Measuring Responsivity in Individuals with Profound Intellectual Disability

Project Summary/Abstract Profound intellectual disability (PID) is characterized by limited conceptual skills, limited understanding of symbolic communication, and total dependence on others for self-care. At present, there are no valid measures of cognition that are sensitive to the range of functioning in individuals with PID; however, these tools are critical

in informing clinical care, person-centered educational/day planning, and clinical trial readiness for this group. This proposal includes a career development plan to support the PI in acquiring the knowledge and skills necessary to develop patient-focused clinical outcome assessments (COAs) for individuals with PID. This

expertise is critical to fill the substantial gap in cognitive and functional measures available for this underserved and understudied group. Training will focus on 1) qualitative and 2) quantitative aspects of measure development, 3) an understanding of regulatory control as it pertains to the implementation of COAs in clinical

trials as well as ethical considerations in conducting research in this group, 4) skills in partnering with patient advocates to promote diverse representation in research and 5) skills in effective academic writing and leadership. Preliminary data support that a foundational aspect of cognition, responsivity, is a meaningful

indicator of functioning for individuals with PID as reported by their caregivers. The proposed research study will utilize both qualitative and quantitative methods to develop a novel performance measure (PerfO) and observer- report measure (ObsRO) of responsivity intended for individuals with PID through the following aims: 1) Refine

the concept of “responsivity” within the context of PID. A concept elicitation approach will be used to develop a reflective model of responsivity for PID through expert panels and caregiver interviews. 2) Develop a novel PerfO and ObsRO of responsivity for individuals with profound ID. Co-design methods including an expert workgroup,

caregiver interviews, and pilot testing will be used to modify measures of responsivity developed for individuals with acquired brain injuries in this pursuit. The primary goal of development is to create feasible, valid, and useful measures of responsivity with the potential to capture meaningful change in individuals with PID. Secondary

goals include maximizing appropriateness for a broad age range and ease of future adaptation (e.g., for specific genetic etiologies, remote administration). 3) Describe the psychometric properties of the novel PerfO and ObsRO. Psychometric aspects including feasibility, central tendency and dispersion, reliability, validity, and

provider perceptions of utility will be examined for both measures. An exploratory sub-study will utilize qualitative and quantitative methods to examine caregiver perceptions of meaningful change on both measures. Through the achievement of these study aims and related training activities, the PI will obtain the mentorship and training

needed to become an independent clinician-scientist in measure development and validation for individuals with PID.