Replies to post #3542 on Repros Therapeutics Inc (RPRX)

[akasidney86]

Dewophile: what exactly do you mean by "with acute administration", and would the trial design, in your view, meet that standard?
TIA
S

[iwfal]

The list of questions I would ask about the anemia trial:

a) What are the differences in the entry criteria for the ph iii vs the ph iib - if any?

b) For the ph iib subgroup with anemia measurements what were the baseline characteristics relative to the ITT group.

c) What, exactly, is the endpoint for the ph iii?

d) For that endpoint, what was the observed efficacy in the ph iib? The p value? The arm sizes for the subgroup with anemia data? (Note that this is ordinarily what you would see in a published trial)

e) What efficacy did you assume in the powering calculation?

Bad answers to any of the above can indicate a real problem.

Note that I still like the management team here more than most - but as with START, the motto should be "Trust but verify".

Clark

[DewDiligence]

>do I now think this placebo arm poses an inordinate risk? I'll leave that to the statisticians on this board, except to point out there is usually not much of a placebo effect on a hard endpoint like this - in fact, much like an antiviral trial, expectations are that the placebo arms will continue to trend negatively - magnifying any positive efficacy signal in the tx arm<

The lack of a placebo effect in this indication has already been taken into account in the statistical model that generated the 85% power figure. It is not a distinct, bullish factor, as you suggest.

As previously noted, the 85% power figure is not especially illuminating without knowing how dropouts and protocol violations were modeled. Regards, Dew