When the customer isn't always right

摘要

It's a heart-wrenching story, and one we hear time and again, People with an incurable illness are offered hope by a new drug, only to have it snatched away by uncaring bureaucracy, This time, parents of children with muscular dystrophy are battling the US medicines regulator, the Food and Drug Administration (FDA). Last week, families accompanied by young boys in wheelchairs told the agency's panel of advisers how an experimental drug called eteplirsen had slowed their child's disease. The manufacturer, Sarepta, claims the drug keeps children walking for longer. But the panel rejected the drug, saying that the small trial hadn't demonstrated its effectiveness.