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FDA Rare Disease Patient Advocacy Day, 2012

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Event participants listen during an open panel discussion at the FDA's Rare Disease Patient Advocacy Day
Event participants listen during an open panel discussion at the Food and Drug Administration's Rare Disease Patient Advocacy Day, March 1, 2012. This event is used to engage and educate the rare disease community on regulatory processes related to rare diseases.
Photos by Maureen Stewart/FDA


Patient groups who represent rare disease are
enormously important to the central mission of
FDA in health care.


Of the thirty new molecular entities that the FDA shepherded through regulatory review and approval last year, more than one-third were indicated for rare diseases. By better understanding this special community of patients, the FDA has further become better prepared to capitalize on novel technologies and emerging trends in personalized medicine, for the general treatment of disease.

On March 1, 2012, the FDA opened its door, under the banner of FDA–Rare Disease: Patient Advocacy Day, coordinated by the Office of Orphan Drug Products, to bring patients and their families together with FDA officials on the White Oak campus. FDA officials, along with experts from the patient advocacy community, provided a full day of lectures and round-table discussions that pertained directly to the needs of patients with rare diseases. The day was devoted to learning—about each other, about the strides being made on diverse fronts in health care, and about the ongoing need for better treatments and interactions with members of the rare disease community.

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