Study: Half of confirmatory studies for accelerated approvals are late

More than half of confirmatory studies used to support accelerated approvals for drugs were not completed by their due date and manufacturers faced few consequences for such delays, according to recent research published in the JAMA Health Forum.

These findings were reported by Anjali Deshmukh, assistant professor at the Georgia State University College of Law, and colleagues from the Program on Regulation, Therapeutics, and Law at Harvard Medical School and Brigham and Women’s Hospital, in JAMA Health Forum last month.

The authors used FDA data to track drugs granted accelerated approval from 1 January 2012 to 31 July 2021. Trials were categorized as late if they were not conducted by the agreed upon deadline, or as of 15 September 2021.

The study examined 177 follow-up trials for 140 new indications granted accelerated approval and found that 54% missed their due date. Among the late trials, 8% were reported as delayed by the manufacturer before the due date, 27 were fulfilled one year after the deadline, three were withdrawn and 24 were completed a median of 1.8 years after the due date.

The study also found that confirmatory trials for non-oncologic indications (93%) were more likely to be late than those for oncologic indications, and trials for small molecule drugs (62%) were more likely to be late than those for biologic drugs.

“Accelerated approval expedites the marketing of medications based on uncertain efficacy evidence, but the process depends on timely follow-up trials” the authors wrote. “We found that more than half of so-called confirmatory studies were not completed in the agreed-on time.”

They added that “incomplete confirmatory clinical trials harm patients who are prescribed expensive drugs despite uncertain clinical benefits. However, drug manufactures face few consequences for delays.”

Yet the authors noted that things may soon be changing under the Consolidated Appropriations Act for 2023, which included accelerated approval reforms. The law grants FDA greater authority to ensure confirmatory trials are under way before approval, and mandates manufacturers submit progress reports every six months.

“It will be important to monitor whether these changes lead to fewer delays or whether additional authority is needed to assure that confirmatory trials are completed in a timely manner for the benefit of patients,” the authors state.

Similar issues related to late confirmatory trials were previously highlighted by the US Department of Health and Human Services Office of the Inspector General in a September 2021 report, which found that more than one-third of accelerated approval applications do not meet their original confirmatory trial dates. (RELATED: OIG raises concerns about accelerated approval pathway, Regulatory Focus 30 September).

JAMA Health Forum

Study: Half of confirmatory studies for accelerated approvals are late

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