Developing Products for Rare Diseases & Conditions
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`http://www.fda.gov/forindustry/DevelopingProduCTsforrareDiseasesCon...
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`Home For Industry Developing Products for Rare Diseases & Conditions
`For Industry
`Developing Products for Rare Diseases & Conditions
`
`The FDA Office of Orphan Products Development (OOPD) mission is to advance the evaluation and development of
`products (drugs, biologics, devices, or medical foods) that demonstrate promise for the diagnosis and/or treatment
`of rare diseases or conditions. In fulfilling that task, OOPD evaluates scientific and clinical data submissions from
`sponsors to identify and designate products as promising for rare diseases and to further advance scientific
`development of such promising medical products. The office also works on rare disease issues with the medical and
`research communities, professional organizations, academia, governmental agencies, industry, and rare disease
`patient groups.
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`OOPD provides incentives for sponsors to develop products for rare diseases. The program has successfully enabled
`the development and marketing of more than 400 drugs and biologic products for rare diseases since 1983. In
`contrast, fewer than 10 such products supported by industry came to market between 1973 and 1983. The Orphan
`Grants Program has been used to bring more than 45 products to marketing approval. The Humanitarian Use Device
`Program has been the first step in approval of more than 50 Humanitarian Device Exemption approvals.
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`The Orphan Drug Designation program1 provides orphan status to drugs and biologics which are defined as those
`intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer
`than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs
`of developing and marketing a treatment drug.
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`The Humanitarian Use Device (HUD) program2 designates a device that is intended to benefit patients by treating or
`diagnosing a disease or condition that affects fewer than 4,000 individuals in the United States per year as per 21
`CFR 814.3(n).
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`3
` provides funding for
`The OOPD administers two extramural grant programs. The Orphan Products Grants Program
`clinical research that tests the safety and efficacy of drugs, biologics, medical devices and medical foods in rare
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`diseases or conditions. The Pediatric Device Consortia (PDC) Grant Program
`consortia to facilitate pediatric medical device development.
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`For more information on any of our programs, please visit the programs’ web pages.
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` provides funding to develop nonprofit
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` Please note our mailing address, telephone number, FAX number and email address:
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`Office of Orphan Products Development
`Food and Drug Administration
`WO32-5295
`10903 New Hampshire Avenue
`Silver Spring, MD 20993-0002
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`Main Telephone Number: 301-796-8660
`Fax Number: 301-847-8621
`Email: orphan@fda.hhs.gov
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`Developing Products for Rare Diseases & Conditions
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`http://www.fda.gov/forindustry/DevelopingProduCTsforrareDiseasesCon...
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`Links on this page:
`/ForIndustry/DevelopingProductsforRareDiseasesConditions
`1.
`/HowtoapplyforOrphanProductDesignation/default.htm
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`2.
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`3.
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`4.
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`/ForIndustry/DevelopingProductsforRareDiseasesConditions
`/DesignatingHumanitarianUseDevicesHUDS/default.htm
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`/ForIndustry/DevelopingProductsforRareDiseasesConditions
`/WhomtoContactaboutOrphanProductDevelopment/default.htm
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`http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions
`/PediatricDeviceConsortiaGrantsProgram/default.htm
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