Potential interactions of the Orphan Drug Act and pharmacogenomics: a flood of orphan drugs and abuses?

To overcome the unattractiveness of small markets, the United States govemment 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 fiood of new drugs targeted at small disease populations could take advantage of the govemment 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 discem 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.^ Traditionally, even when cures for rare diseases were discovered, the large cost associated with sponsoring a drug through clinical trials discouraged pharmaceutical companies from bringing the drugs to market.^ Thus, many drugs that could help rare disease populations were "orphans" without a parent company to sponsor them through clinical trials." The Act provided various financial incentives, including grants and market exclusivity, for companies to bring orphan drugs to market.^ The Act successfully encouraged development of previously unprofitable drugs, but also funded the development of several blockbuster dmgs, which attracted criticism.*