ABOUT "ORPHAN DRUG STATUS"
(***translated from french***) Concept of Orphan Drug in the United States The legal status of the orphan drug was defined in the United States by the law of 4 January 1983 called the Orphan Drug Act. The Act states that "Any orphan medicinal product is indicated for a disease or circumstance that is rare in the United States so that the costs of development and distribution can not be reasonably recovered by sales on the national territory alone. " Several amendments have been made to the Orphan Drug Act to clarify the definition of the orphan drug. The 1984 amendment defines the concept of low prevalence as: "A rare disease or rare circumstance by definition: (1) Affects fewer than 200,000 individuals in the United States ... or ... (2) Affects more than 200,000 individuals United States without allowing the development and distribution costs to be amortized on the national territory. By granting an 'orphan' status to a drug, it allows the sponsor to BENEFIT INCENTIVES for the development of these products up to the AMM (*in french*) (Final Approval of the drug for its commercialisation). Thus, granting orphan drug status allows the sponsor to obtain the following ADVANTAGES for the development of the product: A TAX CREDIT up to 50% of the amounts committed for clinical trials conducted in the United States in the orphan indication; A COMMERCIAL EXCLUSIVITY WITH A DURATION OF 7 YEARS from the date of commercial authorization; Written recommendations provided by the FDA to the applicant regarding the points to be developed in the registration dossier; An ACCELERATED registration procedure at the FDA level to