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Re: Well done ALNYYes. It will be unapprovable for a broad indication. They will have to find a subset to get approval in. Patients that cannot have Patisaran for some reason or perform further studies or re-analysis for some other subset. I just posted something about this on the IONS board. If you have a disease with effectively no treatment, and one new drug with some efficacy and some complications the FDA can decide based on the disease severity and the drug risk/benefit profile to approve or not. If a year after approval the FDA gets a new application that drug has to be at least as good in non-inferiority tests or be better than the standard. If the 2 new drugs are submitted together near the same time the FDA would want to see the data from both. If one is more efficacious and less dangerous then that drug would be approved. But the second one would have to be very comparable in risk/benefit to be approved. If it is less effective or more importantly more toxic it cannot be approved. One death would result in a lawsuit. Why did you approve this drug when there was a safer/better drug. The only way it gets approved is for a limited indication. A subset better represented in their cohort or for patients who cannot receive the better drug. The FDA does not care about convenience or patient's wanting to be at home. You have to wonder why IONS did not release their data versus baseline, just loads of comparative p values. Not even the absolute improvement of mNIS compared to placebo. Or now that ALNY has come out and said "improved compared to baseline" why did IONS not come out and say, oh yeah we did too. You can be assured their big pharma partner saw those IONS figures, assessed risk/benefit and bailed. ego |
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Msg # | Subject | Author | Recs | Date Posted |
33785 | Re: Well done ALNY | jetmanbash | 1 | 9/21/2017 11:30:05 PM |
33787 | Re: Well done ALNY | arthurs1 | 1 | 9/22/2017 10:39:22 PM |