Multiple sponsors can receive orphan drug designation for the same drug and indication; marketing exclusivity would go to the first sponsor to receive marketing approval.
The same drug can be made available by other sponsors for different uses during the 7-year period of exclusivity. Regarding market exclusivity: A drug that is similar to another authorized orphan drug for the same rare disease indication must demonstrate clinical superiority. The FDA cannot approve the same drug for the same indication from a competitor during the period of market exclusivity unless the sponsor of the first authorized orphan product is unable to provide sufficient supply of the product, or the first sponsor provides its consent. Under this program, studies have been funded and 45 products have received market approval.
The prevalence of the condition in the EU must not be greater than 5 in 10,, or it must be unlikely that marketing the medicine would generate sufficient returns to justify the investment needed for its development. No satisfactory method of diagnosis, prevention, or treatment of the condition concerned can be authorized, or, if such a method exists, the medicine must be of significant benefit [defined as a clinically relevant advantage or a major contribution to patient care] 78 to those affected by the condition.
A marketing authorization for another orphan product can be granted if: The original orphan product MAH consent,; or the original MAH cannot supply the product in sufficient quantity to meet demand, or the new applicant for a medicinal product similar to the authorized orphan product can demonstrate that it is safer, more effective, or otherwise clinically superior. Filling an unmet medical need is defined as providing a therapy where none exists or providing a therapy which may be potentially better than available therapy.
Clinically significant endpoint generally refers to an endpoint that measures an effect on irreversible morbidity or mortality or on symptoms that represent serious consequences of the disease. Priority Review status allows for a shortened review target of calendar days. Significant improvement may be demonstrated by the following examples: evidence of increased effectiveness in treatment, prevention, or diagnosis of condition; elimination or substantial reduction of a treatment-limiting drug reaction; documented enhancement of patient compliance that is expected to lead to an improvement in serious outcomes; or evidence of safety and effectiveness in a new subpopulation.
References Orphan Drug Act. Relevant excerpts public law , as amended : Last updated January 21 [Internet]. Silver Spring MD : U. Food and Drug Administration; Jun Human regulatory [Internet]. London: The Agency. Orphan designation; May 22 [cited Dec 4]. Rare disease policies to improve care for patients in Europe. Biochim Biophys Acta [Internet].
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