The headline read, \u201cTreatments for cancers and Alzheimer\u2019s on the verge of a breakthrough.\u201d<\/p>\n
Really? Now, I\u2019ve written about this stuff long enough to know the word \u201cbreakthrough\u201d is a red flag. Anyone who hears the word or sees it in print should be skeptical. It\u2019s a dandy marketing tool.<\/p>\n
Sure enough, the first paragraph of the Guardian\u2019s story noted that new emerging treatments \u201cmay, in the next 10 to 20 years, transform the way people are treated.\u201d Ten or 20 years doesn\u2019t sound like a breakthrough is imminent. But the word sure gets people to pay attention.<\/p>\n
What exactly does \u201cbreakthrough\u201d mean? It sounds as if it signifies a sudden, important development.<\/p>\n
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 \u201cbreakthroughs\u201d and allow drug companies to market them based on preliminary evidence that they might result in important outcomes for patients. That\u2019s according to Larry Sasich, Ph. D., founder of Patient Drug News.<\/p>\n
Drugs with this \u201cbreakthrough\u201d designation can come to market in a shorter time, but approvals can be withdrawn if further trials don\u2019t show meaningful benefit to patients, Sasich said. All it means is that these drugs \u201care allowed to be sold using a lower standard of evidence that requires confirmation of a true benefit for full approval.\u201d<\/p>\n
The idea of an FDA designation for \u201cbreakthrough\u201d drugs has now spread to medical devices. The 21st Century Cures Act, loosens regulatory standards for clinical trials for devices passed the U.S. House last year, and a Senate version is moving toward passage. It contains controversial provisions for labeling.<\/p>\n
The FDA grants one-third of requests it receives for the breakthrough designation for drugs. \u201cIt\u2019s a low bar,\u201d said Dr. Sanket Dhruva, a Yale cardiologist, who studies medical evidence.<\/p>\n
\u201cIf a drug is truly meaningful, we\u2019ll know that through the evidence and won\u2019t need to call it a breakthrough,\u201d he said.<\/p>\n
It\u2019s becoming clear that neither doctors nor patients understand what \u201cbreakthrough\u201d 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 required.<\/p>\n
Sasich added that the JAMA study also showed that doctors don\u2019t have a good understanding of the drug\u2019s FDA-approved professional product label. This, he says, can result in physicians overestimating the benefit and prescribing inappropriately.<\/p>\n
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 \u201cbreakthrough.\u201d The other was described as meeting the FDA criteria for being a breakthrough drug. Ninety-two percent chose the one that was labeled \u201cbreakthrough.\u201d Marketing leads patients to be overly optimistic about what a drug can do.<\/p>\n
If doctors don\u2019t know whether a drug or device is a breakthrough supported by evidence, it\u2019s up to patients to find out on their own.<\/p>\n