Well Davisite … hero or zero for SI I think very much would have depended on the results from the P3 trial in adults. And given MSB’s track record for achieving primary endpoints the cat could hardly have been said to be in the bag.
Posters here continue to be completely bamboozled about how the FDA (the judge) could overturn the Ad Com clinical experts (the jury) vote of 9:1.
Let me offer one reading of the tea leaves.
The FDA calls for Ad Com meetings when it has genuine uncertainty about how to interpret complex clinical trial results. In the context of this genuine uncertainty what independent experts (that the FDA chooses) make of the complex trial results is highly influential.
Now read the FDA briefing notes and there’s a problem with this. The FDA doesn’t express genuine uncertainty about the results at all. They hated pretty much everything MSB had done … and the big mess that had been created (in their eyes) was all MSB’s fault. And note further ... the results are not complex at all - they are as you would expect from a single arm trial very straight forward.
So why hold the Ad Com meeting? Well I had forgotten – sponsors can request a Ad Com meeting in the context of dispute during a rolling BLA. Which if you read the between the lines of MSBs reports – buried very deep – are some vague hints that not all was not going entirely smoothly.
But why did the FDA agree to the Ad Com meeting? Well they did have genuine uncertainty about the next steps. Was a RCT in children possible or did this now have to go into adults.
If you listened to the actual Ad Com meeting MSB were outstanding in prosecuting their case for efficacy. The FDA in contrast were half-hearted in the negative. The reason I think is that the clinical experts were not actually experts at evaluating the methodological strengths of clinical trial results.
And so to the FDA it didn’t matter much what the members thought about something that the FDA are the experts on and had already made their minds up about.
Now from here each side simply does its job. The FDA says to MSB go way and just get on with running the P3 trial in adults. MSB doesn't want to go away at all - it knows the risks in meeting P3 primary endpoints and so will leave no stone unturned to avoid another trial.
(20min delay)