FDA launches workshop to simplify orphan drug development

The U.S. Food and Drug Administration (FDA) has announced the launch of an orphan drug workshop series--an opportunity for biotechnology companies, pharmaceutical firms, academics and other interested parties to spend two days creating applications for orphan status designation, under the guidance of staff from FDA's Office of Orphan Products Development (OOPD). The first workshop is planned for Feb. 25-26, 2010, hosted by the Center for Rare Disease Therapies at Keck Graduate Institute (KGI) in Claremont, Calif.

The intent of this workshop is to simplify and demystify entry into orphan drug development. Each attendee must come prepared to work on an application for a particular drug or therapy.

There will be an introductory presentation, but most of the time over the two-day period will be spent writing an application for orphan designation for specific products, which will be submitted at the close of the workshop.

Having a product designated an "orphan" provides important incentives and can be done at any time prior to submission of a marketing application. To receive orphan designation, the submitting organization must convince the FDA-OOPD that the proposed drug is targeted toward a rare disease (one with a patient population of fewer than 200,000 people) and holds promise for treating the rare disease.