PCORI follows the definition set by the Rare Disease Act of 2002, a rare disease affects fewer than 200,000 people in the United States. There are over 7,000 known rare diseases afflicting an estimated 25-30 million Americans. For this PFA, we will consider as further justification, if needed, that a disease is rare if it is listed as rare in compendia maintained by NIH’s Office of Rare Disease Research, those listed in the European registry known as Orphanet, or ones that have been deemed as rare by the Food and Drug Administration.
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