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Rare Disease Research
- How does PCORI define rare disease?
- What type of rare disease research will PCORI fund?
- Does PCORI have a special interest in any rare disease subcategories (e.g., acquired adult diseases or inherited pediatric diseases)?
- Does PCORI offer funding opportunities to support a pre-existing rare disease registry, or the development of a rare disease registry?
- Does PCORI only fund projects that have a broad impact? Would PCORI consider research in a rare disease to have a broad impact?
- Will PCORI consider funding a rare disease study that includes a usual care group?
- What does PCORI consider sufficient evidence of efficacy for rare diseases?
- Would studies evaluating FDA safety profiles and affordability of orphan drugs be of interest to PCORI?
- Does PCORI fund projects regarding newborn screening or treatment options for rare diseases?
- What non-research funding opportunities are available for private or public-sector organizations with a focus on rare disease?
- Can projects include stakeholders from the International Rare Disease Community?
- Are there any PCORnet projects dedicated to rare diseases?
- Does PCORI have suggestions for how to facilitate studies given the smaller patient populations for rare diseases?
- Will PCORI consider rare disease CER studies where one of the interventions being compared is a drug being used off-label in clinical practices?
- Does PCORI have different criteria against which rare disease applications are evaluated?