Emerging Orphan Drugs and  Rare Diseases

Gaming and Abuse of Orphan Drugs

Pattern of gaming the system by slicing and dicing indications so that drugs qualify for lucrative orphan status benefits and getting taxpayer subsidies for blockbuster drugs, says a veteran scientist in USA. Companies are intentionally exploiting the law by putting up an initial listing of only single narrow indication for an orphan drugs use, which is just enough to qualify for "orphan" drug designation. Under the terms of this act, companies can receive federal taxpayer subsidies of up to half a million dollars a year for up to four years per drug, large tax credits and waivers of marketing application fees that can cost more than $2 million. In addition, the Food and Drug Administration (FDA) can grant companies seven years of marketing exclusivity for an orphan drug to ensure they recoup the costs of research and development. When those financial incentives become sort of an investment opportunity to take advantage of the Orphan Drug Act. The review found that orphan drug spending in the United States totalled $15 billion in 2007 and $30 billion in 2013, an increase from 4.8 percent of total pharmaceutical spending to 8.9 percent. The current study projects orphan drug spending will remain fairly stable as a proportion of total drug spending. That stands in contrast to other published reports that estimate orphan drugs will account for 20 percent of worldwide spending on drugs (other than generics) by 2020.