Repost from SA re FDA- see MTBs excellent article

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biopearl
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Joined: Wed Dec 09, 2015 12:12 am

Repost from SA re FDA- see MTBs excellent article

Post by biopearl » Wed Mar 21, 2018 4:34 pm

biopearl
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MTB, this is exactly the problem with the FDAs failure to approve on what appears to be overwhelmingly positive data because of the absence of a traditional control arm. Because of the structure of the R/R study the "control arm" relies on historical data which is all over the map between 7 month OS and 16. So someone like Tom's wife (not to mention JoAnn and to some extent John) have nowhere to go for Imetelstat. The R/R study is closed. As far as I know the Mayo study is also close to new enrollment. I have not heard of Imetelstat being made available on a compassionate basis as yet. We have been told that the "new" FDA will show more flexibility. This is the perfect situation for it. I truly hope Dr. Tefferi can help patients like Tom's wife, Dr. T is certainly in close proximity to Imetelstat and perhaps could get some compassionate approval but there does not seem to be a way to enroll in an active ongoing Imetelstat MF study at this time as far as I know. This is why I think the pressure on Janssen (and from Janssen) and the FDA must be enormous. The FDA is a sophisticated organization. If the "real world" study gives the closes approximation to the current R/R study group they will have to move on it for ethical reasons not to mention sound and outstanding clinical study results. Best wishes to Tom and his wife and others like them. Hope is on the way. bp

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