An even more egregious error happens when median/mean is relied on. It's also worth noting that one of the few shorts was explicit: they didn't believe an MoA existed. Ouch.
This time around, Celldex had comparison data that strongly suggested its lead candidate, glemba, could provide a survival benefit over chemo for patients with a difficult-to-treat form of breast cancer. Unfortunately, the data came from a subgroup contained within an otherwise failed trial. In retrospect, it looks like several outliers resulted in a lot of wasted resources.
[my bold]
Outliers, on one side of the median/mean, will skew you dead. Not to mention: subgroup post-hoc "analysis" is the Very Big Red Flag biostats. On often wonders why a legit math stat would get within a barge pole's length of such companies. But they do. I guess the money's not bad.
It is all too common for small bios to go all in, given that they have, in general, small pipelines. If I had a nickel for every one of these that generated traffic like, "but there's one guy still alive after 5 years taking WhizzBang250", I'd be sipping tequila on a Caribbean island. ImmunoOncology is one of the bigger deals these days, but there're even spontaneous remissions in cancer and always have been.
With PhARMA constantly braying that it costs $X billion to get "a drug to market" so drugs have to be really expensive to pay for all that, it's worth pondering whether the moolah is funnelled into R&D or mgt. pockets?
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