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OOPD Frequently Asked Questions

The following questions are those that are most frequently asked of the Office of Orphan Products Development (OOPD) 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.

Are orphan drugs approved drugs?

Orphan drugs may be approved or experimental. The term "orphan drug" can refer to either a drug or biologic intended for use in a rare disease or condition. A drug or biologic becomes and "orphan drug" when it receives orphan-drug designation from the Office of Orphan Products Development at the FDA. Orphan-drug designation qualifies the sponsor to receive certain benefits from the Government in exchange for developing the drug for a rare disease or condition. The drug must then go through the FDA marketing approval process like any other drug or biologic which evaluates for safety and efficacy. To date over 1400 drugs and biologics have been designated as orphan-drugs, and over 250 have been approved for marketing.

How much do orphan drugs cost?

The cost of orphan products is determined by the sponsor of the drug and is not controlled by the FDA. The costs of orphan products vary greatly. Generally, health insurance will pay the cost of orphan products that have been approved for marketing.

What is the process to obtain a particular orphan drug?

If an orphan product has been approved by the FDA for marketing, it will be available through the normal pharmaceutical supply channels. If the product has not been approved by the FDA, the sponsor (the party studying the drug) may make the product available on a compassionate use basis. For contact information on sponsors of orphan products, contact the Office of Orphan Products Development.

What are the side effects for a particular orphan drug?

If the particular orphan product about which you are seeking side effect information is approved for marketing, that information can be found on the labeling for the product. If the product is not approved, you or your physician should consult the sponsor.

Will my insurance company pay for the orphan drug I need?

Reimbursement policy for drug expenses is set by the insurance company and is outlined in your policy. Insurance companies will generally reimburse for orphan products that have been approved for marketing, but may not reimburse for products that are considered experimental. Consult your insurance company about specific reimbursement policies.

How do I find a doctor in my area who is knowledgeable about my rare disease?

People with rare diseases often have difficulty locating a physician who is knowledgeable about their particular disease. If the doctor you are working with is unable to identify another physician with expertise in your specific disease, you can contact the National Organization for Rare Disorders (NORD) telephone (203) 744-0100. NORD maintains a database of organizations with expertise in various rare diseases. Another advocacy group, The Genetic Alliance, telephone (202)966-5557, has an online directory where you can search for further information about genetic diseases.

Are orphan products approved faster than other drugs?

Historically, the approval time for orphan products as a group has been considerably shorter than the approval time for other drugs. This reduced approval time is often due to the fact that many orphan products receive expedited review or accelerated approval because they are for serious or life-threatening disease.

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.

Is there a form for an orphan-drug request?

There is no form, but there is a prescribed format for a request for orphan-drug designation. Sections of regulations that pertain to format can be found at 21 C.F.R. 316.20 available at: How to Apply for Designation as an Orphan Product.

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 an application for approval to market the product.

Where should I send my Investigational New Drug application (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:

Food and Drug Administration
Center for Drug Evaluation and Research
5901-B Ammendale Road
Beltsville, MD 20705-1266

For a biologic:

Food and Drug Administration
Center for Biologics Evaluation and Research
1401 Rockville Pike, Room 200N (HFM-99)
Rockville, MD 20852

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

How much money is available for grants?

The current annual budget for funding grants is approximately $13 million. Clinical trials are awarded grants from $200,000 (Phase 1) and $350,000 (Phase 2 and 3) per year in total costs for up to 3 years. The annual Request for Application (RFA), published in the Federal Register 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 OPD 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 panel. Rank ordered priority scores determine final awards.

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