BLINDED SUBJECTIVE RANKINGS AS A METHOD OF ASSESSING TREATMENT EFFECT- A LARGE-SAMPLE EXAMPLE FROM THE SYSTOLIC HYPERTENSION IN THE ELDERLY PROGRAM (SHEP)

Because many randomized clinical trials study more than one important outcome variable, evaluation of efficacy is often difficult and not co mpletely satisfactory. This paper considers the use of a procedure for endpoint determination described by Follmann et al., that allows rate rs to integrate subjectively all relevant information about an individ ual's clinical course into a single univariate assessment. To explore the method's feasibility, we tested the procedure with data from a com pleted clinical trial, the Systolic Hypertension in the Elderly Progra m (SHEP). We provided raters blinded to treatment assignment with card s that schematically represent the clinical trajectories of SHEP study participants. The raters independently ranked these trajectories. The method combined ranks across raters to determine a single rank for ea ch study participant; we used a rank procedure to test treatment effec t. The major findings were: (i) the raters showed a high level of conc ordance of rankings; (ii) tests of treatment effect were highly statis tically significant; (iii) three statistical methods were effective fo r implementing the ranking in the large study size case. These methods were use of (a) scoring rules; (b) incomplete block designs, and (c) categorical ranking. (C) 1997 by John Wiley & Sons, Ltd.