WASHINGTON — Food and Drug Administration Commissioner Scott Gottlieb defended his agency’s “breakthrough therapy” program, which speeds review of drugs that show signs of benefit early on, amid criticism from academics that the therapies aren’t actually breakthroughs at all.

A study published in late April in the Journal of Clinical Oncology showed that, on average, cancer drugs designated as “breakthrough” don’t help patients more than drugs that aren’t designated as “breakthrough.” And a study published earlier in April in the New England Journal of Medicine pointed out other examples of “breakthrough therapies” that really weren’t scientific breakthroughs.

Gottlieb said that there aren’t “any misgivings” around the program, and that it’s been “very important.”


“I’ve seen some of what you perceive as criticism,” Gottlieb said in response to a question about the program at the annual meeting of the Reagan-Udall Foundation for the FDA, a nonprofit created by Congress to support the activity of the agency. “I perceive it as healthy dialogue.”

The agency does sometimes pick a therapy as a “breakthrough” and then later withdraws the designation if concerns come up in the review process.

Most recently, at the end of April, the company Synthetic Biologics withdrew the application for breakthrough status for one of its drugs, which is supposed to prevent infection from the bacteria Clostridium difficile, or C. diff., after the FDA raised safety concerns.

FierceBiotech reported that over a dozen drugs have either lost their breakthrough therapy designation, or the sponsoring companies have withdrawn their bids, since the program started in 2014.

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