Validating Assessments of Real-World Outcome

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DESCRIPTION (provided by applicant): This collaborative study (the P.I. of whose companion application is Dr. Philip D. Harvey) aims to validate assessments of real-world (RW) functional outcomes in people with schizophrenia. Schizophrenia patients are often impaired in their social and occupational functioning, their self-care, and their ability to live independently. Not only do these impairments worsen the patient's quality of life, they also increase the societal cost of his or her illness. While these impairments are evident in most patients, surprisingly little attention has been paid to the validity of assessments of these outcomes. Since the current tool of choice in predicting functional disability in schizophrenia is neuropsychological (NP) testing, proposals to reduce disability have tended to focus on short-term treatment of cognitive impairments. However, such trials are not well suited either to induce or identify improvements in real-world functioning. Some researchers have proposed switching to measures of functional capacity instead, but recent evidence suggests that the correlations obtained between functional-capacity measures, NP test results, and real-world outcomes are largely a function of the real-world rating scale that is used. Thus systematic studies are needed to determine the optimal means for measuring real-world disability and its response to treatment. The present proposal has three distinct goals. It aims to determine: (1) the most accurate and practical scale for rating real-world outcomes in schizophrenia; (2) the optimal user of the scale; and (3) the factors that may impair the validity of self-reports of real-world outcomes on the part of schizophrenia patients. We adopt a construct-validation approach, using NP testing and functional-capacity measures as indicators of the construct and examining the convergence of various real-world outcome measures with these two indicators. Sophisticated data analysis will be performed to determine the convergences, and retesting will be performed to determine the test-retest stability of the candidate measures. The overall goal is to identify improved strategies for measuring real-world disability in schizophrenia and to inform the coming generation of treatment trials aimed at cognitive and functional enhancement through pharmacology and cognitive remediation in terms of the most suitable outcomes measures.