The Orphan Drug Act: An Appropriate Approval Pathway for Treatments of Rare Diseases?

The Orphan Drug Act provides financial incentives to pharmaceutical manufacturers to develop treatments for rare diseases affecting limited patient populations. Since passage of the Act in 1983, the U.S. Food and Drug Administration (FDA) has approved more than 600 orphan drug indications from greater than 450 distinct drug products. The annual number of orphan drug designation approvals has increased significantly in the recent past with much of this increase driven by approval of secondary indications for previously approved treatments. This recent increase has led to concerns regarding the Act as some pharmaceutical manufacturers have reaped outsized financial benefits while avoiding the regulatory requirements and costs associated with nonorphan drug development.