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

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.

PCORI on Rare Disease: Watch all four videos

Research Projects

Explore other research projects in PCORI's portfolio

Engagement in Research Projects

Awarded through the Engagement Awards Program

Awarded through the Pipeline to Proposal Program

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


Posted: April 27, 2017; Updated: April 21, 2021

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