Well if it met all the end-points with a statistically significant P value with an exceptional safety profile, you would think the FDA would have to be both corrupt and evil to do such a thing. Let's be real, it is going to get approved.
Our challenges are
1) double-blinded study being unblinded due to diarrhea side effect and
2) diarrhea as a side effect.
1) Can be disproved by comparing outcomes of both cohorts who had diarrhea ie Patient A with placebo who had diarrhea but no improvements in outcomes
vs Patient B with medicine who had diarrhea with improvements
2) Can be easily managed by modern medicine.
In addition this is not the final decision. So if trofinetide is not approved, the FDA will give guidelines on how to get it approved.
The only thing preventing me from putting my car and my house on this, is corruption. I do not entirely trust the FDA to not make business based decisions, so never know what is going on behind the scenes. If say the FDA was some sort of infallible incorruptible machine, I would have my house on this.
Am I the only one that is not nervous? I couldn't worry less. It would be more entertaining to watch the FDA try to hold approval back and the following shitshow if they do.
Does anyone know of any more challenges that I may have missed? Its like pulling hens teeth. The drug is not effective enough compared to possible future drugs? I can't really think of anything major in our way.
(20min delay)