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FDA to Launch New Pilot Program for Orphan Designation Requests

Posted 26 February 2018 | By Zachary Brennan 

FDA to Launch New Pilot Program for Orphan Designation Requests

With more than 700 orphan designation requests last year, the US Food and Drug Administration (FDA) on Monday announced a new pilot program to make the request process more efficient.

The pilot will include a new form intended to make the submission process easier for sponsors to complete orphan designation requests, and to make the process more efficient for FDA, according to FDA Commissioner Scott Gottlieb.

FDA also released an on-line tutorial to guide sponsors through the submission process and Gottlieb noted there is a new inter-center consult process to streamline and standardize communications.

In addition to the pilot, FDA vowed to eliminate the orphan drug designation backlog, and is planning a public meeting to discuss the scientific and regulatory issues related to cancer treatments that target a tumor’s genetic features rather than its location in the body. In a first, FDA in May granted such an approval to Merck’s Keytruda (pembrolizumab) for patients with a common biomarker rather than the location of the body where the tumor originated.

“We’ll also consider the appropriate application of orphan incentives to this new paradigm of drug development, and how we apply designations to these indications,” Gottlieb said.

Meanwhile, FDA also entered into a new Memorandum of Understanding with the National Organization for Rare Disorders to conduct outreach with patient affairs staff to enhance the incorporation of patient experience into regulatory discussions.

“As part of this process, we’re planning a joint series of pilot listening sessions on rare diseases. We recognize that early and iterative engagement can improve clinical and regulatory understanding of diseases and conditions; provide a common understanding of the most urgent patient needs; and inform drug development programs,” Gottlieb said.

FDA's Orphan Drug Modernization Plan

Rare Disease Day 2018

September 12, 2017 Update to Orphan Drug Modernization Plan

Draft Guidance for Industry, Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases

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