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  1. FDA In Brief

FDA in Brief: FDA Launches Portal for Submission of Orphan Drug Designation Requests

November 23, 2020

Media Inquiries

  Jeremy Kahn
  (301) 796-8671

The following quote is attributed to FDA Principal Deputy Commissioner Amy Abernethy M.D., Ph.D.:

“Developing a treatment for a rare disease can be complex. Collaboration, including through the use of technology, is critical to supporting and facilitating efficient processes for these types of necessary treatments."

"To help achieve this goal, today, we’re pleased to announce the availability of our Orphan Drug Designation Portal to support the development of treatments for rare diseases by enhancing sponsor communication with the FDA and increasing ease of orphan drug designation submissions. This important initiative is part of broader technology modernization efforts at the FDA to develop a more connected information technology system, advanced analytics and improvements in knowledge management. This is especially key during the current public health emergency when the ease of electronic submission options is more important than ever before."

"Through effective collaboration we will continue to further advance rare disease product development and support safe and effective treatments of patients impacted by rare diseases.”

Additional Information:

  • Today, the U.S. Food and Drug Administration is announcing the availability of an Orphan Drug Designation Portal. The Orphan Drug Designation Portal is part of the FDA’s Orphan Drug Technology Modernization efforts. As outlined in the January FDA Voices, this initiative supports the development of treatments for patients with rare diseases by strengthening the information technology process within the FDA’s Office of Orphan Products Development.
  • The Orphan Drug Designation Portal will now allow electronic submission of new orphan drug designation requests through a cloud-based online submission portal. Moving from a paper-based to a cloud-based submission portal provides drug developers with enhanced, direct communication with the FDA regarding each submission. The portal system will help ensure questions are answered and will allow for feedback from sponsors. The FDA has provided detailed information on the agency’s portal landing page.
  • As an additional part of these efforts, the Office of Orphan Products Development has also implemented a new internal orphan drug designation workflow management tool. The Orphan Drug Technology Modernization efforts allow for a more connected internal information technology system, advanced analytics and improvements in knowledge management. The workflow management component facilitates the set of tasks necessary in reviewing an orphan drug designation submission and automates certain review and processing functions to provide added efficiencies in the orphan drug designation review process. The agency will continue to work on these technological enhancements to support the FDA’s mission to advance the evaluation and development of safe and effective treatments for rare disease patients and families.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

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