In 1983, Congress passed the Orphan Drug Act (“ODA”) as part of an effort to provide market incentives for pharmaceutical manufacturers to produce “orphan drugs,” drugs for diseases that impact less than two hundred thousand people in the United States annually. Prior to the passage of the ODA, rare-disease patients had little to no treatment options. The ODA revolutionized the orphan drug space; therefore, the market for orphan drugs is becoming increasingly more lucrative for pharmaceutical manufacturers.

However, the recent prevalence of issues like “salami-slicing” and “indication stacking” provides pharmaceutical companies with excess market benefits and contributes to the prohibitively high cost of medicines. Both “salami-slicing” and “indication stacking” allow companies to obtain regulatory exclusivities for drugs already on the market, thus undercutting patient access by excluding competitors, thanks to the seven-year exclusivity period. Companies are free to raise the prices of their drugs how they see fit because they are now the only ones with regulatory exclusivity. As the market for orphan drugs has changed both domestically and globally, Congress must revisit the ODA to prioritize patient access to much-needed medicines. Congress should clarify the “same drug” language in the ODA by codifying the FDA’s practice of granting orphan drug exclusivity for specific uses or indications that a drug is approved for, as well as increase the bur- den of proof for companies seeking market exclusivity for drugs with stacked indications.