FDA to Allow Online Submissions of Orphan Designation Requests

Regulatory NewsRegulatory News | 17 January 2020 |  By 

As part of work building on the US Food and Drug Administration’s (FDA) orphan drug modernization plan from 2017, the agency said Friday that later this year it will move from a paper-based process to a new cloud-based online submission portal for orphan drug designation requests.

The shift to online submissions will ease the process of making orphan drug designation requests, which are currently mailed into the Office of Orphan Products Development (OOPD) on a CD.

“The Orphan Drug Technology Modernization effort will allow for a more connected information technology system, advanced analytics, and improvements in facilitating knowledge management. In addition, it will provide external sponsors with more efficient submission of documents and enhanced direct communication with the FDA,” Amy Abernethy, principal deputy commissioner and acting CIO and Janet Maynard, director of FDA’s OOPD, wrote in a blog post.

Abernethy and Maynard said this effort is also part of FDA's technology modernization plan.

The shift on orphan drug designation request submissions follows the Center for Drug Evaluation and Research’s (CDER) approval of more than twice as many orphan drugs in the past eight years as in the previous eight years.

In addition, FDA will hold a meeting on rare disease drug development next month to obtain stakeholders' perspectives on challenges and solutions in rare disease product development. A panel discussion with FDA center directors and other dialogue with FDA senior staff will take place.

FDA’s Center for Biologics Evaluation and Research (CBER) is also organizing a public workshop, to be held in March, titled, “Facilitating End-to-End Development of Individualized Therapeutics.” 

In an editorial published in the New England Journal of Medicine in October, Janet Woodcock, director of CDER, and Peter Marks, director of CBER, explained how these new personalized treatments can “permit the delineation of pathways for truly individualized drug development," but currently this type of "drug discovery and development is most advanced for ASOs [antisense oligonucleotides], other types of treatments, including individualized cell and gene therapies, are following closely behind.”


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