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GenH
03-06-2012, 01:17 PM
I just posted this on the Australian forums but hardly anyone seems to post/visit the website, so I am posting here as well:

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 (http://www.idispharma.com/) 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). It looks like vertex might apply to the TGA 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.

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 [email protected] . 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!

GenH
03-06-2012, 01:17 PM
I just posted this on the Australian forums but hardly anyone seems to post/visit the website, so I am posting here as well:

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 (http://www.idispharma.com/) 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). It looks like vertex might apply to the TGA 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.

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 [email protected] . 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!

Incomudrox
03-06-2012, 01:38 PM
This is great information! Welcome to the forum and thanks for posting this! I'm sure many will find this information very useful.

Incomudrox
03-06-2012, 01:38 PM
This is great information! Welcome to the forum and thanks for posting this! I'm sure many will find this information very useful.

GenH
03-06-2012, 02:40 PM
Thanks Incomudrox, I have been following the forums (australia, uk, cf2chat and here) for several years and finally found the time to post! I have been following the research about vx770 (reading articles etc-I have studied biomedical science at uni) for several years as well, and unfortunately did not do the phase 3 trial as it was not held in my state. It is quite frustrating knowing there is a medication out there that should make an instant difference & that I can't access it! I know it will come to the UK and Australia before lots of other parts of the world though, I have heard that Romania & New Zealand dont even pulmozyme for everyone (which is way cheaper!)

GenH
03-06-2012, 02:40 PM
Thanks Incomudrox, I have been following the forums (australia, uk, cf2chat and here) for several years and finally found the time to post! I have been following the research about vx770 (reading articles etc-I have studied biomedical science at uni) for several years as well, and unfortunately did not do the phase 3 trial as it was not held in my state. It is quite frustrating knowing there is a medication out there that should make an instant difference & that I can't access it! I know it will come to the UK and Australia before lots of other parts of the world though, I have heard that Romania & New Zealand dont even pulmozyme for everyone (which is way cheaper!)

mima63
03-06-2012, 10:02 PM
thank you GenH - we are in Australia and we would like to access Kalydeco right now - even if the cost is enormous! Our doctor has been following it up and will help us with any access issues. If you want to email me we can try and help each other and form some sort of group. my email address is [email protected] My daughter is 22 and is also G551D and DF508. We need to get this drug as soon as we can as we are hoping it will make a significant difference to her health.

mima63
03-06-2012, 10:02 PM
thank you GenH - we are in Australia and we would like to access Kalydeco right now - even if the cost is enormous! Our doctor has been following it up and will help us with any access issues. If you want to email me we can try and help each other and form some sort of group. my email address is [email protected] My daughter is 22 and is also G551D and DF508. We need to get this drug as soon as we can as we are hoping it will make a significant difference to her health.

lilmac7
03-12-2012, 10:58 PM
Yup, the medical politricks at hand... hope it goes well and gets to the UK also. I believe the pharmacy here where I live in the Cayman Islands imports quite a bit of meds from the UK.

Edit: Just to clarify, I know this is not for me or at least not yes as I'm not the G551D or whchever it is that it's for but I'm thinking ahead for the other one for DF508 or if this proves effective for DF508 in testing then at least I can judge how long itll take for the other to get to the UK once approved in the US.

lilmac7
03-12-2012, 10:58 PM
Yup, the medical politricks at hand... hope it goes well and gets to the UK also. I believe the pharmacy here where I live in the Cayman Islands imports quite a bit of meds from the UK.

Edit: Just to clarify, I know this is not for me or at least not yes as I'm not the G551D or whchever it is that it's for but I'm thinking ahead for the other one for DF508 or if this proves effective for DF508 in testing then at least I can judge how long itll take for the other to get to the UK once approved in the US.

cfsucks
03-12-2012, 11:32 PM
anyone hear anything about it arriving in canada? i contacted the canadian cf foundation, but they never replied (what a joke)- should i contact vertex?

cfsucks
03-12-2012, 11:32 PM
anyone hear anything about it arriving in canada? i contacted the canadian cf foundation, but they never replied (what a joke)- should i contact vertex?

livinglovinggrace
03-13-2012, 06:35 AM
Hi GenH and mima63,

I am in Australia too. I am a double delta so this drug won't help me yet but please let me know if I can be off any help drumming up support to get this drug onto the PBS. I think it is important for all CF'ers to support this not only for the good of the people that it will help directly but also in the hope that once this drug makes it other similar drugs may get on there quicker.

Feel free to PM me if I can be of help or contact me via my blog which is linked below. Thanks too for providing this info.

livinglovinggrace
03-13-2012, 06:35 AM
Hi GenH and mima63,

I am in Australia too. I am a double delta so this drug won't help me yet but please let me know if I can be off any help drumming up support to get this drug onto the PBS. I think it is important for all CF'ers to support this not only for the good of the people that it will help directly but also in the hope that once this drug makes it other similar drugs may get on there quicker.

Feel free to PM me if I can be of help or contact me via my blog which is linked below. Thanks too for providing this info.

jamesB
03-13-2012, 01:20 PM
Thankyou for the great info GenH.

jamesB
03-13-2012, 01:20 PM
Thankyou for the great info GenH.

mima63
03-14-2012, 04:40 AM
https://www.facebook.com/groups/343431685698805/
I have started a Facebook Group called "Kalydeco for Australians" - as a central place for gathering information and looking at how we can lobby effectively.
Karen

mima63
03-14-2012, 04:40 AM
https://www.facebook.com/groups/343431685698805/
I have started a Facebook Group called "Kalydeco for Australians" - as a central place for gathering information and looking at how we can lobby effectively.
Karen

GenH
03-15-2012, 02:06 PM
CFSucks- you have nothing to lose by contact vertex, they may tell you more information about when it will come to Canada, or if there is another company marketing it there. I would definitely do it!
Livinglovinggrace- thanks for your support, a facebook group has been started if you or anyone else would like to join
Kalydeco for Australians: https://www.facebook.com/groups/343431685698805/

GenH
03-15-2012, 02:06 PM
CFSucks- you have nothing to lose by contact vertex, they may tell you more information about when it will come to Canada, or if there is another company marketing it there. I would definitely do it!
Livinglovinggrace- thanks for your support, a facebook group has been started if you or anyone else would like to join
Kalydeco for Australians: https://www.facebook.com/groups/343431685698805/