Incidence of Advanced Symptomatic Disease as Primary Endpoint in Screening and Prevention Trials

Nancy A. Obuchowski, Paul Schoenhagen, Michael T. Modic, Moulay Meziane, G. Thomas Budd

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.