Скачать или смотреть When the FDA's own experts clash: The shocking internal battle that decided a rare disease drug's...

Delving into the high-stakes world behind regulatory approvals reveals a fascinating internal mechanism at the FDA. When the statistical, clinical, and pharmacology teams cannot reach a consensus on pivotal study data, a powerful arbitration panel is called in to settle the matter. The 2016 fight over Sarepta's Exondys 51 provides a gripping case study: internal reviewers were deeply divided, weighing perceived weak efficacy against the urgent, unmet need for Duchenne's muscular dystrophy patients. This footage uncovers the internal friction that shaped the outcome for this crucial medicine.
#FDA #approval #process #Sarepta #Therapeutics #Exondys #51 #Duchennes #muscular #dystrophy #drug #regulation #clinical #data #interpretation #rare #disease #medicine #medical #ethics #debate