PCORI Rare Disease Research: Portfolio Highlights and Future Directions
Engagement
Live Chat | Q&A | Polling
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Rare disease is a long-standing focus for PCORI. Recently, PCORI acknowledged rare disease as one of twelve Topic Themes of focus for future investment. The absence of approved treatments for many rare diseases makes comparative clinical effectiveness research of head-to-head treatment options a challenge. This session will highlight PCORI-funded rare disease comparative clinical effectiveness research studies (with one example leveraging PCORnet®, The National Patient-Centered Clinical Research Network) and engagement awards and will conclude with an equity-focused roundtable discussion of future directions for rare disease research and engagement.
Sophie Lanzkron, MD, MHS, The Johns Hopkins Hospital; Johns Hopkins School of Medicine
Jane Burns, MD, University of California, San Diego
Ruth Phillippi, Children's National
Anitha John, MD, PhD, Washington Adult Congenital Heart, Children's National; George Washington University School of Medicine
Download Resources
Rare Disease Care Continuum Framework:
Session Resource Links
Learn more about Dr. Jane Burns’ study on Kawasaki Disease:
Learn more about Dr. Sophie Lanzkron’s work on Sickle Cell Disease:
- Engagement award: Expanding Access to the Infusion Center Model for People Living with SCD
- Research award: Comparing Pain Management for Sickle Cell Disease Crises in Emergency Rooms and Infusion Centers
- Dissemination & Implementation award: Supporting Pain Management for Sickle Cell Disease Crisis at Infusion Centers
Learn more about Dr. Anitha John and Ruth Phillipi’s work on Congenital Heart Defects:
Learn more about PCORI’s Advisory Panel on Rare Disease:
Watch PCORI Board of Governors June 2023 meeting, where the Board approved PCORI investing up to $600 million in patient-centered comparative clinical effectiveness research (CER) through the development of funding opportunities for eight intersectional Topic Themes that address health issues spanning chronic conditions to emerging needs, including rare disease:
- Board of Governors Meeting: June 5 & 6, 2023
- PCORI to direct up to $600 million in health research funding spanning eight high-need areas impacting the nation's health
- Funding Applications Encouraged Around Topic Themes | PCORI
- PCORI Creates New Funding Opportunity: Topical PFA | PCORI
Other PCORI Resources on Rare Disease: