The Clinical Trials Mosaic: Toward a Range of Clinical Trials Designs to Optimize Evidence-Based Treatment

Abstract

Objective: Dichotomizing clinical trials designs into nomothetic (e.g., randomized clinical trials or RCTs) versus idiographic (e.g., N-of-1 or case studies) precludes use of an array of hybrid designs and potential research questions between these extremes. This paper describes unique clinical evidence that can be garnered using idiographic clinical trials (ICTs) to complement RCT data. Proposed and illustrated herein is that innovative combinations of design features from RCTs and ICTs could provide clinicians with far more comprehensive information for testing treatments, conducting pragmatic trials, and making evidence-based clinical decisions.

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.