Kalydeco Off Label in Canada

[rbk2337]

Hello,

I am trying to procure Kalydeco for an off label mutation in Canada. My doctor is willing to write a prescription, but from what I heard through my clinic is that Vertex is refusing to fill the prescription.
Has anyone been able to procure Kalydeco off label in Canada? Also, does anyone know if it is illegal or why Vertex is not interested in filling the prescription unless I have an on label mutation?

Thanks

 

[stephen]

rbk2337,

Welcome to cysticfibrosis.com.

I’m quite sure that Vertex does not restrict the availablility of Kalydeco. It’s probably your drug provider. If you have any questions, call Vertex directly.

Kalydeco is available “off label” in the USA and it works!!! My doctor thinks it might be effective for all Class 3 and Class 4 mutations.

 

[CFParent2]

Last thing I heard was that Vertex and the Canadian government were in negotiations on what to charge for the drug. I am guessing that is holding up everyone in Canada from getting the drug unless you are in a study? Pretty crazy that a cure/treatment is finally discovered and governments/drug companies/insurance companies are stopping us from getting the cure.

Not sure if it would be possible to hold anyone legally liable for health decline while people get this stuff figured out.

 

[RSR]

Kalydeco was approved by Health Canada in November of 2012. Since then, negotiations have been going on with Vertex to get the price down from the $348,000 annual cost per patient they want for it in Canada.
Late last month an agreement in principle was reached with Vertex for a new price for Canada, and since health care is a provincial responsibility, each province is now expected to opt in and fund Kalydeco under their drug plans with this new price.
As a result, only about 20 of the roughly 118 people in Canada with the G551D mutation have had access to Kalydeco because they have the private insurance plans to cover it. In addition, some folks have had access to it under a Compassionate Care arrangement with Vertex. Everyone else in need of Kalydeco will have to wait for it until the province they are in has signed their own deal with Vertex for it.