The Duchenne’s treatment made by Sarepta (eteplirsen) has been in the news this week, as a troubling example of the FDA lowering its bar for approval of new medicines. The FDA expert advisory panel decided not to approve this treatment, because the evidence for any benefit is weak; but there was extensive lobbying from well-organised patients and, eventually, the FDA overturned the opinion of its own panel. There have been calls for paper retractions, and so on.
This is not the first time we’ve seen peculiar activity around the treatment. Read the rest of this entry »