Per my recent blog, "Jeremy Bentham for FDA Commissioner, " this comment from a former FDA colleague -- who we'll refer to as "Dr. John Stuart Mill" ...
"One of the points we like to look at, in weighing risk and benefit, is whether there is a subgroup of patients who have a very large effect, one not reflected in the average effect size. A drug with considerable toxicity might be considered acceptable if this were so. Clozapine was approved, for example, despite a 1.5% rate of agranulocytosis, because it worked in people failing other Rx. Lotronex was returned to the market because there were people with immobilizing disease who had clinically highly valuable effects. A way to look at this is to look at the cumulative distribution of effects, a curve that is in fact shown in Aricept labeling. It can reveal a small subset with an unusually large effect. There is no such subpopulation in Aricept treated patients; i.e., there are no highly responsive patients.For a contrast find the recently approved labeling for tetrabenazine, for choreiform movements in Huntington's disease. There is clearly a responsive subset there."
Thank you Dr. Mill.
Thank you Dr. Mill.
