Fewer than half of cancer drugs granted accelerated approval demonstrate clinical benefit
By Elana Gotkine HealthDay Reporter
MONDAY, April 8, 2024 (HealthDay News) — Most cancer drugs granted accelerated approval do not demonstrate clinical benefit, according to a study published online April 7 in the Journal of the American Medical Association to coincide with the annual meeting of the American Association for Cancer Research, held from April 5 to 10 in San Diego.
Ian T.T. Liu, M.D., J.D., M.P.H., from the Brigham and Women’s Hospital and Harvard Medical School in Boston, and colleagues examined whether cancer drugs granted accelerated approval demonstrate clinical benefit in a cohort study using publicly available data to identify cancer drugs granted accelerated approval from 2013 to 2023.
A total of 129 cancer drug-indication pairs granted accelerated approval were included in the analysis. The researchers found that 63 percent of the 46 indications with more than five years of follow-up were converted to regular approval, and 22 and 15 percent were withdrawn and remained ongoing after a median of 6.3 years, respectively. In confirmatory trials, fewer than half (43 percent) demonstrated a clinical benefit. There was a decrease from 9.9 to 3.6 years in the time to withdrawal; the time to regular approval increased from 1.6 to 3.6 years. Of the 48 drug-indication pairs converted to regular approval, 40, 44, 10, 4, and 2 percent were converted based on overall survival, progression-free survival, response rate plus duration of response, and response rate and despite a negative confirmatory trial, respectively. Thirty-eight percent of indications were unchanged comparing accelerated and regular approval, while 63 percent had different indications.
“Patients should be clearly informed about the cancer drugs that use the accelerated approval pathway and do not end up showing benefits in patient-centered clinical outcomes,” the authors write.
One author disclosed ties to Gilead and serving as an expert witness. The trial was funded by Arnold Ventures.
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