Sarepta, eteplirsen: anecdote, data, surrogate outcomes, and the FDA

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 […]

Source: badscience - September 30, 2016 Category: Science Authors: Ben Goldacre Tags: bad science Source Type: blogs