This is What Happens When the FDA Approves Cancer Drugs Too Quickly

August 17, 2017
By Alex Keown, BioSpace.com Breaking News Staff

WASHINGTON – Two reports published in the Journal of the American Medical Association are highly critical of the U.S. Food and Drug Administration's (FDA) accelerated approval practices for new drugs and medical devices.

Some of the new drugs approved under these accelerated measures have failed to show “clear evidence” of safety and effectiveness, Reuters first reported.

From the AMA report, between 2009 and 2013 the FDA granted accelerated approval to 22 drugs for 24 indications, 19 of which involve cancer treatment. A total of 30 preapproval studies supported the 24 indications, the report said.

“Postapproval requirements were completed and demonstrated efficacy in 10 of 24 indications (42 percent) on the basis of trials that evaluated surrogate measures. Among the 14 of 24 indications (58 percent) that had not yet completed all requirements, at least 1 of the confirmatory studies failed to demonstrate clinical benefit in 2 (8 percent), were terminated in 2 (8 percent), and were delayed by more than 1 year in 3 (13 percent),” the report said.

Writing in Endpoints News, John Carroll said that the FDA has “waived old rules on overall survival” as a way to ramp up new drug therapies for patients. As part of the deal, the FDA was willing to accept preliminary safety data, with the promise of completed trial safety data at a later date, in order to approve treatments. It was a move that patient groups have cheered, Carroll noted.

However, critics of the FDA’s practices have conducted several studies that suggest those patient groups should hold off on their cheering. According to reports, the groups examining the FDA approval data took a look at 22 drugs approved under the accelerated process. Of those drugs, the reports show that “only half of the 38 confirmatory studies required by the FDA had been completed,” Carroll wrote. Of those completed, Carroll added most used trial endpoints that “had strayed far from the old gold standard on randomized survival results.” Carroll’s point was similar to one made by Huseyin Naci of the London School of Economics and Political Science, an author of one of the AMA reports. Naci told Reuters that the data he and other researchers found suggests the drugs in the accelerated approval program may not pan out in the long-term for patients.

“Our findings suggest that expediency in drug development and approval can be successful but that drugs approved via the shorter route to market are rarely subject to tests even in the post-approval period that use established and clinically meaningful outcomes,” Naci told Reuters.

The report in the AMA journal suggests that the post-approval data would not be sufficient for regulatory approval under the traditional FDA standards.

A second report shows similar results when it comes to high-risk medical devices. The report indicates that a large percentage of clinical trials for these medical devices given accelerated approval relied on substandard endpoints, Medscape reported. Rita F. Redberg from the University of California at San Francisco, told Reuters that many of the trials for these high-risk devices were randomized or blinded. That means, she said. “it is not known if the device was better than an alternative treatment (or better than no treatment), and whether any purported beneficial effect was actually due to the well-documented placebo effect of procedures and devices.”

Despite these highly critical reports, little is likely to change at the FDA. Carroll noted that new FDA Commissioner is a proponent of accelerated approval. As the FDA shapes up under the new commissioner, Carroll said the accelerated approval standards set by the regulatory agency’s oncology group will be the benchmark for the rest of the agency.