Biotech Values

[biomaven0]

would at least have had to pass a non-inferiority trial

The FDA mandate is to approve drugs that are (1) safe and (2) effective. It's fine (and much more straightforward) to show "effective" against a placebo, as you don't have the uncertainty about the effectiveness of the comparator drug to worry about.

The problem comes when you can't ethically use a placebo at all, such as in antibiotic trials for serious infections. Then you have to resort to non-inferiority trials.

In oncology, one trend has been to use the new drug as an "add-on" to existing therapy - so the trial is new drug + existing drug vs. placebo + existing drug, and the trial will attempt to demonstrate superiority.

The FDA only really gets into drug comparisons when there are safety concerns surrounding the potential new drug. Technically that's beyond their purview, but the new de facto standard seems to be if the new drug isn't an advance over existing drugs and has safety concerns, they find a way to turn it down.

Peter