Comparative Effectiveness of Interventions Targeting Mental Health in Individuals with Intellectual and Developmental Disabilities -- Cycle 1 2022

The Patient-Centered Outcomes Research Institute (PCORI) seeks to fund randomized controlled trials (RCTs) or observational studies that focus on comparative clinical effectiveness of interventions targeting mental health in individuals with intellectual and developmental disabilities (IDD).

PCORI’s 2019 reauthorizing legislation specifies IDD as a research priority. There are approximately 7–8 million people in the United States with IDD, representing about 3 percent of the population. While people with IDD often have co-occurring conditions, mental health conditions in particular are common, with prevalence estimates ranging from 37 percent to 55 percent in those with IDD and up to 65 percent in adolescents with IDD. There is a high degree of heterogeneity for symptom presentation and unique needs across the lifespan for those with IDD and co-occurring mental health concerns. These factors contribute to care complexity. Broad evidence gaps exist around mental health treatment in individuals with IDD, and PCORI stakeholders have identified more research on interventions for those with both IDD and mental health conditions as a priority.

PCORI’s Targeted PFA will solicit applications that respond to the following question:

What is the comparative clinical effectiveness of evidence-based or commonly used approaches (e.g., specific pharmacologic and behavioral interventions) that address mental health in individuals with IDD?

Proposed studies should compare the effectiveness of pharmacological, behavioral, other nonpharmacological or combination interventions for mental health concerns administered via appropriate delivery modalities (e.g., telehealth, family based, group, or individual). Comparison of different healthcare delivery or systems-level models for this population is also acceptable. Interventions must be evidence-based and/or in widespread use and reproducible. Standard interventions with established efficacy (e.g., cognitive behavioral therapy, selective serotonin reuptake inhibitors for depression), those that target relevant symptom clusters or psychological domains (e.g., emotion dysregulation), or mental health interventions developed or adapted for those with IDD may be acceptable. Given the limited number of evidence-based interventions for specific IDD diagnoses, in some cases, usual care, when the components are precisely defined, may also be an acceptable comparator to evidence-based or commonly used interventions.

PCORI is interested in studies focused on individuals of all ages with mild, moderate, or severe impairment due to IDD. Interventions that target relevant mental health concerns more broadly (e.g., modular or transdiagnostic approaches) are acceptable. Underrepresented, historically excluded, and other underserved populations (e.g., individuals in rural settings) are of particular interest.

The following outcomes are of particular interest: symptom severity, functional impairment, quality of life, adverse events, health resource utilization, and relevant caregiver outcomes . Measures validated in the IDD population of interest are preferred, but alternative measures may also be acceptable if accompanied by a strong rationale for their use. Length of follow-up should be determined based on appropriateness for the intervention/design.

Please note: While the PCORI Cost Principles state that PCORI will not cover costs for study interventions that constitute the procedures, treatments, interventions, or other standard clinical care (“patient care”) that is being proposed for comparison in the research project (“patient care costs”), for the Cycle 1 2022 Intellectual and Developmental Disabilities tPFA, PCORI may consider funding support to cover patient care costs related to mental health for meritorious applications, when properly justified. This may include the intervention being studied as well as clinical personnel costs for those providing the care related to mental health. For additional information on the conditions for such coverage, see the Coverage of Intervention Costs section in the funding announcement. Applicants should submit a realistic budget and timeline reflecting the proposed study’s scope and requirements.

Reminder: The primary institution’s Administrative Official must review, authorize, and submit the full application by 5 pm ET on May 3, 2022.