"There are several aspects of Baby K.J.’s story that define the FDA’s plausible mechanism pathway.
…
Fifth, there is an improvement in clinical outcomes or course. In conditions with progressive deterioration, consistent improvements will be viewed favorably by the FDA."
Here, this plausible-mechanism piece seems to describe N-of-1 scientific reasoning that proceeds without need for statistics to glean a faint signal out of masses of data.
Its vagueness and superficiality notwithstanding, the piece at least does not describe approvals of marginally-effective drugs such as I addressed in “ Where are the exceptional responders? ”.