Addressing Health Outcomes for Individuals With Intellectual and Developmental Disabilities (IDD) and/or Rare Diseases (RD) PCORI Funding Announcement -- Cycle 3 2026

Addressing Health Outcomes for Individuals With Intellectual and Developmental Disabilities (IDD) and/or Rare Diseases (RD) PCORI Funding Announcement -- Cycle 3 2026 is sponsored by Patient-Centered Outcomes Research Institute (PCORI). This PCORI Funding Announcement (PFA) supports research focused on improving health outcomes for individuals with intellectual and developmental disabilities (IDD) and/or rare diseases (RD).

* **Applicant Town Hall** * **Letter of Intent (LOI) Deadline** * **LOI Status Notification** * **Application Deadline** This PCORI Funding Announcement (PFA) invites applications for high-impact, patient-centered comparative clinical effectiveness research (CER) projects that focus on people living with intellectual and developmental disabilities (IDD) and/or with rare disease(s) (RD).

## Research Initiative Highlights Developmental disabilities (DD) are chronic disabilities that originate at birth or in the developmental period and result in differing levels of support for physical, learning, language and/or behavioral skills. Intellectual disabilities, which fall under the umbrella term of DD, involve differences in cognitive function (i.e., reasoning, learning, problem solving) and adaptive behavior.

To define rare disease (RD), PCORI uses the Orphan Drug Act definition of an RD: a condition that affects fewer than 200,000 individuals in the United States. PCORI recognizes the variety of terminology used within the disability community, including both person-first (i.e., person with a disability) and identity-first (i.e., disabled person) language.

PCORI acknowledges that language preference varies, and both forms are acceptable in response to this funding opportunity. PCORI acknowledges that some IDD diagnoses are also considered an RD, and some RD diagnoses are also considered an IDD. **Applicants do not need to address both IDD and RD in this PFA.

** Responsive applications can focus on either IDD or RD populations but may also propose to include both populations if appropriate for the research question (Figure 1). There is no advantage in addressing both IDD and RD in response to this PFA.

**Figure 1: Image to clarify that applications responsive to this PFA can focus on either IDD or RD populations but may propose to include both populations if appropriate for the research question. ** This PFA will solicit applications proposing to address patient-centered CER questions that will fill important IDD and/or RD research gaps.

IDD and RD comprise a diverse group of conditions with a variety of lived experiences, even among people who have the same diagnosis. Both populations face challenges in accessing necessary care to address their health needs across the lifespan. Compared to people without IDD, more people with IDD have co-occurring physical and mental health conditions and unmet healthcare needs.

Several barriers, including health-related social and environmental barriers, are associated with a lower level of participation in school, work and play for people with IDD. People with many different types of RD and their caregivers often experience similar challenges such as finding reliable information and relevant resources, receiving an accurate diagnosis and gaining access to adequate and appropriate care.

PCORI is particularly interested in submissions that address the following 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.

**Better Health Outcomes by Improving Access to Patient-Centered Care** Care delivery for those with IDD and/or RD is influenced by many system-level factors that impact patients and caregivers. These challenges are not necessarily condition specific, allowing for a focus on cross-cutting issues and outcomes.

People with IDD and/or RD and their caregivers often face similar challenges such as finding relevant information and resources and accessing adequate and appropriate care, in many cases including the diagnosis itself for those with an RD. PCORI is interested in funding patient-centered CER comparing care delivery strategies to address these challenges and improve health outcomes for people with IDD and/or RD.

These strategies could include interventions to improve access to care, quality of care or the use of technology. PCORI is interested in interventions that improve patient-centered outcomes, including health-related quality of life, community participation and healthcare utilization. Secondary outcomes could include patient-centered burdens and economic outcomes (e.g., time away from work, travel time to visits).

* Comparing approaches to shorten the diagnostic odyssey for individuals with RD. * Comparing team-based models to provide individualized and person-centered care to improve health outcomes. 2.

**Health System Approaches to Improving Care Transitions** Transitions of care are critical for individuals with IDD and/or RD across the lifespan. For example, there are barriers to a successful transition from pediatric to adult-oriented healthcare. For individuals with IDD, these barriers include inadequate transition planning and the substantial drop-off in services offered once people reach age 21.

For individuals with IDD and/or RD, there is a shortage of clinicians who are able or willing to provide care as individuals age into adulthood. Moreover, across the lifespan, care transitions between specialists and generalist practitioners can be challenging. Successful transitions of care as individuals age and require new support or treatment approaches are also critical.

Although evidence-based interventions to support transitions of care are in use for other populations and conditions, research is needed to determine which care models are optimal and feasible using existing system resources for individuals with IDD and/or RD.

PCORI invites applications for CER comparing models of care or components of such models to support care transitions and the continuation of patient-centered primary and specialty health care for individuals with IDD and/or RD. Outcomes may include but are not limited to health outcomes, including physical and mental health and health-related quality of life, and process measures such as healthcare utilization and continuation of care.

* Comparing approaches for symptom management during the transition from pediatric to adult care. * Comparing care coordination models to ensure ongoing access to required support during transitions of care between different settings. 3.

**Pharmacologic and Nonpharmacologic Approaches to Improving Physical and Mental Health** People with IDD experience higher rates of co-occurring physical and/or mental health conditions compared to their peers. Social and environmental barriers, such as inaccessible resources and limited exposure to health promotion, contribute to these health differences.

Individuals with RD manage symptoms associated with their diagnosis, often with limited to no targeted treatment options. These barriers result in reduced health-related quality of life and lower participation in everyday activities. PCORI is interested in funding patient-centered CER to identify approaches to promote health and/or well-being among people with IDD and/or RD.

These could include pharmacological or nonpharmacological approaches (e.g., physical activity, nutrition, complementary/adjunctive care). Outcomes may include but are not limited to improved health-related quality of life, physical health and mental health. * Comparing approaches to improve symptom management and well-being.

* Comparing health promotion interventions focused on nutrition and physical activity to improve health outcomes. 4. **Addressing the Health and Well-Being of Caregivers for Individuals With Intellectual and Developmental Disabilities and Rare Diseases** Caregivers play a key role in supporting the health of individuals with IDD and/or RD.

They face challenges including finding information resources as well as navigating the required care to promote the desired health outcomes for the individual with IDD and/or RD whom they support.

Caregivers of individuals with IDD and/or RD may experience diminished quality of life, increased psychological distress and reduced participation in meaningful activities that impact their overall health with possible downstream effects on the individuals they care for.

PCORI is interested in studies that compare clinical interventions to support the health of caregivers as well as comparisons of multi-level care coordination approaches and other models of care delivery. PCORI recognizes that there is often an interdependence of patient and caregiver social, emotional, psychological and physical health.

Complex interventions that focus on one or more aspects of the caregiver/patient dyad’s health are welcome when appropriate and feasible. Primary outcomes may include but are not limited to caregiver self-efficacy and caregiver quality of life. Studies focused only on caregiver outcomes should provide justification for why collection of meaningful outcomes for care recipients is not feasible.

Secondary outcomes could include patient-centered burdens and economic outcomes (e.g., time away from work to navigate care, costs of nonmedical supplies required for care or sacrificing personal care for care of a loved one). * Comparing interventions to improve the mental health of caregivers.

* Comparing educational, support or training programs to improve patient and caregiver resilience, symptom and care management or well-being and/or health-related quality of life. Applications addressing cross-cutting issues that include more than one IDD or RD diagnosis are responsive. Studies focusing on a single condition are also responsive.

Applications do not need to focus on an IDD that is also considered an RD, or vice versa, to be responsive. Applicants are encouraged to propose individual- or cluster-randomized controlled trials; however, observational studies (including natural experiments) that are well-specified will also be considered.

Proposed studies should be powered with an overall sample size adequate for the precise estimation of hypothesized effect sizes and for assessing heterogeneity of treatment effect where appropriate.

Applicants are encouraged to pay special attention to issues of intervention implementation with the goal 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 with a focus on the development of research methods.

Applicants should propose well-justified and validated outcomes that are clinically meaningful and considered important to people with IDD and/or RD and that can be impacted by the proposed interventions within the study duration. Clinically meaningful outcomes (e.g., using patient-centered clinically important differences) are encouraged.

Applicants are also encouraged to include some assessment of patient-centered burdens and economic outcomes (PCBEOs) from the perspective of patients and/or their caregivers as secondary outcomes. 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.

Studies focused only on caregiver outcomes should provide justification for why collection of meaningful outcomes for care recipients is not feasible. Applicants may propose CER studies comparing systems and/or clinical approaches, which may include pharmacologic and/or nonpharmacologic interventions or system-level strategies delivered in hospital, clinical or community settings to improve patient-centered outcomes.

Interventions should be ready for CER, which generally requires documented evidence of efficacy or widespread use. Interventions focused on people with IDD and/or RD and/or their caregivers are acceptable. Interventions with established efficacy in the general population or interventions that have been adapted from the general population for those with IDD and/or RD may be acceptable.

Adaptations of efficacious interventions used in the general population or other situations may be responsive, but the level of adaptation must be minimal, clearly described and well justified. Applicants aiming to develop and/or compare untested new technologies will be considered nonresponsive.

In PCORI-funded research, patients and other healthcare partners are expected to meaningfully contribute their lived experience or professional expertise to ensure studies are patient-centered, relevant and useful for healthcare decision making. Applicants must address PCORI’s Foundational Expectations for Partnerships in Research — an evidence-based framework supported by tools and resources to guide effective engagement strategies.

View additional details on each of the Foundational Expectations. This funding announcement will accept applications with direct costs up to **$12 million** 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 cost maximum. For additional information, please see the full announcement upon posting. PCORI encourages all investigators interested in applying to use this inquiry form") to submit questions.

PCORI encourages all investigators interested in applying to please contact PCORI (pfa@pcori. org) to schedule a consultation. * Is Your CER Study Responsive to PCORI’s Funding Announcements?

* What You Need To Know To Apply * Foundational Expectations for Partnerships in Research * PCORI’s Portfolio of Funded Research * Population Insights Reports Powered by PCORnet®") * A Roadmap for Accelerating IDD Research Using PCORnet® Download Full Announcement Letter of Intent Deadline Letter of Intent Status Notification August 2027 (Subject to Change) ## Funds and Project Period ## To Apply for This Opportunity 1.

Read the full Addressing Health Outcomes for Individuals With Intellectual and Developmental Disabilities (IDD) and/or Rare Diseases (RD) PFAand FAQs 2. Use the Application Readiness Checklist 3. Submit a Letter of Intent (LOI) through PCORI Connect").

* All new and returning applicants will use the new PCORI Connect submission portal, which has built-in templates (for most PFAs) and resources for a streamlined experience. * PCORI Connect Help Center 4. Submit an application, upon invitation only, through PCORI Connect.

* Application submission requires LOI acceptance and invitation by PCORI to submit a proposal. Additional Applicant Resources