Call for Improving Health Outcomes for Individuals with IDD and Rare Diseases (US)

The Patient-Centered Outcomes Research Institute (PCORI) is accepting applications for high-impact Comparative Clinical Effectiveness Research (CER) focused on individuals living with Intellectual and Developmental Disabilities (IDD) and Rare Diseases (RD). The funding opportunity provides up to $60 million in total funding, with individual projects eligible to request up to $12 million over a project period of up to five years.

About the Program

The Patient-Centered Outcomes Research Institute (PCORI) has launched this funding opportunity to support patient-centered Comparative Clinical Effectiveness Research (CER) that improves health outcomes for individuals living with Intellectual and Developmental Disabilities (IDD) and Rare Diseases (RD).

The program aims to generate high-quality evidence that helps patients, caregivers, clinicians, healthcare systems, and policymakers make informed healthcare decisions. Rather than developing new treatments or technologies, the funding supports studies that compare existing interventions to determine which approaches work best for different patient populations.

PCORI encourages research that addresses significant evidence gaps, improves healthcare delivery, enhances care coordination, and promotes better health outcomes throughout the lifespan for individuals affected by IDD and rare diseases.

Funding Summary

• Funding Organization: Patient-Centered Outcomes Research Institute (PCORI)
• Program Focus: Comparative Clinical Effectiveness Research (CER)
• Total Funding Available: Up to $60 million
• Maximum Funding Per Project: Up to $12 million
• Maximum Project Duration: Up to 5 years

Program Objectives

The funding opportunity aims to:

• Improve patient-centered health outcomes for individuals with IDD and rare diseases.
• Generate evidence comparing existing healthcare interventions.
• Improve access to patient-centered healthcare services.
• Strengthen healthcare systems that support lifelong care.
• Improve physical and mental health outcomes.
• Enhance care coordination across different stages of life.
• Support caregivers through evidence-based healthcare approaches.
• Reduce healthcare disparities experienced by individuals with IDD and rare diseases.

Priority Research Areas

PCORI is particularly interested in projects that address one or more of the following priorities.

Improving Access to Patient-Centered Care

Research may evaluate strategies that improve access to high-quality, patient-centered healthcare services for individuals living with IDD and rare diseases.

Care Transitions Across the Lifespan

Projects may compare approaches that improve healthcare transitions from childhood through adulthood and across different healthcare settings.

Pharmacologic and Nonpharmacologic Interventions

Applicants may compare medications and non-drug interventions designed to improve physical health, mental health, functioning, and overall well-being.

Caregiver Health and Well-Being

Research may examine interventions that improve the health, well-being, and support systems for caregivers of individuals with IDD and rare diseases.

Research Focus

The funding opportunity supports research that addresses important healthcare challenges experienced by people with IDD and rare diseases.

Individuals with intellectual and developmental disabilities often experience:

• Multiple physical health conditions.
• Co-occurring mental health conditions.
• Unmet healthcare needs.
• Barriers to healthcare access.
• Reduced participation in education, employment, and community life.

Individuals living with rare diseases frequently face:

• Delayed diagnosis.
• Limited access to specialized care.
• Lack of reliable health information.
• Challenges accessing appropriate treatments.
• Significant caregiver burden.

Eligible Study Designs

PCORI encourages several research designs, including:

• Individual randomized controlled trials (RCTs).
• Cluster-randomized controlled trials.
• Well-designed observational studies.
• Natural experiments.
• Hybrid effectiveness-implementation studies.

Eligible Activities

Funding may support:

• Comparative clinical effectiveness research.
• Patient-centered health outcomes research.
• Clinical intervention comparisons.
• Healthcare delivery research.
• Care coordination studies.
• Stakeholder engagement.
• Data collection and analysis.
• Outcome evaluation.
• Dissemination of research findings.

Who Can Apply?

Eligible applicants include:

• Universities and academic institutions.
• Research organizations.
• Healthcare organizations.
• Nonprofit organizations.
• Other organizations capable of conducting comparative clinical effectiveness research consistent with PCORI’s funding requirements.

Applications may focus on:

• A single intellectual or developmental disability.
• A single rare disease.
• Cross-cutting issues involving multiple IDD or rare disease diagnoses.

Project Requirements

Applicants should ensure that proposed research:

• Focuses on comparative clinical effectiveness research.
• Addresses one or more priority research areas.
• Evaluates interventions already supported by evidence or widely used in practice.
• Demonstrates meaningful patient-centered outcomes.
• Includes appropriate stakeholder engagement.
• Uses scientifically rigorous study designs.
• Supports future implementation of findings where appropriate.

What is Not Supported?

This funding opportunity is not intended to support:

• Development of new drugs or therapies.
• Creation of new medical technologies.
• Development of new research methodologies.
• Early-stage efficacy testing of interventions without sufficient existing evidence.

Why This Program Matters

People living with intellectual and developmental disabilities and rare diseases often experience significant healthcare disparities and limited evidence to guide clinical decision-making. By funding comparative clinical effectiveness research, PCORI seeks to identify the most effective healthcare interventions and improve healthcare delivery for these underserved populations.

The research generated through this program can help clinicians, patients, caregivers, and healthcare systems make better-informed treatment decisions while improving quality of care and long-term health outcomes.

How to Apply

Applicants should follow these steps:

1. Review the funding announcement and research priorities.
2. Identify an important healthcare question affecting individuals with IDD or rare diseases.
3. Develop a comparative clinical effectiveness research proposal.
4. Select an appropriate study design.
5. Engage patients, caregivers, clinicians, and relevant stakeholders.
6. Prepare a detailed research methodology and evaluation plan.
7. Develop a realistic project budget and timeline.
8. Submit the complete application through the PCORI application system according to the published guidelines.

Tips for Applicants

• Focus on patient-centered healthcare questions.
• Compare interventions already supported by existing evidence.
• Clearly explain the expected health impact.
• Include meaningful patient and caregiver engagement.
• Demonstrate strong scientific methodology.
• Consider hybrid effectiveness-implementation approaches where appropriate.
• Ensure outcomes are relevant to patients and healthcare providers.

Common Mistakes to Avoid

• Proposing development of new treatments rather than comparing existing interventions.
• Selecting research questions outside the priority areas.
• Weak stakeholder engagement plans.
• Insufficient justification for the proposed intervention.
• Poorly defined patient-centered outcomes.
• Inadequate research methodology.
• Lack of implementation planning where appropriate.

Frequently Asked Questions (FAQs)

What is the purpose of this funding opportunity?

The program supports comparative clinical effectiveness research that improves healthcare outcomes for individuals living with intellectual and developmental disabilities and rare diseases.

How much funding is available?

PCORI has made up to $60 million available under this funding opportunity.

What is the maximum funding for an individual project?

Individual projects may request up to $12 million.

How long can funded projects last?

Projects may be funded for up to five years.

What research designs are encouraged?

PCORI encourages randomized controlled trials, cluster-randomized trials, observational studies, natural experiments, and hybrid effectiveness-implementation studies.

Can projects focus on multiple rare diseases or disabilities?

Yes. Applications may address a single condition or cross-cutting issues involving multiple intellectual and developmental disabilities or rare diseases.

Does the program support development of new treatments?

No. The funding supports comparative evaluation of existing interventions rather than developing new drugs, technologies, or research methods.

Conclusion

The PCORI Comparative Clinical Effectiveness Research Funding Opportunity for Intellectual and Developmental Disabilities and Rare Diseases represents a major investment in patient-centered research aimed at improving healthcare for underserved populations. By supporting rigorous comparative studies of existing interventions, the program will generate evidence that strengthens clinical decision-making, improves patient outcomes, enhances caregiver support, and promotes more equitable healthcare across the lifespan.