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FDA on Mylotarg: Too fast or too slow?

The U.S. Food and Drug Administration issued a release today about a cancer drug being withdrawn from the market that underscores the agency's troubles with its accelerated approval process for certain drugs: The FDA can speed up the steps to get them onto the market, but can it speed up the steps to pull them off?

Here's the background on the latest situation: In 2000, the agency approved the use of the drug Mylotarg for certain cases of acute myeloid leukemia under the accelerated approval process. That essentially means that it based its decision on findings that were promising, but not definitive. As part of the deal, it required the company (then Wyeth, which was since acquired by Pfizer) to conduct a follow-up study.

Now, some 10 years later, it appears that in at least one study the drug caused an increase in the numbers of deaths of people who used it, which, according to a story by Bloomberg, is the result of deadly liver complication. Because of this, the FDA requested that Pfizer, the drug's maker, take it off the market, and the company voluntarily did so. This is the first case of a drug that's gone through the accelerated approval process being withdrawn because of FDA action.

The results sheds light on some glitches in the system. The fatalities came to light because of the follow-up study that was launched in 2004 - four years after the FDA approved the drug. An item on the Huffington Post reports that company then halted the study some five years later, in August 2009, because of the negative results. What's more, evidence that the drug wasn't as promising as had been thought were aired in 2007 when European regulators refused to approve the drug, saying that studies had not shown it saved lives.

So why did it take 10 years for such a decision to be made? Some might say that the accelerated approval process is a bad idea, but an FDA official quoted in the Bloomberg story suggests that the problem is in the lack of adequate follow-up studies and that four years should not pass between accelerated approval and the launch of these studies:

The delay underscores the need for more oversight of follow-up studies with drugs that win accelerated approval, said Robert Kane, acting deputy director for safety in the FDA’s Division of Hematology Products.

“Accelerated approval still has a place in the approval process but it’s also more important that we learn over the years that the whole development plan be set up well in advance,” Kane said today in a phone interview. “The trials should be planned and if possible underway when the sponsor submits the application.”

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