PCORI is interested in studies that examine both the benefits and harms of treatments for FDA-approved orphan drugs as they become more common for use in the target population. PCORI, however, is not interested in surveillance studies. Applicants must propose patient-centered comparative effectiveness research (CER) or descriptive studies that can form the basis for a CER question (e.g., describing treatments being used and outcomes). Outcomes must be those of interest to patients.
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