Bioethics: Misuse of the Orphan Drug Act

In 1983, the Food and Drug Administration (FDA) adopted the Orphan Drug Act (ODA) to encourage pharmaceutical companies to develop drugs for rare diseases by offering incentives including credits for clinical trials and market exclusivity. The legislation has proven successful with over 300 drugs brought to market for over 12 million Americans suffering from rare diseases since its passage. One recent orphan drug designation was granted for Niemann Pick Type C after the mother of affected daughters attended a KGI workshop and filed the application herself. There is no question that the ODA has been beneficial to millions. However, that is not to say that it does not come with its own controversial bioethics issues. 

James Love, the director of Knowledge Ecology International comments "A drug company can drive a Brinks truck through the loopholes in the Orphan Drug Act." This bold statement refers to the numerous ways that drug companies can manipulate the ODA to fund the development of blockbuster drugs. One highly contentious misuse of the ODA is referred to as ‘salami slicing,’ or the segmentation of a disease with a large market to obtain a population small enough to qualify it as a rare disease. The act also ignores foreign drug sales and makes no distinction between drugs that require daily use and those that are used episodically. Although Tim Cote, the director of the FDA’s Office of Orphan Product Development (OOPD), comments that the office has handled this problem fairly well on a case-by-case basis, he also notes that the OOPD is working towards revision of existing regulations to transparently explain what kinds of subsets are acceptable and those that are not.

While the ODA is certainly providing much needed treatment to ailing populations, the potential for exploitation by shrewd businesses calls for future reforms by Mr. Cote and other leading officials.

By Christina Lai (MBS '11)