FDA Observes Rare Disease Day with Help from NORD, NIH

Mathew Shanley

In observance of World Rare Disease Day, the U.S. Food and Drug Administration (FDA) has announced a collaboration with the National Organization for Rare Disorders (NORD), in which a pilot project would assist FDA medical reviewers in better understanding rare disease patient experiences.

2018 marks the 11th anniversary of the first-ever World Rare Disease Day, and the 10th time that it has been recognized in the United States. With this pilot patient engagement activity, the FDA and NORD will partner to identify patient community representatives and coordinating listening sessions where patients can share their experiences with the FDA review division staff.

“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 in a press release. “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.

At the request of review divisions, the listening sessions will be prepared through the FDA’s Patient Affairs Staff (PAS) in cooperation with NORD. PAS will coordinate with the review division to outline questions for the patients and work with NORD to facilitate the conversation between the requesting review division and patient community.

“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 continued. “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.”

Since the approval of Scott Gottlieb, M.D. as the FDA Commissioner last May, the FDA has worked toward bringing the patient perspective to Washington D.C. Gottlieb has instituted an aggressive plan to streamline the process by which orphan drugs are reviewed, attempted to reduce the enormity of the orphan drug backlog, and has implemented new ways of increasing rare disease research.

Earlier this week, the FDA launched the 2018 version of its Rare Disease Day website. On it, Gottlieb stresses his commitment to the rare disease community.

“Despite our successes, there are still no treatments for the vast proportion of rare diseases or conditions,” he states. “FDA is committed to do what we can to stimulate the development of more products by improving the consistency and efficiency of our reviews, streamlining our processes and supporting rare disease research.”

Also on the website, visitors will find a series of new videos and a wide array of other resources that explain in detail exactly what plans the FDA has while it continues to develop medical products for the rare disease community.

On March 1, the FDA will join the National Institutes of Health (NIH) to commemorate Rare Disease Day with a one-day program at NIH Masur Auditorium. There, the FDA’s Office of Orphan Products Development will present FDA rare disease highlights and its grants research collaboration with NIH for rare diseases.

For more information about Rare Disease Day and events that are taking place to globally celebrate the occasion, follow Rare Disease Report on Facebook and Twitter.
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