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
Center for Drug Evaluation and Research (CDER) Director Janet Woodcock ultimately made the accelerated approval decision, finding that the small amount of dystrophin produced by the drug was reasonably likely to predict clinical benefit. Her decision was backed by FDA Commissioner Robert Califf, who said he found no evidence that she was unduly influenced by the patient community or other external pressures
