PLOS One recently published Changes in evidence for FDA Novel Drug Approvals: 2016-2024, a new study that examines trends in evidentiary standards for drug approvals before and after implementation of the 21st Century Cures Act of 2016, which was part of a broader regulatory and legislative effort to expedite the development and review of medical products.
Researchers constructed a database of all novel FDA approvals from January 1, 2016, through December 31, 2024, and each study linked to an approved drug was catalogued by study number, sponsorship, and timing of results reporting relative to completion.
The study found that between 2016 and 2024, there was a linear decline in the mean number of studies used per drug approval. In 2016, the average number of studies associated with a drug approval was 3.41, dropping to 3.11 in 2017, then falling to 1.5 in 2022, followed by a spike to 2.49 in 2023, though this was driven by a single outlier, Brenzavvy, which was approved based on nine clinical trials. However, by 2024 the average dropped to 1.39.
Furthermore, the number of drug approvals supported by three or more trials fell from 59.1% in 2016 to 11.8% in 2021 and then fell even lower to 5.6% in 2024. A majority of drug approvals are now based on a single clinical trial, making up 76.5% of drug approvals in 2021, and dropping slightly to 69.4% in 2024.
Additionally, the study found that industry funding consistently dominated study sponsorship. In 2016, industry-funded trials accounted for 44.6%, while non-industry sources accounted for 55.4%. Over time, the industry’s share steadily increased, reaching 55.2% in 2018 and climbing to 66.4% by 2019 and 80.4% by 2024.
Researchers state that although these trends may accelerate access to new therapies, it raises concerns about the strength of evidence for safety and effectiveness, and could point to the need for policy adjustments.
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