More than half of novel drug approvals were based on single studies in 2022

The US Food and Drug Administration (FDA) based its approvals for most novel drugs on single studies during 2022, according to findings from a cross-sectional analysis published as a research letter in JAMA Network Open.

Among 37 novel drugs approved in 2022, 65% of approvals were based on single study evidence, while just four drugs had three or more studies that were considered as part of the approval process, the study found.

Robert M. Kaplan, PhD, of the Clinical Excellence Research Center at Stanford University, and his colleagues used the FDA Novel Drug Approvals website to identify new drugs approvals in 2022 that had not been previously approved for any indication and compiled all information available through ClinicalTrials.gov for each medication. Along with the number of trials associated with each approved drug, they also looked at whether trials were randomized, who sponsored the trials, whether they were completed, and if the results were posted.

“We are concerned about the trend toward approving products on the basis of a single study,” Kaplan told Regulatory Focus. “Further, we worry about whether the single study cited in the approval might be an outlier or a false positive result. We have great respect for the FDA but hope they will take these issues into consideration in the approval process.”

Among the 413 studies associated with novel drug approvals in 2022, 55% were randomized and 21% used single-group designs, according to the findings. More than three-quarters of studies (79%) were sponsored by industry, 2% were a collaboration between the National Institutes of Health (NIH) and a non-industry source, and less than 1% were sponsored by NIH. Additionally, the researchers reported that results were posted for 103 studies (25%).

The researchers wrote that the findings “highlight a trend toward less rigorous standards for novel drug approvals,” which has also been reported in other studies. (RELATED: Study tracks sharp drop in FDA approvals based on 2 pivotal trials, Regulatory Focus 24 April 2020)

Kaplan said the FDA has been pushing for a more streamlined approval process over the last two decades and that the 21^st Century Cures Act, enacted in 2017, codified greater flexibility in how to apply evidence-based standards for drug approval. For comparison, in 2016, 20% of novel drugs were approved based on single-study evidence and 55% of novel drug were approved based on evidence from three or more studies, the researchers reported.

“New drug approvals in 2022 appeared to be based on fewer studies than before the passage of the 21^st Century Cures Act,” the researchers wrote. “We believe consumers deserve access to the full range of evidence for the drugs they are considering, not just from the selected studies released to the public.”

The researchers reported having no conflicts of interest.

JAMA Network Open

More than half of novel drug approvals were based on single studies in 2022

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