FDA-EMA Meeting on Orphan Drug Development: Offers Overview of Program and One on One Sessions with Product Developers

The Food and Drug Administration (FDA) recently announced its Office of Orphan Products Development will be holding a meeting entitled “The Food and Drug Administration/European Medicines Agency Orphan Product Designation and Grant Workshop.”

The Orphan Drug Act (ODA) provides for granting special status to a drug or biological product (“drug”) to treat a rare disease or condition upon request of a sponsor. This status is referred to as orphan designation (or sometimes “orphan status”). For a drug to qualify for orphan designation both the drug and the disease or condition must meet certain criteria specified in the ODA and FDA’s implementing regulations at 21 CFR Part 316.

Orphan designation qualifies the sponsor of the drug for various development incentives of the ODA, including tax credits for qualified clinical testing. A marketing application for a prescription drug product that has received orphan designation is not subject to a prescription drug user fee unless the application includes an indication for other than the rare disease or condition for which the drug was designated.

The program has successfully enabled the development and marketing of more than 400 drugs and biologic products for rare diseases since 1983.  In contrast, fewer than 10 such products supported by industry came to market between 1973 and 1983.  The Orphan Grants Program has been used to bring more than 45 products to marketing approval. The Humanitarian Use Device Program has been the first step in approval of more than 50 Humanitarian Device Exemption approvals.

This 1-day workshop is intended to provide valuable information about the FDA and European Medicines Agency (EMA) Orphan Drug Designation programs, the FDA Humanitarian Use Device (HUD) Designation program, and the FDA Orphan Products Grant program to participants representing pharmaceutical, biotechnology, and device companies, as well as academics.

The meeting will be held on October 4, 2013, from 8:30 a.m. to 4:00 p.m. and will be held at FDA’s White Oak Campus in Silver Spring, Maryland. Interested participants may register for this meeting here.

Online registration for the workshop will be limited to 240 participants for the morning session, of which approximately 50 teams (up to 150 participants) may register for the one-on-one sessions. There will be no registration fee for the workshop.

For participants who cannot attend the morning meetings, simultaneous live interactive Webcasts will be made available. Participants may access the drug and biologics Webcast here and devices here.

The meeting is being conducted in partnership with the European Organisation for Rare Diseases, Genetic Alliance, and the National Organization for Rare Disorders.

The morning program includes two simultaneous sessions. The first will provide an overview of the FDA and EMA Orphan Drug Designation programs, respectively, while the second will provide an overview of the FDA HUD Designation Program and Pediatric Device Consortia Grant Program. Both morning sessions will also cover the Orphan Products Grant Program as they relate to drugs, biologics, and devices. Both of these morning sessions will also be available by Webcast.

The Humanitarian Use Device (HUD) program designates a device that is intended to benefit patients by treating or diagnosing a disease or condition that affects fewer than 4,000 individuals in the United States per year as per 21 CFR 814.3(n).

The afternoon session (no Webcast), provides an opportunity for appropriately registered participants to have one-on-one meetings with FDA staff members onsite, to discuss the specifics on how to apply for an orphan product grant, a HUD designation, or orphan drug designation. It also provides for videoconference sessions with EMA staff representatives on EMA orphan drug designation.

Participants requesting one-on-one meetings are expected to bring information for at least one candidate orphan drug or device that holds promise for the treatment of a rare disease or condition in order to discuss the processes for putting together an application. In addition, participants in the HUD or orphan drug designation one-on-one sessions are highly encouraged to come prepared with a working draft submission of their particular promising therapy in order to maximize the utility of the one-on-one meetings.