Patient advocates played a very visible role in FDA's review of Sarepta Therapeutics Inc.'s Exondys 51 (eteplirsen) for Duchenne muscular dystrophy, but agency documents show just how difficult it can be for patient testimonials and caregiver surveys to sway FDA scientific staff when they believe the underlying clinical trial data fall short.
Agency reviewers could not reconcile what they saw as a disconnect between the clinical trial participants' experiences on eteplirsen versus...