Session III:
"Multi-site Treatment Trials"
Chair: Michael E. Thase, M.D.
Principles for Efficacy Trials: The Example of VA
Cooperative Study #430
Mark S. Bauer, M.D.
Bibliography
Abstract
Despite the availability of efficacious treatments for bipolar disorder, their
effectiveness in general clinical practice is greatly attenuated, resulting in
what has been called an "efficacy-effectiveness gap.” In designing VA
Cooperative Studies Program (CSP) Study #430 to address this gap, nine
principles for conducting an effectiveness (in contrast to an efficacy) study
were identified. These principles are presented and discussed, with specific
aspects of CSP #430 serving as illustrations of how they can be implemented in
an actual study.
CSP #430 is a 11-site, randomized controlled clinical trial of a multi-modal,
clinic-based intervention vs. usual VA care running from 1997-2003. The
intervention consists of (a) algorithm-driven somatotherapy, (b) standardized
patient education, and (c) easy access to a single primary mental health care
provider to maximize continuity-of-care.
The trial has enrolled 330 subjects, randomizing them either to the intervention
or to continued usual VA care. Subjects are assessed bimonthly for three years
across domains of disease outcome, functional outcome, and healthcare costs.
Minimal exclusion criteria maximize the external validity of the study. To
ensure internal validity, the intervention is highly specified in a series of
operations manuals for each of the 3 components. However, site and personnel
selection, and ongoing training and feedback using continuous quality
improvement (CQI) techniques, reflect the effectiveness emphasis of the trial.
Statistical techniques deal with drift of care in both the intervention arm and
secular changes in usual VA care.
CSP #430 is designed to have impact well beyond the VA, since it evaluates a
basic health care operational principle: that augmenting ambulatory access for
major mental illness will improve outcome and reduce overall treatment costs. If
results are positive, this study will provide reason to reconsider the
prevailing trend toward limitation of ambulatory services that is characteristic
of many managed care systems today.
STAR*D: An Effectiveness Trial for Major Depressive Disorder
A. John Rush, M.D.
Bibliography
Abstract
Not currently available.
Design and Promise of NIMH Multicenter Effectiveness Trials: A Step
Forward
Gary S. Sachs, M.D.
Bibliography
Abstract
The art of medical practice rests on a foundation of evolving scientific
evidence. Laboratory discoveries and clinical trial data provide evidence which
advance medical knowledge. Although randomized double trials often contribute
crucial knowledge about treatment efficacy, traditional efficacy designs are of
limited utility in providing guidance for large numbers of psychiatric patients
seeking treatment. The NIMH has launched a series of effectiveness trials which
aim to provide results generalizable to patients typically seen in clinical
practice. This presentation will focus on the design of the NIMH sponsored
Systematic Treatment Enhancement Program for Bipolar Disorder (STEP-BD).
STEP-BD provides infrastructure needed to conduct a variety of studies
addressing important clinical questions related to bipolar disorder. The
complexity of bipolar disorder necessitates enrollment of an large heterogeneous
sample including subjects differing in illness subtype, current episode, episode
frequency, comorbid conditions, and duration of illness. Determining outcome is
made difficult by the potential misleading result of any single measure. STEP-BD
uses a hybrid design in which all patients are enrolled into an open disease
management program. Subsets are offered randomized care only when their illness
brings them to the entry point of one of the STEP-BD randomized pathways which
address priority public health questions. STEP-BD trains the staff of
participating treatment centers to evaluate and manage bipolar patients in
accordance with a common set of model practice procedures. At study entry
subjects complete an extensive battery of formal assessments. The main study
outcomes are, however, derived from the standardized routine clinical assessment
made by the STEP-BD treating psychiatrist at each clinical visit. The design of
STEP-BD pathways and progress of the study will be discussed.
Panel Discussion
Discussants:
Barry D. Lebowitz, M.D.
Bibliography
Gregory Simon, M.D., M.P.H.
Bibliography
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