Post-hoc statistical adjustments and single-arm designs cannot substitute for randomized clinical evidence.
Faced with trial design deviations, regulatory agencies reject attempts to substantiate efficacy through post-unblinding modifications or uncontrolled single-arm studies.
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The FDA's skepticism over Replimune's candidate stems from the inherent difficulties of proving efficacy using a single-arm study design without a randomized control.
A dispute over statistical plans and post-hoc analyses led the advisory committee to reject the trial data as too fragile to support approval.
The FDA challenged Capricor's efficacy claims because they relied on post-hoc analytical adjustments rather than robust, pre-specified randomized clinical evidence.
Regulatory panelists sided against the drug because of deviations from the original statistical analysis plan and fragile clinical results.
The regulatory standoff stems from the developer altering its statistical plan post-hoc to show success after the original parameters failed.
A single-arm program benchmarked against a shifting external database and buttressed by after-the-fact re-analyses cannot substitute for randomized proof, and the market punished the gap.
The advisory committee rejected the therapy because its efficacy claims relied on post-hoc statistical adjustments rather than prespecified randomized trial endpoints.
Regulators rejected the drug candidate because the trial failed to show effectiveness under the original prespecified plan before post-unblinding adjustments were made.