The Reasons for Failure at the FDA

Here's a good retrospective in JAMA, from the FDA, about what's happened when the agency has rejected a new molecule entity (NME). The authors look over the data set from 2000-2012 to see what the most common reasons for trouble were, and what happened after that in each case. Overall, 302 new molecules were submitted during those years. Half of them were rejected the first time through - a figure that does not jibe with most investors' ideas of the prospects for any given drug, for sure. But Tolstoy was right - approved drugs are all alike, but every rejected one is rejected in its own way. Looking over those, it appears that a bit under half of the rejects eventually did get approved (meaning that, ultimately, about 73% of all NMEs do eventually make it). But the median delay to approval, after that first rejection, was well over a year. And that has to do with the reasons for rejection. They vary, and none of them are easily fixed: Of the unsuccessful first-time applications, 24 (15.9%) included uncertainties related to dose selection, 20 (13.2%) choice of study end points that failed to adequately reflect a clinically meaningful effect, 20 (13.2%) inconsistent results when different end points were tested, 17 (11.3%) inconsistent results when different trials or study sites were compared, and 20 (13.2%) poor efficacy when compared with the standard of care. But you'll note that while some of these are just nasty results in the clinic (like insufficient efficacy), so...
Source: In the Pipeline - Category: Chemists Tags: Regulatory Affairs Source Type: blogs