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When There Is a Large Effect

The approval of about 10 percent of devices and drugs by the US Food and Drug Administration isn't based on data from randomized clinical trials, especially when initial studies exhibited a large treatment effect, according to a new analysis appearing in JAMA Network Open.

In their paper, researchers led by the City of Hope's Benjamin Djulbegovic note that randomized clinical trials are not always practical due to difficulties in enrolling patients or other logistical limitations and that regulatory bodies have been exploring other means of evaluating treatments.

For their review and meta-analysis, Djulbegovic and his colleagues sifted through 606 drug applications with Breakthrough Therapy designations and 71 medical device applications for the Humanitarian Device Exemption. Of these, 10 percent were approved without a randomized clinical trial, they note. In those cases, the researchers found that the effect sizes tended to be larger — about 2.5-fold higher — than the effect sizes for drugs or devices approved based on randomized clinical trial data.

"We concluded that the FDA has not requested subsequent RCTs when a treatment has displayed a larger magnitude of estimated treatment effects," they write in their paper.

This, the researchers note, is similar to what they found in a previous analysis of European Medicines Agency approvals.