title: Incidence of advanced symptomatic disease as primary endpoint in screening and prevention trials.
abstract: 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.
authors.affiliation: Department of Quantitative Health Sciences, Wb4, The Cleveland Clinic, 9500 Euclid Ave., Cleveland, OH 44195, USA.
authors.forename: Nancy A
authors.initials: NA
authors.lastname: Obuchowski
authors.forename: Paul
authors.initials: P
authors.lastname: Schoenhagen
authors.forename: Michael T
authors.initials: MT
authors.lastname: Modic
authors.forename: Moulay
authors.initials: M
authors.lastname: Meziane
authors.forename: G Thomas
authors.initials: GT
authors.lastname: Budd
mesh_terms: 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