When FDA officials can’t agree, who gets the last say? For individuals afflicted with Duchenne muscular dystrophy (DMD), this question has had life-altering consequences, and the answer is still unclear.
After years of drug trials and months of internal bickering at the FDA, one woman–Dr. Janet Woodcock, director of the Center for Drug Evaluation and Research–overturned an FDA panel decision rejecting approval for a Duchenne treatment. An exciting new medicine, eteplirsen by Sarepta Therapeutics, was thus approved, and Duchenne patients and their families celebrated accordingly. Dr. Ronald Farkas, who opposed the drug, left the FDA amidst apparent agency strife. But a larger question remains: Why should one bureaucrat, unelected to any office, have the authority to determine the fates of individuals suffering from a chronic, deadly illness?
