Co-chairs: Charles Bowden, MD; Nina R. Schooler, PhD

Comparative effectiveness studies emphasize comparisons of alternative interventions for which functional and tolerability measures are equal in emphasis to efficacy measures.  This symposium aims to define the key methodological elements, summarize contrasts from traditional efficacy trials, the clinical, public health and consumer values which they inform and discuss issues in implementation which have the potential to facilitate or impede achievement of these aims.  Effectiveness studies are conducted in populations typically seen in routine care settings.  Consequently, they do not exclude most patients with co-occurring conditions and therefore yield externally valid results which community clinicians can apply in patients who are similarly diverse in severity, complexity and ethnicity.

Comparative effectiveness research requires maintenance of a high proportion of patients for the full course of the project.  Comparative effectiveness studies should enroll patients with all of the illness features for which outcomes are reported.  For example, a study with outcome measures and recommendations for management of negative symtomatology in schizophrenia must enroll patients who have negative symptomatology.  Effectiveness trials must address tolerability and functional status with valid assessment measures, applied with the same rigor as is expected of symptom scales.  Separate presentation of results, particularly single point in time results with LOCF assumptions are inadequate for comparative effectiveness purposes.    Development of integral, multidimensional measures is complex, requiring research in its own right, and, likely, disease specific solutions. The symposium presents analytical methodologies which may be particularly useful in synthesizing and conveying results of such studies in ways that yield more effective new treatments and adequately manage multidimensional outcome variables.  

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