The Sarepta dilemma: Bioethics expert Arthur Caplan says it’s time to rethink how to regulate compassion
Arthur Caplan was never enthusiastic about the idea of an FDA approval for Exondys 51 (eteplirsen) for Duchenne muscular dystrophy. When it came through, the noted NYU bioethics expert saw it as a deviation from the FDA’s policies on how drugs should be studied and reviewed, focusing on safety and a clear signal of efficacy.
But there were a lot of things wrong with it.
“The trial was poor,” Caplan tells me, “and even with small numbers I think it could have been done better.” The vehement public lobbying by Duchenne families to get it across the finish line also didn’t convince him that the agency needed to make it available.
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