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anecdotal evidence isn't useless-- but it is "n = 1"

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In Reply to: RE: Is anecdotal evidence really all that useless? posted by Tweaker456 on April 18, 2017 at 14:27:01

-- and must be considered in light of that.

In my early "pro" days, I worked for a biotech company with an interesting anticancer immunotoxin "platform". Their original Phase 1 studies (which are used to assess the safety of a new drug, and not its efficacy) were conducted not in healthy volunteers but in (relapsed) cancer patients who had failed conventional therapies for their specific cancer.

Several of these very sick patients exhibited partial remissions after treatment with the drug, and there was at least one total remission.

The drug later failed to demonstrate efficacy in a standard Phase 2/3 protocol (cancer studies, at least in those days, were typically NOT placebo controlled, for rather obvious ethical reasons).

The PR and CR results in Phase 1 were anecdotal :-) The study wasn't designed to demonstrate, much less quantify efficacy, and the drug was later shown NOT to be efficacious in an appropriately "powered" study.

BUT, for the folks who exhibited CR or PR, it WAS indeed efficacious.

Anecdotal evidence.
Not "false evidence" -- just difficult to interpret.

The company in question later developed a different platform for immunotoxins and subsequently did develop effective cancer drugs.

all the best,
mrh

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