NORD to Collaborate with FDA on Pilot Patient Engagement Activity

Categories: Advocacy
Posted by Christina Jensen, National Organization for Rare Disorders website, February 28, 2018


Washington, D.C., February 28, 2018—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 affect 200,000 or fewer Americans. The vast majority still have no treatment.


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