Be cautious about ‘breakthrough’ drugs

A story in The Guardian, a British news outlet that now has a publishing arm in the U.S., grabbed my attention. The headline read, “Treatments for cancers and Alzheimer’s on the verge of a breakthrough.”

Really? Now, I’ve written about this stuff long enough to know the word “breakthrough” is a red flag. Anyone who hears the word or sees it in print should be skeptical.

There are very few genuine breakthroughs, but there’s lots of enthusiasm for the term on the part of the media, physicians, academic medical centers and others who use it to attract attention to a product or a service.

It’s a dandy marketing tool. Who wouldn’t want a “breakthrough” cure for cancer or Alzheimer’s disease?

Sure enough, the first paragraph of the Guardian’s story noted that new emerging treatments “may, in the next 10 to 20 years, transform the way people are treated.”

Ten or 20 years doesn’t exactly sound like a breakthrough is imminent. But the word sure gets people to read the story and pay attention when they see advertising for the product.

What exactly does the term “breakthrough” mean? It sounds as if it signifies a sudden, important and dramatic discovery or development.

The Food and Drug Administration has a specific meaning for the term.

A few years ago Congress gave the FDA, which regulates drugs, the authority to designate certain drugs as “breakthroughs” and allow drug companies to market them based on preliminary evidence that they might result in important outcomes for patients.

That’s according to Larry Sasich, founder of Patient Drug News, who once worked for the Washington consumer advocacy group Public Citizen.

Drugs with this “breakthrough” designation can come to market in a shorter time, but approvals can be withdrawn if further trials don’t show meaningful benefit to patients, Sasich told me.

All it means is that these drugs “are allowed to be sold using a lower standard of evidence that requires confirmation of a true benefit for full approval.”

The idea of an FDA designation for “breakthrough” drugs has now spread to breakthrough medical devices.

A bill, the 21st Century Cures Act, that loosens regulatory standards for clinical trials for devices passed the House of Representatives last year, and a Senate version is moving toward passage. It contains controversial provisions for labeling some devices as breakthroughs.

Dr. Sanket Dhruva, a Yale cardiologist who studies medical evidence, told me, “This is more a marketing sales pitch than something that’s truly a breakthrough that’s going to benefit patients.” The FDA grants one-third of requests it receives for the breakthrough designation for drugs. “It’s a low bar,” he says.

“If a drug is truly meaningful, we’ll know that through the evidence and won’t need to call it a breakthrough.”

Furthermore, it’s becoming clear that neither doctors nor patients really understand what breakthrough really means.

A study published in the journal JAMA reported that board-certified internists and specialists significantly overestimated what the FDA requires to be a breakthrough drug. They believed those drugs were backed by stronger evidence than what the FDA actually required.

Sasich added that the JAMA study also showed that doctors don’t have a good understanding of the drug’s FDA-approved professional product label. This, he says, can result in physicians overestimating the benefit and, thus, prescribing inappropriately.

Another study, published in JAMA Internal Medicine, asked a sample of Americans which of two drugs they would take for a potentially deadly condition. One was described as a “breakthrough.”

The other was described as meeting the FDA criteria for being a breakthrough drug. Ninety-two percent chose the one that was labeled “breakthrough.”

Marketing works, leading patients to be overly optimistic about what a drug can and cannot do.

If doctors don’t know whether a drug (and maybe also a medical device if the Cures Act passes) is really a breakthrough supported by scientific evidence, it’s up to patients to find out on their own.

One place to start is the DailyMed website,, offered as a free service of the National Library of Medicine, part of the National Institutes of Health. The website contains drug labeling information for more than 87,000 drugs.

You can find drug guidance, regulatory information, and warnings about medicines you may be prescribed — for example, that the FDA’s strongest warning, a black box warning, means there is reasonable evidence that a serious hazard is associated with the drug.

Remember, before you ask your doctor about some “breakthrough” drug you’ve seen advertised, it’s best to do your homework.

Trudy Lieberman, a journalist for more than 40 years, is a contributing editor to the Columbia Journalism Review, where she blogs about health care and retirement at She can be reached at [email protected]. This column was distributed by The Rural Health News Service.