The effects of releasing early results from ongoing clinical trials

Steffen Ventz (), Sergio Bacallado, Rifaquat Rahman, Sara Tolaney, Jonathan D. Schoenfeld, Brian M. Alexander and Lorenzo Trippa
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Steffen Ventz: Harvard T.H. Chan School of Public Health
Sergio Bacallado: University of Cambridge
Rifaquat Rahman: Harvard Medical School
Sara Tolaney: Harvard Medical School
Jonathan D. Schoenfeld: Harvard Medical School
Brian M. Alexander: Harvard Medical School
Lorenzo Trippa: Harvard T.H. Chan School of Public Health

Nature Communications, 2021, vol. 12, issue 1, 1-7

Abstract: Abstract Most trials do not release interim summaries on efficacy and toxicity of the experimental treatments being tested, with this information only released to the public after the trial has ended. While early release of clinical trial data to physicians and patients can inform enrollment decision making, it may also affect key operating characteristics of the trial, statistical validity and trial duration. We investigate the public release of early efficacy and toxicity results, during ongoing clinical studies, to better inform patients about their enrollment options. We use simulation models of phase II glioblastoma (GBM) clinical trials in which early efficacy and toxicity estimates are periodically released accordingly to a pre-specified protocol. Patients can use the reported interim efficacy and toxicity information, with the support of physicians, to decide which trial to enroll in. We describe potential effects on various operating characteristics, including the study duration, selection bias and power.

Date: 2021
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Persistent link: https://EconPapers.repec.org/RePEc:nat:natcom:v:12:y:2021:i:1:d:10.1038_s41467-021-21116-4

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DOI: 10.1038/s41467-021-21116-4

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