MAINTAINING INCENTIVES FOR RARE DISEASE DRUG DEVELOPMENT
The Orphan Drug Act (ODA) is an issue of huge importance to the rare disease community, patients, and families. Its impact on their lives is beyond measure. The incentives the ODA established for rare disease drug development—including the Orphan Drug Tax Credit—have successfully spurred innovation in treatment options for rare diseases (i.e., those that impact fewer than 200,000 people in the U.S.). Since the ODA was enacted in 1983, the Food and Drug Administration (FDA) has approved 486 orphan products, granted 3,280 orphan drug designations, and received 4,378 orphan drug designation requests.1,2 Yet even these numbers do not reflect the full impact of the ODA on rare disease drug development since some of these innovative products have been approved for more than one disease or condition. For the rare disease community, these orphan products may not only significantly improve quality of life, but may be life-saving. The patient stories included below show the impact of orphan drugs on these individual patients, and serve as examples of the potential impact of orphan drugs on rare disease patients’ lives.