The road to the creation and passage of the Orphan Drug Act
(ODA) was long and arduous.
For reasons that are not clear, Congress amended the Orphan Drug Act
in 1984 by expanding the definition of "orphan drug" to include products for any disease or condition that affects less than 200,000 people in the US.
Para conseguir entender a origem da associacao EURORDIS, e considerando a influencia da associacao francesa AFM, na sua criacao e da associacao NORD (National Organization for Rare Disorders) na aprovacao do Orphan Drug Act
, e necessario analisar o historico destas associacoes para perceber o entrelacamento entre elas.
The FDA Orphan Drug Act
provides incentives for companies to develop products for rare diseases affecting fewer than 200,000 people in the United States.
The Orphan Drug Act
provides for granting special status to a drug or biological product to treat a rare disease or condition upon request of a sponsor.
Significant growth is expected in the therapeutics pipeline for ITP in the near future primarily driven by the rising demand for combination therapies due to their superior safety and efficacy profile as compared with other treatments, rising prevalence of viral diseases that are a major risk factor for ITP, favourable government regulations such as the Orphan Drug Act
in the U.S.
(http://info.evaluategroup.com/rs/607-YGS-364/images/EPOD15.pdf) That changed with the passage of the (https://www.fda.gov/RegulatoryInformation/LawsEnforcedbyFDA/SignificantAmendmentstotheFDCAct/OrphanDrugAct/default.htm) Orphan Drug Act
of 1983 , which allowed special grants for research, a 50 percent tax credit on trial costs, shorter FDA approval times and a guaranteed seven years of patent exclusivity.
Charles Grassley (R., Iowa) is considering a possible inquiry into alleged abuses of the Orphan Drug Act
as a factor behind high drug prices.
Defined by the Orphan Drug Act
of 1983 as a condition affecting fewer than 200,000 people nationwide, there are more than 6,000 orphan diseases known today ranging from well-known ALS to little-known NGLY1.
To encourage the development of drugs for uncommon diseases and conditions that affect less than 200,000 people in the United States, (8) Congress passed the Orphan Drug Act
of 1983 (9) and the Rare Diseases Act of 2002.
(1) It may be worth considering before using tetrabenazine, a medication approved in the United States under the Orphan Drug Act
, and launched at $34.25 for a 12.5 mg tablet and $68.50 for a 25 mg tablet.