Survival benefit associated with participation in clinical trials of anticancer drugs

About The Study: Many studies suggest a survival benefit for cancer trial participants. However, these benefits were not detected in studies using designs addressing important sources of bias and confounding. Pooled results of high-quality studies are not consistent with a beneficial effect of trial participation on its own.

 Quote from corresponding author Jonathan Kimmelman, Ph.D.:

“Many physicians, policymakers, patient advocates, and research sponsors believe patients have better outcomes when they participate in trials, even if they are in the comparator arm. Educational materials for patients often encourage patients to consider the better care they might receive in trials. As a member of various review committees, I hear this said all the time. Yet evidence of such a ‘participation effect’ is lacking.

“Many studies have looked at the ‘participation effect’ for patients with cancer. If you pool them altogether, it looks like cancer patients in trials do live longer than patients outside them. But if you restrict analysis to studies that control for various sources of confound (for example, the fact that trials tend to enroll younger and healthier patients) or bias (many studies of participation benefit are probably never published) those survival benefits disappear.

“Our findings provide reassurance that inability to enroll in a cancer trial doesn’t disadvantage a patient, at least in terms of survival. Our findings can help patients (and physicians) focus their consent discussions on the most relevant and evidence-based benefits of trial participation: the prospects of advancing the care of future patients.”

Contact information for Jonathan Kimmelman, Ph.D.: email jonathan.kimmelman@mcgill.ca.

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(doi:10.1001/jama.2024.6281)

Editor’s Note: Please see the article for additional information, including other authors, author contributions and affiliations, conflict of interest and financial disclosures, and funding and support.

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Media advisory: This study is being released to coincide with presentation at the Society for Clinical Trials Annual Meeting.

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