The Role of Patient Participation in Drug Approvals: Lessons from the Accelerated Approval of Eteplirsen

Kyle T. Edwards


In September 2016, the Food and Drug Administration (FDA) controversially approved Exondys 51 (eteplirsen) for the treatment of Duchenne muscular dystrophy (DMD). Submitted under FDA’s Accelerated Approval pathway, eteplirsen is the first drug approved to treat DMD, a rare and fatal genetic disorder characterized by progressive loss of muscle function. Just five months earlier, FDA’s scientific advisory committee had voted against approval over the objections of a crowd of more than one thousand patients and advocates who had arrived to observe and provide testimony during the committee’s public hearing. Despite anecdotal accounts from patients and their caregivers of the drug’s benefits, the committee determined that Sarepta’s twelve-person trial had failed to present substantial evidence of the drug’s effectiveness. FDA’s internal scientific review team agreed. Yet, in the face of significant pressure from patients and their advocates, FDA reversed the negative recommendations of the advisory committee and its internal review team and approved the drug. This Article presents an account and critical appraisal of the role that patients and their advocates played in securing eteplirsen’s approval, using the case to understand FDA’s attempts to meet congressional demands over the past few decades for greater patient involvement and expanded expedited review programs. In light of the 21st Century Cures Act’s recent directive to FDA to significantly develop both its patient involvement and expedited approval programs, the lessons of the eteplirsen approval are particularly timely.