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The following questions are those that are most frequently asked of the Office of Orphan Products Development along with answers. This section is updated regularly to reflect topics of current interest. The "Comments" section of this Webpage is a major source of questions for the FAQ page. To make a comment or offer a question, simply click on the comments button at the end of the page.
What is the difference between an orphan grant and an orphan designation?
The orphan designation process is the mechanism by which sponsors of drugs and biologics for rare diseases may qualify for incentives of the Orphan Drug Act such as tax credits and marketing exclusivity. 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, biologics, medical devices, and medical foods for rare diseases and conditions. A product does not have to be designated to be eligible for the grant program.
What Studies qualify?
Only clinical studies qualify for consideration. Each application should propose one discrete clinical study designated to facilitate FDA approval of the product for use in a rare disease or condition. The study may address an unapporved new product or an unapproved new use for a a product already on the market. All studies must be conducted under an Investigational New Drug Application (IND) or an Investigational Device Exemption (IDE). Only medical foods are exempted from the IND/IDE requirement.
How much money is available for grants?
The current annual budget for funding grants is approximately $13 million. Clinical trials are awarded grants from $200,00 (Phase 1) to $350,000 (Phase 2 and 3) per year in total costs for up to 3 years. The annual Request for Applications (RFA) will provide more up-to-date information on dollar limits.
How are awards made?
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 12-15 new awards annually.
Who may apply?
Academic institutions and other responsible organizations: public, private, non-profit, or for-profit. Small businesses are encouraged to apply.
How are applications reviewed?
Applications are first reviewed by the OOPD program staff for relevance and responsiveness to the RFA. Responsive applications are reviewed and evaluated for scientific and technical merit by an ad hoc panel of independent experts. A second level review is done by a National Advisory Council for concurrence with the recommendations made by the ad hoc review panel. Rank ordered priority scores determine final awards.