Advances in Pediatric Rehabilitation Clinical Trials: Design, Methods, and Analysis

Rehabilitation and habilitation services across all ages have become more complex. As the future unfolds towards precision rehabilitation/habilitation pediatric researchers in these areas face unique challenges. The services we provide children and families can alter entire developmental trajectories and change family dynamics. This affords a responsibility that requires the field to learn and take advantage of many varied research constructs that can help us advance evidence-based and cost-effective practices. However, only in the last few decades has the field of pediatric rehabilitation/ habilitation strongly embraced the contribution of science as an aid to guide its future. As a result, we are still beginning to unfold where to begin. Almost by definition, the field deals with chronic conditions that evolve across the passage of time and development that change with and because of a complex set of inter-related variables. Thus, even understanding what and when a meaningful outcome occurs is often difficult. Researchers must consider the complexity of the problems faced considering mechanistic underpinnings, behavioral challenges, and societal contexts. Unique research designs and carefully constructed variables to operationalize interventions and measurements are a research necessity. Researchers must also give detailed thought to cost and benefits of all we do from a variety of perspectives (e.g. patients, care providers, and funders), elevating patient-centered outcomes.

This issue will seek to develop a thematic guide that focuses on the many highlights and great strides that pediatric rehabilitation field has made in the past few decades, particularly in the research arena; while simultaneously, creating an issue designed to educate current and future rehabilitation researchers.

Articles in this issue will focus on the specific challenges and opportunities facing pediatric rehabilitation/ habilitation. A choice of trial design, eligibility criteria, patient heterogeneity, selection of age-appropriate and sensitive measures, assessment of brain structure and function, finalization of treatment protocols, measuring fidelity of rehabilitation interventions, and conducting longitudinal data analyses that delineate the “active ingredients” in treatment and detect differential responses among subgroups, all of which are central issues to the field. Each require high levels of specialized expertise that stem from multiple disciplines, including persons with disabilities and other areas of lived experience.