Times are changing at the FDA and other federal agencies. In recent years, legislation and regulation has pushed for a greater patient voice in decisions. The FDA's job wasn't simply to look at the science, as it has done for many years. They have "to look to what is meaningful benefit on the part of the patients," Furlong said. "What do the patients value?"
In this case, the parents were saw real promise in this drug, despite the results from the flawed study. They also noted that the drug appears to be safe, so there wasn't much risk. And the research did show that boys on the drug were producing a small amount of a potentially helpful protein called dystrophin, which could be a sign that the drug provides some benefit.
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