DIA/FDA Orphan Drug Designation Workshop

In Collaboration with the National Organization for Rare Disorders (NORD) and Genetic Alliance FDA announced the launch of the Orphan Drug Designation Workshop series presenting an opportunity for academics, biotechnology companies and larger pharmaceutical firms to spend two days in creation of applications for orphan status designation. This workshop is designed to simplify and demystify entry into orphan drug development by small biotechnology companies, academics and larger industry entities.

Selected participants will be expected to bring specific products for at least one candidate orphan drug that holds promise for the treatment of a rare disease. This will allow you to directly engage in the practical matter of constructing an application for orphan status designation. To maximize the benefi ts of this workshop, we encourage you to come prepared with a working draft submission of a particular promising therapy.

Over two full days, FDA staff from the Office of Orphan Products Development (OOPD) will provide regulatory assistance to sponsors developing orphan drug designation applications.

The chief task of the orphan drug designation application is to convince the FDA’s Office of Orphan Product Development that:

1. The proposed drug is for a rare disease (i.e., that fewer than 200,000 persons in the US have the rare disease of condition); and
2. There is a medical rationale for believing the proposed drug has “promise” for treating the rare disease or condition.

Prior to registration for the Orphan Drug Designation workshop, prospective participants will be screened to ensure they have a proposed orphan product and the associated rare disease/condition. The turnaround time for notifying the prospective participants that they have been selected to attend is two days. All information submitted to the FDA will be kept confidential; and identification of participants and their organization will not be disclosed by the FDA or DIA.

Selected teams and participants will attend a plenary session with an introductory lecture and the two full days will be devoted to application writing and four one-on-one sessions with FDA officials to develop a strong orphan designation application. Final applications are expected to be submitted to the FDA on site at the close of the workshop or within 30 days.

WORKSHOP HIGHLIGHTS
• Application Writing
• One-on-One Guidance and Counseling with FDA – Sponsors will have one 45-minute and one 30-minute consultative session with an FDA OOPD staff member and two 20-minute follow-up sessions with an FDA OOPD staff member
• Final applications will be submitted to the FDA onsite at the close of the workshop or within 30 days.