The orphan designation process is the mechanism by which sponsors of drugs and biologics for rare diseases qualify for the tax credit and marketing exclusivity incentives of the Orphan Drug Act. The Office of Orphan Products Development also administers a clinical research grants program, whereby researchers may compete for funding to conduct clinical trials to support the approval of drugs for rare diseases. The number of grant awards varies each year depending on the availability of funds. On-going studies are funded first with the remainder of funds going to new studies. In recent years, OOPD has funded approximately 20-30 new awards annually.
Small businesses are encourage to apply.
Responsive applications are reviewed and evaluated for scientific and technical merit by an ad hoc panel of experts. A second level review is done by a National Advisory Council for concurrence with the recommendations made by the initial review group. Rank ordered priority scores determine final awards.