Potential Interactions of the Orphan Drug Act and Pharmacogenomics: A Flood of Orphan Drugs and Abuses?

Abstract

To overcome the unattractiveness of small markets, the United States government provides financial aid and incentives for drug manufacturers to create cures for rare diseases under the Orphan Drug Act (“the Act”). Recent research integrating genetic information and pharmacology holds promise for creating more effective drugs targeted at smaller populations than ever before. In the near future, it seems that a flood of new drugs targeted at small disease populations could take advantage of the government benefits under the Act. Drug applicants will include true orphan drugs along with “Trojan” applicants that seek to co-opt the benefits for drugs that should not qualify as orphans. Currently, the FDA appears ill prepared to discern between the two types of applicants and prevent abuse of the system.In 1983, the federal government passed the Act. Congress designed the Act and subsequent modifications to provide incentives for companies to bring drugs for rare diseases to market.