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Frequently Asked Questions Concerning the Orphan Product Designation

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 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.

How many copies of a designation request are required?

When submitting a request for orphan designation, the sponsor must submit the original and one copy.

Are orphan drugs exempt from FDA User Fees?

A human drug application for a prescription drug product that has been designated as a drug for a rare disease or condition pursuant to section 526 of the Federal Food, Drug, and Cosmetic Act, shall not be subject to a fee unless the human drug application includes an indication for other than a rare disease or condition.

A supplement proposing to include a new indication for a rare disease or condition in a human drug application shall not be subject to a fee, if the drug has been designated pursuant to section 526 of the Federal Food, Drug, and Cosmetic Act as a drug for a rare disease or condition with regard to the indication proposed in such supplement.

There is no specific exemption for orphan drug products from annual product and establishment fees. However, sponsors of orphan drugs can request a waiver, under section 736(d)(1) of the Federal Food, Drug, and Cosmetic Act.

Can an orphan drug application be submitted anytime in the development process?

Under the law, application for designation as an orphan product can be made any time prior to the filing of a marketing application for the product.

Where should I send my IND submission?

Initial IND submissions should be submitted in triplicate (original and two photocopies) and should be accompanied by a form FDA-1571. The submission should be sent to one of the following offices depending on whether the product is a drug or a biologic:

For a drug:

For a biologic:

All subsequent submissions to an IND should go directly to the appropriate FDA reviewing division.

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