Canada's cystic fibrosis patients make a plea for access to KALYDECO

TORONTO, Oct. 22, 2012 /CNW/ - As part of the Common Drug Review, Cystic Fibrosis Canada made a submission today on behalf of cystic fibrosis patients to the Canadian Agency for Drugs and Technologies in Health (CADTH). This submission contains valuable patient feedback on the benefits of the drug KALYDECO and can be viewed at http://www.cysticfibrosis.ca/en/Advocacy/Kalydeco.php.

KALYDECO is the first therapy that targets the underlying cause of cystic fibrosis. This drug helps improve the function of the defective protein - leading to better lung function, weight gain and lower sweat chloride levels. For a cystic fibrosis patient with the specific G551D mutation, access to KALYDECO could lead to a healthier, fuller, longer life.

"We are very grateful to the Canadian Agency for Drugs and Technologies in Health for giving patients a strong voice in this very important process," said Ken Chan, VP Advocacy, Research and Healthcare for Cystic Fibrosis Canada. "We heard from more than 40 percent of the affected CF patient population with the G551D gene mutation and their feedback is absolutely unequivocal: access to KALYDECO could make a huge difference in their quality of life."

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 strongly believe that using KALYDECO will allow them to avoid lung transplantation for end-stage lung disease.

"Cystic Fibrosis Canada is calling for a compassionate approach from governments and drug manufacturers when it comes to making new, personalized medicines accessible to patients," said Chan. "The manufacturer and provincial public drug plan administrators have a responsibility to work together on resolving the high price of Kalydeco and ensure CF patients can access this drug in a fair, equitable and affordable manner."

As Canada's only cystic fibrosis patient group, Cystic Fibrosis Canada makes access to medications for patients a priority.

Background: Access to KALYDECO in Canada

KALYDECO (Ivacaftor) is currently under priority review by Health Canada. It has been approved for use by the U.S. Food and Drug Administration and the European Medicines Agency within their respective jurisdictions.

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: FDA)

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 decision making. The CDR will occasionally consider drugs for a pre-NOC Priority Review, which is designed to potentially accelerate access. (Source: CADTH).

The high price of KALYDECO has meant CF patients in certain jurisdictions face barriers to 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 £180,000 per patient per year.

Cystic Fibrosis

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, visit www.cysticfibrosis.ca.

SOURCE: Cystic Fibrosis Canada

Melinda McInnes, Cystic Fibrosis Canada
Program Director, Public Relations and Advocacy Communications
Tel: [phone] ext. 240 | 1-800-378-2233 ext. 240 | Cell: 416-371-5196
Email: [email protected]