FDA Grants Accelerated Approval for Sarepta Muscular Dystrophy Drug Eteplirsen

Tech Times, September 20, 2016. The U.S. Food and Drug Administration (FDA) has approved the first drug for treating patients with Duchenne muscular dystrophy. “The agency has set a dangerous precedent,” said National Center for Health Research president Diana Zuckerman. “To prove something works, you have to compare it to something else — a placebo or a treatment. They didn’t do that.”

Read More »

FDA Approves First Drug to Treat Rare Form of Muscular Dystrophy

Kaiser Health News, September 19, 2016. The Food and Drug Administration Monday approved the first drug to treat Duchenne muscular dystrophy, a rare and lethal muscle weakening disorder that affects about 15,000 Americans. Diana Zuckerman, president of the National Center for Health Research, said “it sets a dangerous precedent if the FDA is going to start approving drugs that aren’t compared to anything.”

Read More »

Feds to Crack Down on Those Who Fail to Report Clinical Trials

Politico Pro, September 16, 2016. Drug companies and research institutions will have to publicly report more clinical trial data, including results that show their products or experiments failed, under new policies rolled out Friday by HHS. But NCHR president criticizes their decision not to provide summaries of treatment results that patients can understand.

Read More »