FDA Clarifies Criteria for Breakthrough Therapy Designation Amid Confusion

The FDA’s definition of “breakthrough” isn’t the same as Merriam-Webster’s.

A recent study published in the Journal of the American Medical Association (JAMA) uncovered that more than three-quarters of physicians erroneously believe the FDA’s breakthrough therapy designation is based on “high-quality evidence” showing that the drug is more effective than current treatments in the space.

In reality, the FDA’s main criteria for this designation include that the drug “treats a serious or lift-threatening condition” and has “preliminary clinical evidence to suggest it may be a substantial improvement over available therapies.”

Meanwhile, the majority of physicians mistakenly refer to the dictionary definition of “breakthrough,” which colloquially connotes an important, definitive advance.

“The misconceptions identified may lead physicians to overprescribe newly approved drugs—particularly breakthrough therapies—and inadequately communicate how well these drugs work to the patients who will use them,” the researchers behind the JAMA study concluded.

To clear up the confusion, Richard A. Moscicki, MD, deputy center director for science operations at the FDA’s Center for Drug Evaluation and Research (CDER), fielded questions about the breakthrough therapy designation in a 2-part Q&A on the FDA’s website.

“Many times, the ‘breakthrough’ designation is interpreted by the public…as though these drugs have already been conclusively shown to be a major game-changing advance,” Dr. Moscicki wrote. “What I’d like the public to understand is that the FDA tags drug candidates early in the development process with the ‘breakthrough’ designation because they have the potential to be true breakthroughs in the long run.”

Since the FDA’s breakthrough therapy program began in 2012, the CDER has received 342 requests for designations from pharmaceutical companies and granted them to 111 drugs, though only 41 of those drugs have reached the market.

It’s important for pharmacists to make sure patients are aware that a breakthrough therapy designation doesn’t necessarily mean the use of the drug will result in better outcomes.