The FDA is now fast-tracking new drug approvals — why that’s so worrying

The Food and Drug Administration is now regularly approving new drugs after just one or two clinical trials — a significant departure from the more rigorous vetting process the agency was previously known for, newly published research reveals.

Furthermore, the authors say, there’s now less information available to the public about the results of all trials.

Of the 37 drugs approved by the FDA last year, 24 (about 65%) were approved based on just one study, according to a paper published in JAMA Network Open.

Only four of those 37 drugs, or about 11%, reported three or more studies before approval.

“Everything is supposed to be transparent with this FDA process,” Veronica Irvin, an associate professor at Oregon State University’s College of Health, said in a news release.

“The purpose of ClinicalTrials.gov was to have a way for the non-scientific community to access the trials and their results, in a way that people can understand,” she said.

Researchers agree that it’s critical to minimize delays in making drugs for serious diseases like cancer available to patients, but these findings highlight a need for greater transparency around how drugs get approved.

For example, many drugs have been tested in multiple clinical trials, but drug companies are only required to share the results from two of those trials, leaving questions about why they selected those two trials for submission — and what happened during the other trials.

“We’re not saying that cancer drugs need a lot more studies; just that they should show all the results or trials that are completed,” said Irvin, a co-author of the paper.

“It doesn’t mean they wouldn’t get approved, but it means we’d have a more complete picture,” she added.

Another piece of new research, published in Health Affairs Scholar, found that of the 46 new drugs approved in 2017, 19 of them (41%) were approved based on a single study — though the drugmakers conducted an average of 2.2 studies per drug, including 165 studies for the popular weight-loss drug Ozempic.

The ease with which novel drugs are approved is in part the result of the 21st Century Cures Act, passed in 2016 to speed the approval of new medicines so patients could gain access to life-saving treatments.

As part of that law, the FDA relaxed some standards to allow treatments for priority health conditions such as cancer to be approved with fewer supporting studies, and with less emphasis on randomized clinical trials.

But in the years following the passage of the 21st Century Cures Act, the FDA has faced a firestorm of criticism over the approval process for some new drugs.

Last year, a congressional investigation found that the FDA’s “atypical collaboration” with drugmaker Biogen to approve Aduhelm, a high-priced Alzheimer’s drug, was “rife with irregularities.”

And a drug for treating agitation in people with Alzheimer’s disease, brexpiprazole (Rexulti), was granted fast-track approval by the FDA in May.

But a damning report in BMJ revealed that brexpiprazole failed to provide a clinically meaningful benefit even as it increased the risk of death among patients.

Nonetheless, the FDA fast-tracked its approval, and the drug’s manufacturers (Otsuka and Lundbeck) predict $1 billion in annual sales. 

“We are very disappointed that the FDA approved this additional label indication for brexpiprazole on such weak data,” Nina Zeldes, health researcher at the consumer advocacy organisation Public Citizen, told BMJ.

“The FDA has set a dangerous precedent about the data it may require for future drug approvals for this vulnerable patient group,” Zeldes added.

When the FDA states that it has reviewed drugmakers’ two submitted studies, Irvin noted, the public is missing information about how many other studies were conducted, what those studies showed and why only those specific two studies were chosen for evaluation.

“We want doctors and patients to be able to see the whole picture,” she said.