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PCORI

Patient-Centered Outcomes Research Institute

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    About Us

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    Fact Sheets: Learn More About PCORI

    Download fact sheets about out work, the research we fund, and our programs and initiatives.

    Find It Fast

    Browse through an alphabetical list of frequently accessed and searched terms for information and resources.

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    Research & Results

    • Explore Our Portfolio
    • Research Fundamentals
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    • Putting Evidence to Work
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    Evidence Updates from PCORI-Funded Studies

    These updates capture highlights of findings from systematic reviews and our funded research studies.

    Journal Articles About Our Funded Research

    Browse through a collection of journal publications that provides insights into PCORI-funded work.

    Explore Our Portfolio of Funded Projects

    Find out about projects based on the health conditions they focus on, the state they are in, and if they have results.

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    Topics

    • Addressing Disparities
    • Arthritis
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    • Men's Health
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    • Multiple Chronic Conditions
    • Multiple Sclerosis
    • Obesity
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    • Rare Diseases
    • Rural Health
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    • Women's Health

    Featured Topic: Women's Health

    Learn more about the projects we support on conditions that specifically or more often affect women.

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    Engagement

    • The Value of Engagement
    • Engagement in Health Research Literature Explorer
    • Influencing the Culture of Research
    • Engagement Awards
    • Engagement Resources
    • Engage with Us

    Engagement Tools and Resources for Research

    This searchable peer-to-peer repository includes resources that can inform future work in patient-centered outcomes research.

    Explore Engagement in Health Literature

    This tool enables searching for published articles about engagement in health research.

    Research Fundamentals: A New On-Demand Training

    It enables those new to health research or patient-centered research to learn more about the research process.

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    Funding Opportunities

    • What & Who We Fund
    • What You Need to Know to Apply
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    • Awardee Resources
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    PCORI Funding Opportunities

    View and learn about the newly opened funding announcements and the upcoming PFAs in 2021.

    Tips for Submitting a Responsive LOI

    Find out what PCORI looks for in a letter of intent (LOI) along with other helpful tips.

    PCORI Awardee Resources

    These resources can help awardees in complying with the terms and conditions of their contract.

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    Meetings & Events

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    PCORI 2021 and Beyond

    During this webinar, PCORI leaders shared ways to get involved in PCOR, improvements to our funding opportunities, and more.

    Confronting COVID-19: A Webinar Series

    Learn more about the series and access recordings and summary reports of all six sessions.

    2020 PCORI Annual Meeting

    Watch recordings of all sessions, and view titles and descriptions of the posters presented at the virtual meeting.

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  • Engagement
  • Engage with Us
  • PCORI's Advisory Panels
  • Advisory Panel on Rare Disease
  • PCORI Resources on Rare Disease

PCORI Resources on Rare Disease

Listings of PCORI-funded clinical effectiveness research projects, projects on coordination and engagement with the rare disease research community, and related resources.

Advisory Panel on Rare Disease

  • Members, meetings, and resources

Applicant Resources

  • Guidance for Rare Disease Organizations for Research Awards
  • FAQs for Rare Disease Applicants

Journal Article Highlight

An overview of the impact of rare disease characteristics on research methodology

Whicher D, Philbin S, Aronson N. An overview of the impact of rare disease characteristics on research methodology. Orphanet J Rare Dis. 2018 Jan 19;13(1):14. doi: 10.1186/s13023-017-0755-5. Review. PubMed PMID: 29351763.

View more articles on rare disease here.

Blogs, Feature Stories, Videos & Other Resources

Blogs

  • Changing the Landscape of Rare Disease Research (February 2018)
  • Informing Patients with a Rare Disease About Their Treatment Options (February 2017)
  • Big Data versus a Rare Disease (November 2016)
  • Patient-Centered Approaches to Sickle Cell Disease (September 2016)
  • How Patient-Centered Outcomes Research Helps Families with Rare Diseases (February 2016)
  • PCORI's Focus on Rare Diseases: Progress over the Past Year (February 2015)
  • Making Small Numbers Add Up: The Challenge of Studying Rare Diseases (February 2014)

PCORI Stories

  • Genetic Test Results: Putting Parents and Doctors on the Same Page
​

PCORI on Rare Disease: Watch all four videos

Videos (PCORI on Rare Disease)

  • PCORI Advisory Panel on Rare Disease
  • How I Became Involved in Research
  • Creating Collaboration for Rare Disease Research
  • What Can Big Data Do for Rare Disease Research?

PCORI Research Topic & Spotlight

  • Research Topic: Rare Disease
  • Research Spotlight on Rare Diseases (pdf)

Webinars & Other Events

  • Webinar: PCORI Funding Opportunities and Resources for Rare Disease Organizations (2017)
  • Webinar: PCORI Funding for Rare Diseases (2015)
  • Town Hall: Management of Care Transitions for Emerging Adults with Sickle Cell Disease (2016)
  • Rare Diseases Roundtable (2013)

Research Projects

Studies with a rare disease condition focus
  • A Comparison: High Intense Periodic vs. Every Week Therapy in Children with Cerebral Palsy (ACHIEVE)
  • A Personalized Decision Aid to Help Women with Lupus Nephritis from Racially and Ethnically Diverse Backgrounds Make Decisions about Taking Immune-Blocking Medicines
  • A Randomized Controlled Trial of Anterior Versus Posterior Entry Site for Cerebrospinal Fluid Shunt Insertion
  • A Stakeholder-Driven Comparative Effectiveness Study of Treatments to Prevent Coronary Artery Damage in Patients with Resistant Kawasaki Disease
  • Collaborative Assessment of Pediatric Transverse Myelitis: Understand, Reveal, Educate (CAPTURE) Study
  • Community Health Workers and Mobile Health for Emerging Adults Transitioning Sickle Cell Disease Care (COMETS Trial)
  • Comparative Effectiveness and Safety of Inhaled Corticosteroids and Antimicrobial Compounds for Non-CF Bronchiectasis
  • Comparative Effectiveness of a Decision Aid for Therapeutic Options in Sickle Cell Disease
  • Comparative Effectiveness of Peer Mentoring versus Structured Education-Based Transition Programming for the Management of Care Transitions in Emerging Adults with Sickle Cell Disease
  • Comparative Effectiveness of Therapy in Rare Diseases: Liver Transplantation vs. Conservative Management of Urea Cycle Disorders
  • Comparative Efficacy of Therapies for Eosinophilic Esophagitis
  • Comparing Patient Centered Outcomes in the Management of Pain between Emergency Departments and Dedicated Acute Care Facilities for Adults with Sickle Cell Disease
  • Comparing Strategies for Treating Juvenile Idiopathic Arthritis
  • Comparing Treatments for Children with Crohn's Disease
  • Decision Support for Parents Receiving Genetic Information about Child's Rare Disease
  • Developmental Trajectories of Impairments, Health, and Participation of Children with Cerebral Palsy
  • Engaging Parents of Children with Sickle Cell Anemia and their Providers in Shared-Decision Making for Hydroxyurea
  • Engaging Patients and Caregivers Managing Rare Diseases to Improve the Methods of Clinical Guideline Development
  • Enhancing Genomic Laboratory Reports to Enhance Communication and Empower Patients
  • Evaluating Whether Oxygen Treatment Helps People with Pulmonary Fibrosis Breathe Easier
  • Home or Away from Home: Comparing Clinician and Patient/Family-Centered Outcomes Relevant to the Care of Pediatric Acute Myeloid Leukemia during Periods of Neutropenia
  • Improving Trial Design and Analysis for Studying Treatments for Rare Diseases
  • Intervention and Outcomes in Duarte Galactosemia
  • Patient-Centered Comprehensive Medication Adherence Management System to Improve Effectiveness of Disease Modifying Therapy with Hydroxyurea in Patients with Sickle Cell Disease
  • Posterior Fossa Decompression with or without Duraplasty for Chiari Type I Malformation with Syringomyelia
  • Prospective Multicenter Observational Cohort Study Of Comparative Effectiveness of Disease-Modifying Treatments for Myasthenia Gravis (MG)
  • Realization of a Standard of Care for Rare Diseases Using Patient-Engaged Phenotyping
  • Relapsed Childhood Neuroblastoma as a Model for Parental End-of-Life Decision-Making
  • Taking Charge of Systemic Sclerosis: Improving Patient Outcomes Through Self-Management
  • The Impact of Self-Management with Probiotics on Urinary Symptoms and the Urine Microbiome in Individuals with Spinal Cord Injury and Spina Bifida
  • Tools and Information to Guide Choice of Therapies in Older & Medically Infirm Patients with AML
  • Treatment Alternatives in Adult Rare Disease; Assessment of Options in Idiopathic Subglottic Stenosis

Explore other research projects in PCORI's portfolio

Engagement in Research Projects

Awarded through the Engagement Awards Program

  • 2019 NEC Symposium
  • Adult Experiences in Glut1 Deficiency
  • Annual Cholangiocarcinoma Foundation Conference
  • Arterial Turtuosity Syndrome PCOR/CER Summit Series
  • Building Momentum Through Congenital Muscular Dystrophy Stakeholder Participation
  • Building Research Partnerships to Improve Sexual and Reproductive Health for Women with CF
  • Cholangiocarcinoma Foundation 6th Annual Conference
  • Cholangiocarcinoma Foundation: 2018 Annual Conference
  • Cholangiocarcinoma Foundation Annual Conference
  • Conference Wilms Tumor in WAGR Syndrome
  • Developing and Testing "Best Practices" in Training for Academic/Community Research Partnerships
  • Discovering What Matters Most
  • Engaging Patients in Outcomes Research in TANGO2-related disorder
  • Expanding Engagement in Dravet Syndrome and Developing a Best Practice Paradigm for Other Rare Disease Communities
  • Family & Science: Bidirectional Translation of Knowledge and Need in Sex Chromosome Aneuploidy
  • Health and Research Ambassador (HARA) PCOR/CER Training Program
  • IBEMC Stakeholder Network
  • Inborn Errors Of Metabolism Of Uncertain Clinical Consequence -- What Patients Really Want To Know
  • It's Time to Talk! It's Time to Listen!
  • KAND Scientific and Family Engagement Conference
  • National Alopecia Areata Foundation Patient Support Conference
  • Necrotizing Enterocolitis Symposium: A Transdisciplinary Approach to Improved NEC Outcomes
  • Non-Cystic Fibrosis (CF) Bronchiectasis Patient Network and Research Roadmap
  • Nontuberculous Mycobacteria Research Consortium Stakeholder Engagement and Planning Meeting
  • Novel Stakeholder Engagement for Nemaline Myopathy, Patient Centered Research
  • Pain in Cerebral Palsy
  • Patient-Centered CMT/HNPP Pain Summit
  • Patient-Empowering Outreach Seminar Series
  • Patients Engagement to Reduce Zika Disease and Improve Maternal Child Health Blueprint for Research
  • Patient and Stakeholder Alliance for SATB2-Associated Syndrome
  • Patient Advisory Committee for Clinical Trials (PACCT+)
  • PCOR Conferences: Charcot-Marie-Tooth (CMT) & Inherited Peripheral Neuropathies (IPN)
  • PCOR Training: A Program for Rare Disease Patient Advocates
  • PDSA ITP Patient Conference: Developing Patient-Centered Outcomes Research Framework and Priorities
  • Phelan-McDermid Syndrome Patient-Centered Outcomes Workshop
  • Real World Data Research Readiness: Engaging the Prader-Willi Syndrome Community
  • Stronger Together: Nonprofit Groups Propelling Patient-Driven Research of Rare Neuromuscular Diseases
  • Tennessee Sickle Cell Disease Network
  • The National Sickle Cell Advocate Network (NSCAN)
  • The STRETCH Project: To Build Capacity Advancing Patient-centered Research in Ehlers-Danlos Syndrome
  • The SWF Patient Engagement Conference
  • Translation to Transform
  • Turner Resource Network Symposium: Turner Science in the 21st Century

Awarded through the Pipeline to Proposal Program

  • Advancing Reproductive Health Wellness Between Patients with Cystic Fibrosis, Their Caregivers, Healthcare Providers, and Researchers
  • Bridging Rare Disease Patients and Data through Novel Research Partnerships
  • Building a Patient-Centered Care Network for MRKH
  • COMPASS: COMmunity Participation to Advance the Sickle cell Story
  • Community Engagement to Address Tuberculosis Infection in Persons Experiencing Homelessness
  • Community Engagement to Improve Prevention and Treatment of Congenital Cytomegalovirus Infection
  • Developing Comparative Effectiveness Research in Dravet Syndrome
  • Establishing a Patient-Centered Research Community for Cystic Fibrosis
  • Optimizing Palliative Care for Patients with Brain Cancer & Their Families
  • Establishing the Ehlers-Danlos Co-morbidity Coalition
  • MoPAC - Moving from Pediatric to Adult Care
  • Vascular Ehlers-Danlos Syndrome Collaborative
  • We'll Take the Village: Engaging the Community to Better Health

Explore other engagement in research projects in PCORI's portfolio.


Posted: April 27, 2017; Updated: August 9, 2019

About Us

  • Our Programs
  • Governance
  • Financials and Reports
  • Procurement Opportunities
  • Our Staff
  • Our Vision & Mission
  • Contact Us

Research & Results

  • Explore Our Portfolio
  • Research Fundamentals
  • Research Results Highlights
  • Putting Evidence to Work
  • Peer Review
  • Evidence Synthesis
  • About Our Research

Engagement

  • The Value of Engagement
  • Engagement in Health Research Literature Explorer
  • Influencing the Culture of Research
  • Engagement Awards
  • Engagement Resources
  • Engage with Us

Funding Opportunities

  • What & Who We Fund
  • What You Need to Know to Apply
  • Applicant Training
  • Merit Review
  • Awardee Resources
  • Help Center

Meetings & Events

March 2
Cycle 1 2021 PCORnet Phase 3 Applicant Town Hall
March 8
Engagement Awards 2021 Special Cycle -- Applicant Office Hours (One)
March 15
Priorities on the Health Horizon: Informing PCORI's Strategic Plan (Webinar)

PCORI

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Patient-Centered Outcomes
Research Institute

1828 L Street, NW, Suite 900
Washington, DC 20036
Phone: (202) 827-7700 | Fax: (202) 355-9558
[email protected]

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