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.
Are orphan drugs approved drugs?
Orphan drugs (and biologicals) may be approved or may still be experimental. A drug becomes an "orphan" when it receives orphan designation from the Office of Orphan Products Development at the FDA. Orphan designation qualifies the sponsor to receive certain benefits from the Government in exchange for developing the drug. The drug must then go through the new drug approval process like any other drug. To date approximately 890 orphan products have been designated and over 170 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 for marketing, it will be available through the normal pharmaceutical supply channels. If the product has not been approved, the sponsor 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 may want to call the National Organization for Rare Disorders (NORD). NORD maintains a database of doctors with expertise in various rare diseases. The Metabolic Information Network (MIN), (800)945-2188, maintains a database of physicians with expertise in various inborn metabolic 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 all drugs. This is due to the fact that many orphan products receive expedited review 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 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.
Is there a form for an orphan drug application?
There is no form, but there is a prescribed format for application for orphan drug status. The section from the regulations that describes the format can be found on this website on the The Orphan Drug Act and Related Law and Regulations page
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?
Legislation reauthorizing FDA's user fees also created an exemption for orphan products from fees imposed when an application to approve the product for marketing is submitted. While orphans are not exempt from product fees or establishment fees, waivers can be requested where appropriate or necessary.
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 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 AdministrationCenter for Drug Evaluation and ResearchCentral Document Room12229 Wilkins AvenueRockville, MD 20852For a biologic:
Food and Drug AdministrationCenter for Biologics Evaluation and Research1401 Rockville Pike, Room 200N (HFM-99)Rockville, MD 20852All 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 $12 million. Clinical trials are awarded grants from $100,000 to $200,000 per year in direct costs for up to 3 years. The annual 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 20-30 new awards annually.
Who may apply?
Academic institutions and other responsible organizations: public, private, non-profit, or for-profit. Small businesses are encourage 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 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.
What are "designated" products?
To find out what designated products are, go the Orphan Product Designation page.