Intellectual and Developmental Disabilities Topical PCORI Funding Announcement -- Cycle 1 2024

Notice of Upcoming PCORI Funding Announcement

The Patient-Centered Outcomes Research Institute (PCORI) intends to issue a Topical PCORI Funding Announcement (PFA) on January 9, 2024, seeking to fund high-quality, patient-centered comparative clinical effectiveness research (CER) projects that focus on people with intellectual and developmental disabilities (IDD). This preannouncement provides potential applicants additional time to identify collaborators; obtain patient and stakeholder input on potential studies; and develop responsive, high-quality proposals.

Research Initiative Highlights

In the forthcoming PFA, PCORI recognizes the diversity of terminology used within the disability community, including both person-first (i.e., people with disabilities) and identity-first (i.e., disabled) language.

For this PFA, developmental disabilities (DD) are chronic disabilities that originate at birth or in the developmental period and cause impairment in physical, learning, language and/or behavioral areas. Intellectual disabilities, which fall under the umbrella term of DD, involve limitations to cognitive function (i.e., reasoning, learning, problem solving) and adaptive behavior. Data estimate that 17.8 percent of US children and adolescents and approximately 0.5 to 1.5 percent of US adults have an IDD diagnosis, impacting 7 to 8 million people living in the United States. Compared to people without IDD, a higher proportion of people with IDD have co-occurring physical and mental health conditions and unmet healthcare needs. Several barriers, including social determinants of health, are associated with a lower level of participation in school, work and play for people with IDD. Physical and mental disparities among those with IDD may be especially pronounced for people belonging to racial and ethnic groups.

To increase evidence that addresses meaningful decisional dilemmas faced by patients, clinicians, and members of the broader health and healthcare community when seeking and obtaining health care and to improve patient-centered outcomes, this opportunity aims to fund the comparison of interventions encountered by individuals of all ages with IDD with a special interest in the areas of sleep health, Down syndrome and improving care delivery with a focus on ableism. Comparisons can be clinical interventions or systems approaches and can include pharmacological or nonpharmacological interventions. Some examples include evaluating prevention, screening, diagnosis or treatment strategies; or evaluating dissemination, implementation or health communication strategies that aim to increase the uptake and sustainment of evidence-based practices, improve shared decision making, improve risk communication or improve communication among care teams.

Interventions with established efficacy in the general population or interventions that have been developed or adapted for those with IDD may be acceptable. Applications proposing novel interventions and/or aiming to develop new technologies (such as mobile apps) or decision support tools/aids will be considered nonresponsive. Adaptations of efficacious interventions used in the general population may be responsive, but the level of adaptation must be minimal, clearly described and justified a priori. Additionally, secondary outcomes may focus on improving quality of life and support for caregivers.  

PCORI is particularly interested in submissions that address the following three Special Areas of Emphasis (SAE). The purpose of identifying these SAEs is to encourage submissions to these areas, not to limit submissions to these topics. Applicants addressing one of the below SAEs should identify the area that is best associated with their research approach:  

1. Promoting Sleep Health: Individuals with IDD are at increased risk for insomnia and other indicators of poor sleep health, and sleep disturbance is associated with more severe daytime symptoms and worse quality of life. PCORI is interested in the comparative clinical effectiveness of pharmacologic and/or nonpharmacologic interventions that improve sleep health in individuals with IDD. Populations of interest should have IDD and have a diagnosed sleep disorder or poor sleep health of sufficient severity to be disruptive to daily life and/or associated with clinically significant distress.
2. Improving Health and Quality of Life in Down Syndrome: People with Down syndrome are more likely to experience co-occurring conditions like obesity and cardiovascular disease, although there is a lack of evidence on whether treatment approaches developed for the general population are also effective in individuals with Down syndrome. PCORI is interested in comparing interventions to improve the health and well-being of individuals with Down syndrome, particularly in approaches addressing mental health/well-being, cardiovascular disease, obesity, diabetes, obstructive sleep apnea and Alzheimer’s disease. Research can be conducted in clinical or community settings, and interventions can be at the individual and/or system level.  
3. Improving Care Delivery: Despite the availability of evidence-based treatments, there remain barriers to accessing care for people with IDD. PCORI is interested in comparisons of care delivery strategies to improve health outcomes for individuals with IDD. These strategies could include interventions focused on improving access to care, quality of care, or care navigation involving differences in settings of care, utilization of the IDD workforce, informal caregiving, or use of technology.  PCORI is especially interested in interventions that also address ableism, bias, stigma and/or discrimination.  The proposed care delivery strategies should directly target patient-centered outcomes such as quality of life through increased participation in school or work, healthcare utilization and/or patient-centered economic outcomes.

Applicants are strongly encouraged to propose individual or cluster randomized controlled trials; however, well-specified natural experiments and well-designed observational studies will also be considered. Proposed studies should examine diverse populations with an overall sample size that will allow precision in the estimation of hypothesized effect sizes and, as appropriate, analysis of heterogeneity of treatment effect. Applicants are encouraged to pay special attention to issues of intervention implementation with an aim of facilitating widespread uptake of findings after completion of the study by utilizing hybrid effectiveness-implementation approaches. However, strict implementation or dissemination studies will not be considered responsive, nor will studies focused on the development of research methods. Applicants should propose well-justified and validated outcomes that are clinically meaningful and considered important by patients, and that can be impacted by the proposed interventions within the study duration. 

Applicants should consider, as appropriate, the full range of IDD-related clinical and patient-centered outcomes data relevant to patients and other stakeholders. PCORI’s Principles for the Consideration of the Full Range of Outcomes Data in PCORI-Funded Research inform the expectations for applicants and the corresponding evaluation of applications submitted in response to this PFA. 

This funding announcement will accept applications with direct costs up to $12 million dollars and up to five years in duration. For this funding announcement, applicants may request coverage of patient care costs (including medical products, procedures and care services) for potential funding by PCORI. These costs are included as part of the overall direct costs.