NORD Issues Statement on FDA Rejection of Citizen Petition

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FdaOn September 10, the Food and Drug Administration (FDA) notified the National Organization for Rare Disorders (NORD) that it was rejecting NORD’s Citizen Petition submitted in 2011 to request that a documented policy be established regarding the review of potential treatments for people with rare diseases.

NORD made the request in September 2011 in conjunction with a report submitted by an FDA committee to Congress in June 2011: “Improving the Prevention, Diagnosis and Treatment of Rare and Neglected Diseases.”  In the petition, NORD expressed concern that while the FDA had demonstrated a history of flexibility in the review of “orphan”—for rare diseases—products, noting that rare diseases present special challenges to researchers seeking to develop therapies, no policy then-existed specific to orphan drugs.

NORD President and CEO Peter L. Saltonstall said, “In the time since, FDA has repeatedly demonstrated its flexibility in approving new orphan drugs.  This flexibility was documented in a study and a follow up evaluation conducted by NORD Board member and attorney Frank Sasinowski and published in the DIA (Drug Information Association) Journal.  NORD feels comfortable that FDA is implementing a policy of flexibility in reviewing orphan drugs, while still assuring that the drugs meets all standards for safety and effectiveness.”