreal Children's Hospital. "Even though
KALYDECO™ as a stand-alone treatment will only help a limited segment
of affected patients, it shows promise as a combination therapy for
other patients with cystic fibrosis, and points the way towards the
development of tailored treatments that will correct the underlying
problem for each and every affected person with cystic fibrosis."
"This treatment represents a paradigm shift in therapy; it's the first
one to target the basic defect in the cells of CF patients," said Dr.
Elizabeth Tullis, Head of Respirology and Director of the Adult Cystic
Fibrosis Program at St. Michael's Hospital in Toronto. "While KALYDECO™
is currently applicable only to a small proportion of CF patients; it
gives us hope that a similar approach can be developed for all people
with cystic fibrosis."
"The future of some babies diagnosed through CF newborn screening is
going to be revolutionized by KALYDECO™," said Dr. Mark Chilvers, CF
Clinic Director, BC Children's Hospital. "CF Clinicians now have access
to a life-changing therapy that targets this specific genetic
In October 2012, Cystic Fibrosis Canada made a submission to the Common
Drug Review (CDR) process in an effort to convince provincial drug
plans to provide Canadian CF patients with access to KALYDECO™.
Testimonials submitted for affected patients who are currently being
treated with KALYDECO™ were compelling. They report transitions from
debilitating illness and exhaustion to having more energy, being able
to finally exercise, being able to spend more time with their families,
and an overall enhanced quality of life. Many patients strongly believe
that using KALYDECO™ will allow them to avoid lung transplantation for
end-stage lung disease. Cystic Fibrosis Canada is awaiting the CDR
recommendation before deciding on next steps.
Background: Access to KALYDECO™ in Canada
Of the 4,000 Canadians living with this devastating disease, about 100
CF patients in Canada have the specific G551D mutation of the CFTR gene that can be treated by KALYDECO™. (Source: Canadian Patient Data
Registry). When taken twice a day with fat-containing food, KALYDECO™
helps the protein made by the CFTR gene function better, improving lung function and weight gain. (Source:
The Common Drug Review (CDR) was established by the federal, provincial,
and territorial government to provide publicly-funded drug plans
(except Quebec) with high-quality drug reviews that will inform their
The high price of KALYDECO™ has meant CF patients in certain
jurisdictions face barriers to accessing the drug despite KALYDECO™
receiving regulatory approval. For example, in England where KALYDECO™
has been approved for use since July 2012, the drug still remains out
of reach to CF patients because of its cost ― estimated at over
£180,000 per patient per year. In the U.S., KALYDECO™ costs around
$294,000 per patient per year.
Vertex, the manufacturer of KALYDECO™, has submitted the drug to the
CDR, a process run by the Canadian Agency for Drugs and Technologies in
Health (CADTH). A team of independent experts will review KALYDECO™,
and compare how well the drug works to current CF treatments, and
whether the drug provides value for money. Based on their findings, a
recommendation on whether or not drug plans should cover the drug will
be issued. After the CDR recommendation has been made, provincial
health ministries (except for Quebec, which has a separate process)
will review KALYDECO™ for reimbursement by publicly funded drug plans.
Cystic fibrosis is the most common fatal genetic disease affecting
Canadian children and young adults. It is a multi-system disease that
affects mainly the lungs and the digestive system. In the lungs, where
the effects are most devastating, a build-up of thick mucus causes
severe respiratory problems. Mucus and protein also build up in the
digestive tract, making it difficult to digest and absorb nutrients
from food. As improved therapies have helped to address the
malnutrition issues, ultimately most deaths related to cystic fibrosis
are due to lung disease. Currently, there is no cure.
Cystic Fibrosis Canada
Cystic Fibrosis Canada is one of the world's top three charitable
organizations committed to finding a cure for cystic fibrosis and is an
internationally-recognized leader in funding CF research, innovation,
and clinical care. We invest more funding in life-saving CF research
and care than any other non-governmental agency in Canada. Since 1960,
Cystic Fibrosis Canada has invested more than $140 million in leading
research and care, resulting in one of the world's highest survival
rates for Canadians living with cystic fibrosis. For more information,
SOURCE: Cystic Fibrosis Canada
For further information:
Melinda McInnes, Cystic Fibrosis Canada
Program Director, Public Relations and Advocacy Communications
Tel: 416-485-9149 ext. 240 | 1-800-378-2233 ext. 240 | Cell: 416-371-5196