2 ms·
In the future this sort of thing will inevitably be viewed the way we view bloodletting with razors and leeches -- if not more harshly. They didn't know any be
by A_D_E_P_T 3y ago
In the future this sort of thing will inevitably be viewed the way we view bloodletting with razors and leeches -- if not more harshly. They didn't know any better, way back when. We ought to know better.
It's as simple as this: Double-blind, placebo-controlled studies are absolutely not required in drugs that are have extremely large effect sizes. In these cases, open-label trials, compassionate use programs, and post-marketing surveillance can be employed to continue gathering data on safety and efficacy without denying any patients access to critical treatments. And, in the end, the data you gather is just as useful, if not more useful.
- mlyle 3y ago3/32 vs 9/15 is -probably- a pretty big effect size, but I am not sure how you know that a priori without trying it on a couple dozen people first and comparing. They didn’t require huge n for approval, but it took a long time to just try it on a minimal number of patients.