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Orphan Drug Designation

BayBiotech.NET

Orphan Drug is a special status designated to a drug or biologics to treat a rare disease or condition. In order to obtain this designation, drug or biologics must meet certain criteria.

Orphan designation qualifies the sponsor for the tax credit and marketing incentives as per Orphan Drug Act. In addition, orphan drug is not subject to a prescription drug user fee unless used for other indications.

For application purposes, sponsor shall submit two copies of a completed, dated, and signed request for designation that contains the following:

(1) A statement that the sponsor requests orphan drug designation for a rare disease or condition.

(2) The name and address of the sponsor; the name of the sponsor's primary contact person.

(3) A description of the rare disease or condition for which the drug will be investigated.

(4) A scientific rationale for the use of the drug for the rare disease or condition.

(5) Where the sponsor of an already approved orphan drug seeks orphan drug designation for the subsequent drug for the same rare disease or condition, an explanation of why the proposed variation may be clinically superior to the first drug.

(6) If a drug is under development for only a subset of persons with a particular disease or condition, a justification that the subset is medically plausible.

(7) A summary of the regulatory status and marketing history of the drug in the United States and in foreign countries.

Application must have supportive documents to demonstrate that the disease or condition for which the drug is intended affects fewer than 200,000 people.
More information about Orphan Drug Status and tips for application can be found at:
Designating an Orphan Product: Drugs and Biologics, http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/HowtoapplyforOrphanProductDesignation/default.htm

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