KHN Morning Briefing

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$145K-A-Year Drug For ALS Approved By FDA

It's only the second drug available to treat the disease.

The New York Times: A Second Drug Is Approved To Treat A.L.S.
A new drug for amyotrophic lateral sclerosis, or Lou Gehrig’s disease, was approved on Friday by the Food and Drug Administration. The drug, called Radicava or edaravone, slowed the progression of the degenerative disease in a six-month study in Japan. It must be given by intravenous infusion and will cost $145,524 a year, according to its manufacturer, MT Pharma America, a subsidiary of the Japanese company Mitsubishi Tanabe Pharma Corporation. (Grady, 5/5)

In other pharmaceutical news —

Stat: Follow-Up Studies Vary For Drugs Approved On Limited Evidence
As the Food and Drug Administration increasingly relies on just one key clinical trial or surrogate markers for approving certain medicines, a new study suggests that sufficient follow-up evidence demonstrating these drugs actually work varies substantially. The study examined 117 novel drugs that were approved by the FDA between 2005 and 2012 for 123 indications on the basis of a single pivotal trial, which is intended to provide evidence that a drug works; pivotal trials that used surrogate markers to gauge whether a drug can work; or both approaches. (Silverman, 5/5)

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