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FDA at Rare Disease Day / February 28, 2011

Developing Orphan Products: FDA and Rare Disease Day

Organizations around the world are observing February 28, 2011, as the 4th annual Rare Disease Day. The Food and Drug Administration (FDA) supports Rare Disease Day and the global effort to raise awareness about rare diseases, the importance of research to develop diagnostics and treatments, and the challenges faced by patients with rare diseases and their families on a daily basis. In recognition of Rare Disease Day, we would like to highlight a new section of the Office of Orphan Products Development (OOPD) website which includes the personal stories of individuals with rare diseases.

The (FDA) is committed to advancing rare disease therapies through the development of orphan products. There are about 7000 rare diseases that collectively affect over 30 million Americans. Some US citizens, in the early 1980’s, turned to Congress for help, and in 1983 the Orphan Drug Act (ODA) became law. The OOPD is within the FDA and administers a part of the special efforts required to develop orphan products for rare diseases. When the Orphan Drug Act (ODA) of 1983 was signed into law, no one could have imagined then that it would become one of the most important pieces of health care legislation today. More than 350 orphan products for treatment of rare diseases have been approved by FDA since this groundbreaking legislation went into effect. In the decade before the ODA was passed, only 10 treatments had been developed for rare diseases.

Rare Diseases and Orphan Products:

  • A “rare disease” is a disease or condition that affects less than 200,000 people in the United States.
  • An “Orphan Product” is a drug, biologic, device or medical food that is used for the prevention, diagnosis, or treatment of a rare disease.

Why are they called orphan products?

Products used for rare diseases are called “orphan products” because they generally lack sponsors to develop them. The market for an orphan product is likely to be small. With costly research and the prospect of limited profit, companies have few incentives to bring an orphan product to market.

How does OOPD help?

OOPD assists potential sponsors of orphan products by directing the following programs:

  • Orphan Drug Designation Program – qualifies a product for special financial incentives.
  • Orphan Products Grant Program – provides funding for clinical investigations.
  • Pediatric Device Consortia (PDC) Grant Program – facilitates pediatric medical device development.
  • Humanitarian Use Device (HUD) Program – motivates businesses to develop medical devices for rare diseases and conditions.


Orphan drug designation is the process by which a sponsor for a drug or biologic product can take advantage of three special incentives provided by the ODA and its subsequent amendments:

  • Exclusivity – The first sponsor of a designated orphan drug to obtain FDA marketing approval for the designated rare disease or condition receives seven years of marketing exclusivity.
  • Tax credit – A sponsor may claim as tax credits half of the qualified clinical research costs for a designated orphan product.
  • Waiver of Prescription Drug User Fees – The sponsor’s fee as prescribed by the Prescription Drug User Fee Act (PDUFA Fees) at the time of submitting a marketing application to FDA are waived for a designated product.


OOPD administers grants to defray the costs of clinical research needed to investigate orphan products as authorized by the ODA. OOPD has provided more than $260 million in orphan product research grants to fund over 600 clinical investigations. OOPD currently expends about $14 million annually to fund approximately 100 clinical investigations in various stages of evaluation of potential therapies for rare diseases and conditions.

Some key details about OOPD grants:

  • Phase I clinical investigations may receive up to $200,000 per year for up to three years and Phase II and Phase III clinical investigations, may receive up to $400,000 of total costs per year for up to four years.
  • Drugs, biologics, devices and medical foods for a rare disease or condition are eligible for OOPD grants.


The goal of the FDA’s PDC Grant Program is to support the development of nonprofit consortia designed to stimulate projects which will promote pediatric device development. The consortia will facilitate the development, production, and distribution of pediatric medical devices.


The Humanitarian Use Device (HUD) and Humanitarian Device Exemption (HDE) programs were developed to encourage development of medical devices for the treatment of rare diseases or conditions that occur in fewer than 4,000 individuals in the United States per year. The HUD/HDE marketing pathway is a marketing approval process that under certain conditions will allow medical devices for the treatment of rare disease/conditions to be commercialized if they meet safety and probable benefit criteria.

FDA Highlights of Accomplishments in Rare Diseases

Since it was created in 1982, the OOPD has devoted significant effort to promoting the development of products that demonstrate promise for the prevention, diagnosis and/or treatment of rare diseases or conditions. In doing so, OOPD interacts with the medical and research communities, professional organizations, academia, and the pharmaceutical industry, as well as rare disease groups and other concerned citizens. OOPD continually seeks commercial sponsors for orphan products and strives to increase awareness of the assistance available through OOPD in the form of orphan designation incentives and funding through OOPD grants program.

Highlights of accomplishments during the 2010 calendar year:

  • OOPD granted 192 new orphan drug designations to promising drugs and biologic agents, and 19 new humanitarian use device designations.
  • OOPD continues its highly success¬ful grant program to support new and continuing extramural research projects that test the safety and efficacy of promising new drugs, bio¬logics, medical devices, and medical foods for rare diseases and conditions through human clinical trials.
  • OOPD continues a new month-long course in collaboration with the National Institutes of Health (NIH) Office of Rare Diseases, “The Science of Small Clinical Trials,” designed to increase awareness about the special requirements of perform¬ing drug research and evaluation in small populations.
  • FDA and NIH announced a collaborative initiative to fast-track innovations to the public.


  • OOPD continued efforts to find potentially promising drugs mentioned in FDA’s records which have been abandoned by their developers for reasons other than their clinical safety and effectiveness (for example, due to a business decision or merger).
  • FDA continues to collaborate with international regulatory agencies to address unmet medical needs of patients with rare diseases globally. OOPD and EMA work together regularly and have a common application for orphan product designation. (See EMA activities for Rare Disease Day at: www.ema.europa.eu disclaimer icon)
  • OOPD initiated “Do A Designation” workshops, with three held this year throughout the United States. These workshops provide one-on-one guidance sessions to develop applications for orphan status designation.
  • The Center for Drug Evaluation and Research (CDER), Office of New Drugs (OND), established the Rare Diseases Program to facilitate and support the research, development, regulation, and approval of drug and biologic products for the treatment of rare disorders.

For More Information:

Rare Disease Day
www.rarediseaseday.orgdisclaimer icon

FDA Office of Orphan Products Development (OOPD)

NIH Office of Rare Diseases (ORD)
www.rarediseases.info.nih.govdisclaimer icon

National Organization for Rare Disorders (NORD)
www.rarediseases.orgdisclaimer icon

Genetic Alliance
www.geneticalliance.orgdisclaimer icon

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