This is how I feel about it, nicely put SJ. If ad
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Posted On: 01/27/2020 4:29:44 PM
This is how I feel about it, nicely put SJ. If additional cancer patients continue to demonstrate the same results we've seen in the first two patients... well, then I don't know what accelerated approval is for if not for this situation. Drug with a well-evidenced safety record, in a deadly indication with no approved treatment, showing high response rates with (hopefully) strong responses. Seems like a slam dunk.
As to the direct question about is X patients enough, I recall a Sarepta drug (exondys 51, for DMD) receiving AA with a very small number of patients overall, but that was a little bit different situation. Small N, and even smaller number of responders, but a longer duration trial. There was also quite a bit of controversy around that one. My thought would be that if exondys 51 got through with limited N, limited response rates, and some safety concerns... then extremely limited N (for efficacy only, safety proven here) shouldn't be overly problematic in this instance.
As to the direct question about is X patients enough, I recall a Sarepta drug (exondys 51, for DMD) receiving AA with a very small number of patients overall, but that was a little bit different situation. Small N, and even smaller number of responders, but a longer duration trial. There was also quite a bit of controversy around that one. My thought would be that if exondys 51 got through with limited N, limited response rates, and some safety concerns... then extremely limited N (for efficacy only, safety proven here) shouldn't be overly problematic in this instance.
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