Orphan Drug
Pursuant to section 526 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bb), JS-K orphan-drug designation request of 0 2-(2,4-Dinitrophenyl)1-((4- Ethoxycarbonyl)piperazin-1-ium-1,2-diolate was granted for the treatment of acute myeloid leukemia.
Congress passed the Orphan Drug Act of 1983. The law provides 7-year marketing exclusivity to sponsors of approved orphan products, a tax credit of 50 percent of the cost of conducting human clinical testing, and research grants for clinical testing.
In 1997, Congress created an additional incentive when it granted companies developing orphan products an exemption from the usual drug application or “user” fees charged by the Food and Drug Administration (FDA). In fiscal year 2020, these fees will total almost $3,000,000.
Many blockbuster drugs will lose their exclusivity in next 5 years. Therefore, the current economic situation plus the huge generic competition is shifting the focus of pharmaceutical companies to a the new business model with longer runways using orphan drugs.
Orphan drugs may help pharma companies to reduce the impact of revenue loss caused by patent expiries of blockbuster drugs.
These incentives for drug development provided by government, as well as support from the FDA and EU Commission in special protocols, are a further boost for JS-K.
Benefits from Orphan Drug designation:
7-year marketing exclusivity
a tax credit of 50 percent of the cost of conducting human clinical testing
federal research grants for clinical testing
exemption from the usual drug application or “user” fees charged by FDA
Orphan drugs generally follow the same regulatory development path as any other pharmaceutical product; testing focuses on pharmacokinetics and pharmacodynamics, dosing, stability, safety, and efficacy.
However, some statistical burdens are lessened in an effort to maintain development momentum. For example, orphan drug regulations generally acknowledge the fact that it may not be possible to test 1,000 patients in a phase III clinical trial, as fewer than that number may be afflicted with the disease in question. Sometimes only 1 clinical trial is needed to get an NDA approved.