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National Organization for Rare Disorders to Collaborate with FDA on Pilot Patient Engagement Activity

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3.2 from 14 votes
Thursday, March 01, 2018

Washington, D.C — The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, has announced a collaboration with the Food and Drug Administration (FDA) on a pilot project that would help FDA medical reviewers better understand patient experiences with their rare diseases.

"We at the National Organization for Rare Disorders are excited about this effort to bring the rare disease patient perspective directly to FDA medical reviewers," said Peter L. Saltonstall, president and CEO of NORD. "These listening sessions will provide FDA review division staff with better insight into what is important to patients in managing their diseases and improving their quality of life.

"Our goal is to facilitate the patient's voice being heard more directly in the regulatory decision- making process, an invaluable opportunity for the rare disease community as a whole to advance medical research and new product approvals," Saltonstall said. "It is important for FDA to understand, from the patient perspective, disease burden, management of symptoms, daily impact on quality of life, and patients' risk tolerance. Patients and caregivers bring a pragmatic, realistic perspective about what they are willing to deal with in terms of potential risks and benefits for new therapies."

The FDA and NORD will work together in identifying patient community representatives and coordinating listening sessions where patients can share their experiences with FDA review division staff. At the request of review divisions, the listening sessions will be organized through the FDA's Patient Affairs Staff (PAS) in collaboration with NORD. PAS will work with the review division to draft questions for the patients, and work with NORD to facilitate the discussion between the requesting review division and the patient community.

The FDA has instituted a number of programs to bring the patient perspective to the product review process, consistent with legislation enacted by Congress and advanced by NORD.

There are 7,000 rare diseases that each affect 200,000 or fewer Americans. The vast majority still have no treatment.

About the National Organization for Rare Disorders (NORD)®
The National Organization for Rare Disorders (NORD) ® is the leading independent advocacy organization representing all patients and families affected by rare diseases. NORD is committed to the identification, treatment and cure of the 7,000 rare diseases that affect 30 million Americans, or 1 in every 10 people. NORD began as a small group of patient advocates that formed a coalition to unify and mobilize support to pass the Orphan Drug Act of 1983. For 35 years, NORD has led the way in voicing the needs of the rare disease community, driving supportive policies and education, advancing medical research, and providing patient and family services for those who need them most. NORD represents more than 270 disease-specific member organizations and their communities and collaborates with many other organizations in specific causes of importance to the rare disease patient community.

Source: National Organization for Rare Disorders
3.07692307692
3.2 from 14 votes
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