The effects of releasing early results from ongoing clinical trials

Steffen Ventz, Sergio Bacallado, Rifaquat Rahman, Sara Tolaney, Jonathan D. Schoenfeld, Brian M. Alexander, Lorenzo Trippa

Research output: Contribution to journalArticlepeer-review

5 Scopus citations

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.

Original languageEnglish (US)
Article number801
JournalNature communications
Volume12
Issue number1
DOIs
StatePublished - Dec 1 2021
Externally publishedYes

Bibliographical note

Publisher Copyright:
© 2021, The Author(s).

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