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F.D.A. Approves Muscular Dystrophy Drug That Patients Lobbied For

The agency’s approval went against the recommendation of its experts. The main clinical trial of the drug was small, involving only 12 boys with the disease known as Duchenne muscular dystrophy, and did not have an adequate control group of boys who had the disease but did not take the drug. A group of independent experts convened by the agency this spring said there was not enough evidence that it was effective.


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The approval delighted the drug’s advocates and sent the share price of the drug’s maker, Sarepta Therapeutics, soaring. But it was taken as a deeply troubling sign among drug policy experts who believe the F.D.A. has been far too influenced by patient advocates and drug companies, and has allowed the delicate balance in drug approvals to tilt toward speedy decisions based on preliminary data and away from more conclusive evidence of effectiveness and safety.

“The agency has set a dangerous precedent,” said Diana Zuckerman, president of the National Center for Health Research in Washington. “To prove something works, you have to compare it to something else — a placebo or a treatment. They didn’t do that.”

Thing is, no one wants to take the placebo. Everyone wants to take a chance on the medicine. 

Are double-blind drug trials the most reliable?

I don't know for sure. I do see them a lot when I'm researching. 

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