Academic journal article American Journal of Law & Medicine

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

Academic journal article American Journal of Law & Medicine

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

Article excerpt


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").1 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.2 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.3 Thus, many drugs that could help rare disease populations were "orphans" without a parent company to sponsor them through clinical trials.4 The Act provided various financial incentives, including grants and market exclusivity, for companies to bring orphan drugs to market.3 The Act successfully encouraged development of previously unprofitable drugs, but also funded the development of several blockbuster drugs, which attracted criticism.6

Beginning in October 1990, the Human Genome Project ("HGP") endeavored to bring a further understanding of human genetics to biological and medical science. With a combination of government and private actors, the HGP "catalyzed the multi-billion dollar U.S. biotechnology industry and fostered the development of new medical applications."7 Pharmacogenomics developed through the combination of genetic knowledge with pharmacological science.8

Pharmacogenomics concerns the interaction of pharmacology with an individual's specific genetic makeup.9 Biotechnology and pharmaceutical companies already utilize the technology to subdivide patients with the same disease into different genetic classes, changing the way drugs are delivered.10 One day, the technology could seriously alter the way drugs are developed and enable tailored treatments for different genetic classes within a disease population." Thus, pharmacogenomics holds the potential to increase drug efficacy via administration and development, independently.

Under the current version of the Act, pharmacogenomic technology might create an avalanche of new orphan drugs. Drug developers could genetically subdivide diseases that affect a large portion of the population into groups small enough to qualify for orphan drug status. While targeted at one specific subgroup, these pharmacogenomic orphan drugs could retain efficacy for treating other subgroups of the disease. For example, if a condition that affected millions of Americans was determined to have a genetically identifiable subgroup with a population less than 200,000, a treatment for that subgroup would qualify as an orphan drug. After developing this specific treatment, the parent company could promote it as a drug for the entire disease population. If done intentionally, this would violate the original purpose of the Act and co-opt the benefits of the Act to serve as government subsidies for a fledgling pharmacogenomic industry.

Additionally, the orphan drug approval process could be exploited via pharmacogenomic manipulation of clinical trials. Drug sponsors could screen out patient populations genetically prone to unfavorable drug reactions in an effort to improve the results of clinical trials. The Act allows "clinically superior" new orphan drugs to puncture the market exclusivity shield of old orphan drugs. …

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