Looking at the table, I just can't figure out how they failed on the primary endpoint. How could they have screwed up the scoring rules to #$%$ defeat from the jaws of success? My observations:
1. The placebo results look like a random distribution around the baseline, 21 patient-steps positive, 21 patient-steps negative. A net zero change. Arikayce has 17 patient-steps positive (7 due to unrelated death penalty), 36 patient-steps negative. An improvement of net -19 patient steps. Is it possible that all of the positive step change scores in the Arikayce arm were due to trial-dropout penalties?
2. The best improvement in the placebo arm was a single patient with -3 steps. That means that the 3 culture conversions in the placebo arm were from a baseline of only 2 or 3. The Arikayce arm had 1 patient with a -6 step improvement.
Edge - efficacy in the Primary endpoint was determined by patient numbers alone, i.e. the number who recorded an improvement of at least one step.
41% of the patients on Arikace had done so by Week 12. Presumably that number needed to be 60% or thereabouts to be considered of statistical significance.
By Week 24 the percentage may have been higher. But more importantly, the 41% was significantly higher than the 29% in the Placebo arm - which is supportive of the finding of efficacy in the far more important endpoint we did hit.
Hitting that clinically significant endpoint earned serious bonus points :-)
Fud, I know what you're saying. And I know that the secondary endpoint is more important. But I am an engineer, and It just hurts me to look at data like that. To my eye, the data shows placebo to be perfectly inert, and Arikayce to be clearly effective, but we get headlines saying that the trial was a failure.
To me, the dropouts due to adverse events should have been treated as a safety issue, and not a source of penalties that skews the true results.
Someone clearly messed up in how they selected the endpoint. Maybe this was a Gupta faux pas? It doesn't matter as it doesn't negate the success of the outcome for many of the patients. Couple this with the outstanding results on the secondary endpoint and we have the only treatment on the planet with this level of success. All of the negative comments are just gibberish meant to obfuscate this fact. And, if you are short, you had better think twice about your position because the steamroller starts picking up speed in a couple of days.
It reminds me of the story of the young boy waking up in the morning and going to the kitchen oven, and opening the door. His mother is standing there, and asks her son what he's looking for. He says he was looking for some of the hot $nAtch that dad was raving about last night.