One in Six Patients in Phase 2 Cancer Trials Receive Drug When Eventually Approved

WEDNESDAY, Feb. 26, 2025 (HealthDay News) — One in six patients in phase 2 clinical cancer trials receives treatments that are eventually approved, according to a study published online Feb. 25 in the Journal of the National Cancer Institute.

Charlotte Ouimet, from the McGill University School of Population and Global Health in Montreal, and colleagues assessed the probability that patients in phase 2 trials receive treatment that eventually advances to U.S. Food and Drug Administration approval for their condition. The analysis included a random sample from 400 trials identified in Clinicaltrials.gov for cancer phase 2 trials initiated between Nov. 1, 2012, and Nov. 1, 2015.

The researchers found that among 25,002 patient-participants in 608 specific treatment cohorts, 16.2 percent of patients received a treatment that advanced to FDA approval. When considering National Comprehensive Cancer Network off-label recommendations, the therapeutic proportion increased to 19.4 percent but decreased to 9.3 percent for FDA-approved regimens that are considered of substantial clinical benefit by the European Society for Medical Oncology-Magnitude of Clinical Benefit Scale. There were no statistical differences in proportions based on drug class, trial phase, or sponsorship.

“With respect to consent, many educational materials emphasize that trials present an opportunity to receive tomorrow’s treatments,” the authors write. “Our findings move beyond such nondescript statements — namely, that by entering a phase 2 trial, a patient has a one in six chance of receiving a treatment that will later be approved for their condition.”

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