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Incidence of advanced symptomatic disease as primary endpoint in screening and prevention trials
Nancy A Obuchowski1, Paul Schoenhagen, Michael T Modic
1Department of Quantitative Health Sciences, Wb4, The Cleveland Clinic, 9500 Euclid Ave., Cleveland, OH 44195, USA.
Objective:
Randomized clinical trials (RCTs) using disease-specific mortality as the primary outcome are the gold standard for evaluating the efficacy of screening tests. These trials require thousands of subjects and 8-10 years of follow-up; often the imaging technology has changed by the end of the trial.
Conclusion:
We propose the incidence of symptomatic disease as an alternative to disease-specific mortality. This endpoint is sensitive to the benefit of screening, correlates with patients' quality of life and societal costs, and can dramatically reduce the sample size and follow-up requirements of RCTs.
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