In october 2000, one share of CTIC was $97000. That must be a mistake right? I guess that's why they invented log scales.
not siginficantly so - but if I was a patient I'd chose the one that gave the extra 2 months - and, more importantly, side effects are less than standard of care. Which one would you chose for yourself? Always need to think about clinical signicance versus statistical significance. hey but I'm only a arthritis doc.
these small numbers continuing 10 weeks after stopping therapy (26 wk rx) is impressive. But if only at 36 wks and not 27 then got to wonder why. Of course, we dont' know if 27 wk data were unimpressive as assumed or not. Could have been even more impressive at 36 wks if continued rx for full interval. NIH.gov does not even mention 36 weeks as secondary outcome - strange. And no one expected a drug so far removed from an "unmet need" would gain approval on phase 2 alone. That's why you do phase 2 - to optimize your selection of endpoints. Were they hard at work mining data or is this legit?
Interesting study of human nature. Even with data as plain as day showing deterioration starting at 24 weeks at the exact time the delayed arm begins their treatment, a few losers will chose to ignore the obvious and cling to an outlandish hope that this drug is not harmful. Even though clearly it is.
What a scam! The sponsor conveniently ignores the most striking feature of the data: patients immediately deteriorate after starting the drug after a very clean 24 wk pretreatment interval. Sorry, but the FDA will notice this.
is a dangerous drug. The only obvious thing from the phase II data is that there is an immediate decline in function after 24 weeks exactly timed with the start of eteplirsen in the delayed rx arm. Take off those ridiculous rose-shaded glasses and look at the data objectively for ONE second and you will save yourself a bundle of cash.
If the study had been well-designed with a 48 week placebo cross-over, then it may have shown the differences to be real, but hey that's why you do phase II so you can get it right in phase III. As it is, there is no difference between placebo and intervention during the only 24 weeks for which the FDA will have any interest. They may get another chance to generate some approvable data, but you need convincing data for early approval. These are not at all convincing.
As you all probably know, the FDA will analyze these data from scratch on their own. Like every other study they have ever evaluated, they will place the t=0 arrow at the time the drug was started, not at a completely arbitrary time point that happens to represent a inflection point that makes the sponsor look good. Move the arrow the graph in the street update to week 24 (the true t=0 for the placebo arm) instead of week 36 and you have a drug with no benefit. "Time before (or after) detectable" is completely arbitrary depending on how much you claim is detectable... picomoles, nanomoles, micromoles