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The Clinical Trials Mosaic: Toward a Range of Clinical Trials Designs to Optimize Evidence-Based Treatment2017-11-01, 20:51:39
Method: Mixed model trajectory analysis and unified structural equations modeling were coupled with multiple baseline designs in (a) a true N-of-1 pilot study to improve severe autism-related communication deficits and (b) a small sample preliminary study of two complimentary interventions to relieve wheelchair discomfort.
Results: Evidence supported certain mechanisms of treatment outcomes and ruled out others. Effect sizes included mean phase differences (i.e., effectiveness), trajectory slopes, and differences in path coefficients between study phases.
Conclusions: ICTs can be analyzed with equivalent rigor as, and generate effect sizes comparable to, RCTs for the purpose of developing hybrid designs to augment RCTs for pilot testing innovative treatment, efficacy research on rare diseases or other small populations, quantifying within-person processes, and conducting clinical trials in many situations when RCTs are not feasible.
Keywords: Clinical trials, statistical analysis, trajectories, structural equations modeling, idiographic, nomothetic, treatment mechanisms, N-of-1, personalized medicine, pragmatic trials