I am reposting my original post as the last topic has stopped working (cant read the bottom few posts, I have emailed the website and it has not changed).
"A few weeks ago I went to the TGA website and saw that Kalydeco was listed as an orphan drug but not listed on the TGA. I phoned the TGA to find out if Vertex had applied to get Kalydeco listed in Australia. The TGA told me this information was confidential, but that Vertex may be able to answer my question.
I emailed Vertex and they responded that a company called Idis (<a href="http://www.idispharma.com/" target="_blank">http://www.idispharma.com/</a>) was responsible for the distribution of Kalydeco outside of the US. I emailed Idis and was told that “Vertex has every intention of introducing a compassionate use program for Kalydeco in Australia. However this program will not launch until preliminary discussions have occurred with the Therapeutic Goods Administration so that we fully understand the implications of such a program and potential impact, if any, on the regulatory filing. We anticipate that these preliminary discussions will take place in the middle of 2012.”
I emailed back asking them to clarify what “compassionate use program” meant, but they realised from my email that I was a patient so they could not answer (they have a policy that they will only provide information to doctors). I am hoping my doctor will find out what that means, as it could either refer to access or cost.
In order for people with CF in Australia to have access to Kalydeco this needs to be listed on the TGA, and most likely then on the PBS ($5.80 cost per month) so we can afford it (its $294,000 in the US per year, but there are rumours it will be less in the UK/Aus. Given that our hospital admissions are about $20,000 each I think it would have to be less than $100,000 to be cost effective in Australia). It looks like it might be listed on the TGA in the second half of this year. I've heard there were 5-10 people in Australia who did the Vertex trial and would have been eligible for the open label extension trial, however this would end at the beginning of next year so hopefully we have access to Kalydeco by then (or they would have to stop taking it).
When I spoke to the TGA a few weeks ago I was also told that my doctor could apply through the special access scheme to get access to a medication not listed on the TGA. I am trying to get my doctor to do this, however I will then have the cost barrier as a fairly significant issue if I am allowed to import it from the US!
I think it would be a good idea for anyone in Australia who wants more information to email/get their doctor to email global@idispharma.com . You never know, if they receive a few emails from Australia & we stay in the front of their minds we may speed the process up!'
We have started a facebook group, please join if you are interested in lobbying for the approval and funding of Kalydeco in Australia:
https://www.facebook.com/groups/343431685698805/
"A few weeks ago I went to the TGA website and saw that Kalydeco was listed as an orphan drug but not listed on the TGA. I phoned the TGA to find out if Vertex had applied to get Kalydeco listed in Australia. The TGA told me this information was confidential, but that Vertex may be able to answer my question.
I emailed Vertex and they responded that a company called Idis (<a href="http://www.idispharma.com/" target="_blank">http://www.idispharma.com/</a>) was responsible for the distribution of Kalydeco outside of the US. I emailed Idis and was told that “Vertex has every intention of introducing a compassionate use program for Kalydeco in Australia. However this program will not launch until preliminary discussions have occurred with the Therapeutic Goods Administration so that we fully understand the implications of such a program and potential impact, if any, on the regulatory filing. We anticipate that these preliminary discussions will take place in the middle of 2012.”
I emailed back asking them to clarify what “compassionate use program” meant, but they realised from my email that I was a patient so they could not answer (they have a policy that they will only provide information to doctors). I am hoping my doctor will find out what that means, as it could either refer to access or cost.
In order for people with CF in Australia to have access to Kalydeco this needs to be listed on the TGA, and most likely then on the PBS ($5.80 cost per month) so we can afford it (its $294,000 in the US per year, but there are rumours it will be less in the UK/Aus. Given that our hospital admissions are about $20,000 each I think it would have to be less than $100,000 to be cost effective in Australia). It looks like it might be listed on the TGA in the second half of this year. I've heard there were 5-10 people in Australia who did the Vertex trial and would have been eligible for the open label extension trial, however this would end at the beginning of next year so hopefully we have access to Kalydeco by then (or they would have to stop taking it).
When I spoke to the TGA a few weeks ago I was also told that my doctor could apply through the special access scheme to get access to a medication not listed on the TGA. I am trying to get my doctor to do this, however I will then have the cost barrier as a fairly significant issue if I am allowed to import it from the US!
I think it would be a good idea for anyone in Australia who wants more information to email/get their doctor to email global@idispharma.com . You never know, if they receive a few emails from Australia & we stay in the front of their minds we may speed the process up!'
We have started a facebook group, please join if you are interested in lobbying for the approval and funding of Kalydeco in Australia:
https://www.facebook.com/groups/343431685698805/