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Thread: Kalydeco for Australians

  1. #1

    Kalydeco for Australians

    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 &ldquo;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.&rdquo;

    I emailed back asking them to clarify what &ldquo;compassionate use program&rdquo; 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/

    0 Not allowed!

  2. #2

    Kalydeco for Australians

    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 &ldquo;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.&rdquo;

    I emailed back asking them to clarify what &ldquo;compassionate use program&rdquo; 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/

    0 Not allowed!

  3. #3

    Kalydeco for Australians

    New information:
    In order for people in Australia to access the medication, it needs to be listed on the TGA and then approved by the PBAC to be listed on the PBS. This is the timeline that we are looking at: Idis have said they will apply to get Kalydeco listed on the TGA in mid 2012. The PBAC holds meetings in March, July and November and applications need to be submitted at least a month beforehand. This means Kalydeco could go to the PBAC meeting in November 2012 or March 2013 (unless Idis applies to get Kalydeco listed on the TGA earlier- then there is a slim chance of getting to the July 2012 meeting). It is unlikely to go to the July/November meetings unless the company is very motivated and prepares their submission to the PBAC quickly, so next March is the most likely (I'm judging this based on how long companies normally take. Although they can re-use part of their submissions for the US & Europe markets, they would still have to change alot before they submit it in Australia). Hopefully if we lobby enough we can speed up the process and get it to the November meeting (or even the July one if the TGA occurs earlier).

    0 Not allowed!

  4. #4

    Kalydeco for Australians

    New information:
    In order for people in Australia to access the medication, it needs to be listed on the TGA and then approved by the PBAC to be listed on the PBS. This is the timeline that we are looking at: Idis have said they will apply to get Kalydeco listed on the TGA in mid 2012. The PBAC holds meetings in March, July and November and applications need to be submitted at least a month beforehand. This means Kalydeco could go to the PBAC meeting in November 2012 or March 2013 (unless Idis applies to get Kalydeco listed on the TGA earlier- then there is a slim chance of getting to the July 2012 meeting). It is unlikely to go to the July/November meetings unless the company is very motivated and prepares their submission to the PBAC quickly, so next March is the most likely (I'm judging this based on how long companies normally take. Although they can re-use part of their submissions for the US & Europe markets, they would still have to change alot before they submit it in Australia). Hopefully if we lobby enough we can speed up the process and get it to the November meeting (or even the July one if the TGA occurs earlier).

    0 Not allowed!

  5. #5

    Kalydeco for Australians

    I just did a search trying to find the specific cut off dates for submissions to the PBAC and found this link. I was very alarmed as soon as I saw that the cut off date for the July meeting was the 7/3/12 for major submisssions, so I rang the PBAC and they confirmed that Kalydeco would be a major submission. This means we are looking at the November meeting as a best case scenario, but with the cut off date 11/7/12 that may not occur either. They have not published the cut off date for the March 2013 meeting, but it would be early November. This makes it much more important that we get an expanded access/compassionate use program here as soon as possible. If someone is quite sick they should do everything they can to get the medication before the TGA/PBAC process, as it will not get through before the end of this year (more likely to be next year). Once the PBAC recommend Kalydeco to be listed, it still takes some time to be listed on the PBS and then available in Australia (we saw this with TOBI last year, it was recommended to be listed in March, then the government delayed it being listed on the PBS to save money, and we finally accessed it in October.) The link is quite interesting, here is a summary: 4 months before the meeting- cut off for major submissions 3 months & 6 weeks before- pre submission meetings with companies 4 weeks before- closing date for consumer comments (I already knew this and thats why I knew that it had to be submitted at least a month before) http://www.health.gov.au/internet/main/publishing.nsf/content/health-pbs-general-listing-dates.htm After looking at that link I'm not surprised that it is a 16 week process, with lots of meetings & communication before the final PBAC meeting. It would be very hard to come to an informed decision in just the three day PBAC meeting without this process beforehand.

    0 Not allowed!

  6. #6

    Kalydeco for Australians

    I just did a search trying to find the specific cut off dates for submissions to the PBAC and found this link. I was very alarmed as soon as I saw that the cut off date for the July meeting was the 7/3/12 for major submisssions, so I rang the PBAC and they confirmed that Kalydeco would be a major submission. This means we are looking at the November meeting as a best case scenario, but with the cut off date 11/7/12 that may not occur either. They have not published the cut off date for the March 2013 meeting, but it would be early November. This makes it much more important that we get an expanded access/compassionate use program here as soon as possible. If someone is quite sick they should do everything they can to get the medication before the TGA/PBAC process, as it will not get through before the end of this year (more likely to be next year). Once the PBAC recommend Kalydeco to be listed, it still takes some time to be listed on the PBS and then available in Australia (we saw this with TOBI last year, it was recommended to be listed in March, then the government delayed it being listed on the PBS to save money, and we finally accessed it in October.) The link is quite interesting, here is a summary: 4 months before the meeting- cut off for major submissions 3 months & 6 weeks before- pre submission meetings with companies 4 weeks before- closing date for consumer comments (I already knew this and thats why I knew that it had to be submitted at least a month before) http://www.health.gov.au/internet/main/publishing.nsf/content/health-pbs-general-listing-dates.htm After looking at that link I'm not surprised that it is a 16 week process, with lots of meetings & communication before the final PBAC meeting. It would be very hard to come to an informed decision in just the three day PBAC meeting without this process beforehand.

    0 Not allowed!

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