Incidence of advanced symptomatic disease as primary endpoint in screening and prevention trials.
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.


CONCLUSIONS
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.
Obuchowski
Nancy A
NA
Department of Quantitative Health Sciences, Wb4, The Cleveland Clinic, 9500 Euclid Ave., Cleveland, OH 44195, USA.
Schoenhagen
Paul
P
Modic
Michael T
MT
Meziane
Moulay
M
Budd
G Thomas
GT
D002908:Chronic Disease / Q000401:mortality*; D015991:Epidemiologic Measurements; D006801:Humans; D015994:Incidence; D008403:Mass Screening / Q000379:methods / Q000706:statistics & numerical data*; D016016:Proportional Hazards Models; D016032:Randomized Controlled Trials as Topic / Q000379:methods*; D018570:Risk Assessment / Q000379:methods*; D012307:Risk Factors; D016019:Survival Analysis; D015996:Survival Rate