Gottlieb marks Rare Disease Day with update on orphan drug processes

As Rare Disease Day approaches, the US Food and Drug Administration has released a statement outlining steps the agency is taking to promote the development of therapies in this area.

Commissioner Scott Gottlieb says the agency will introduce a new pilot for more efficient orphan designation requests, including a new online form to make the submission process easier, and more straightforward.

In addition, the FDA has entered into a new Memorandum of Understanding with the National Organization for Rare Disorders to find ways to incorporate the “patient experience into regulatory discussions.”