Worried about creating an orphan drug designation application?

Then, you're not alone. Typically, submissions to the FDA evoke considerable consternation among drug sponsors. This workshop is designed to make the process of orphan drug development transparent and accessible to those uninitiated in regulatory affairs.

The chief task of the orphan status designation application is to convince the FDA's Office of Orphan Product Development of two things: that the proposed drug is for a rare disease (i.e. that fewer than 200,000 persons in the US have the rare disease or condition) and that there is a medical rationale for believing the proposed drug has "promise" for treating the rare disease/condition (i.e. clinical data, animal model data OR, rarely, in vitro data, but not exclusively theoretical considerations). Orphan status designation can be secured anytime prior to the submission of a marketing application; it need not be under consideration as an investigational new drug (IND) nor are preclinical toxicology studies nor manufacturing certifications required. If you have a product envisioned for treatment of a rare disease you want to see move forward and there is sufficient data to show it may be promising, then you're ready.

At this workshop, under the guidance of Dr. Tim Coté, Director of the FDA's Office of Orphan Drug Development, and other FDA staff will directly engage in the practical matter of constructing an application for orphan status designation. Finalized applications will be submitted to the FDA on site at the close of the workshop. Attendees should note that participation in the workshop confers neither expedited processing nor preferential consideration of their applications.
REGISTRATION WILL BE LIMITED to 50 companies or organizations (maximum of 3 persons per organization) and accepted on a first come-first served basis. Each attendee must identify the proposed orphan product for which an application will be developed along with the anticipated rare diseases to which the proposed product will be applied. Participants need not be the holders of intellectual property rights of the compounds for which they seek orphan designation, but need only to be a "party of interest" and will be considered to be "sponsors" of designations that may be ultimately granted.

*Fees are charged by Keck Graduate Institute to cover the costs of the workshop. FDA does not receive any of these funds and does not endorse any fundraising activities related to this workshop.