Limited Competition: Conducting Rare Disease Research Using PCORnet - Cycle 2 2020
This limited competition opportunity is open to all PCORnet Clinical Research Networks (CRNs)/Health Plan Research Networks (HPRNs) and their partner organizations to perform an observational research study that will answer one or more important research questions about the care of patients with rare diseases. A secondary goal is to enhance the capabilities for the conduct of multi-site rare disease research by creating partnerships, methods, tools, and linkages that will facilitate future comparative effectiveness studies for the rare disease community. Through this initiative, PCORI seeks to fund high-quality observational cohort studies of people with rare diseases or conditions. This initiative will require broad use of PCORnet resources for research, collaboration between a minimum of two CRNs/HPRNs, and the establishment of partnerships with rare disease patient organizations, with attention to representing the diversity of the population affected by the disease.
- Can an institution that is not a network partner within PCORnet serve as the prime contractor for this award?
- Can an institution that is not a network partner in PCORnet serve as a subcontracting site for the conduct of the study?
- How can individuals/organizations interested in rare disease research find network partners within PCORnet to collaborate with for this award?
- Can a Clinical Research Network, Health Plan Research Network, or PCORnet network partner submit more than one LOI?
- How does PCORI define Rare Disease?
- Will PCORI consider applications that address a rare disease population already included in the PCORI research portfolio?
- Does PCORI offer funding opportunities to support the development of a rare disease registry?
- What type of rare disease research will this PFA fund?
- What does PCORI consider sufficient evidence of efficacy for rare diseases?
- Will PCORI consider funding a rare disease study that includes a usual care group?
- Will PCORI consider studies that evaluate FDA safety profiles of orphan drugs?
- Will PCORI consider rare disease CER studies that include newborn screening or treatment options for rare diseases?
- Will PCORI consider rare disease CER studies that includes a drug being used off-label in clinical practices as a comparator?
- Can projects include stakeholders from the international rare disease community?
- Does the PI need to be one of the current PIs of the existing CRN or HPRN?