Orphan Drugs10 Jul 2023 10:43
Orphan drugs are drugs designed to treat "orphan", or rare, diseases. Because these diseases affect so few patients, there has historically been less research into these diseases compared to more prevalent conditions. To incentivize rare disease research, FDA has created several programs that help reduce the cost and time of developing drugs for severe rare disease. The most widely known is the Orphan Drug Act, but there are several other FDA programs that are arguably just as, if not more, important in reducing the cost of orphan drug development (although these programs are not specific for orphan drugs): Accelerated Approval, Breakthrough Designation, Fast Track, and Priority Review.