GTC Biotherapeutics (fka GTCB)

[exwannabe]

Re: 2 points.. Point 1

In any non-inferiority trial of A vs B the goal is to show that A is better than "half" of the B "treatment effect". How that exact value is defined seams to be very open.

So in ANY NI trial, we must pre-determine what the "half" value is. To do this, we MUST look at historical B vs placebo (or SOC). This sets the NI target for the trial to beat relative to B.

In this trial, it's somewhat confusing because the control arm itself is semi-historical, but this matters nadda.

The comparison in this trial is between rAT vs (AT arm + NIDelta), where the NI delta is something like historical (placebo-AT)/2.

Given that I assume the comparison arm will come out at near 0, the biggest variable in the goal will be the NI Delta, which will be based on the historical rate of symptomatic DVT.

WRT the lack of a SPA, I have a serious question.

Would GTCB have discussed the NI delta with the FDA before hand?

If not, then a "close call" trial result would be a serious issue.

[I assume the efficacy data will come in very sound, and this is all irrelavent]