On January 4th, the rare disease community reflects on the lasting impact of the Orphan Drug Act (ODA), a landmark law signed in 1983 to address the lack of treatments for rare conditions. Before the ODA, drug development focused largely on common diseases, leaving many rare disease communities without viable treatment options due to high development costs and small patient populations.
The Orphan Drug Act changed this trajectory by creating incentives—such as tax credits, federal grants, and market exclusivity—to encourage pharmaceutical companies and researchers to invest in rare disease therapies. Championed by U.S. Representative Henry Waxman and supported by patient advocates and public figures like actor Jack Klugman, the ODA affirmed a simple but powerful principle: people with rare diseases deserve the same attention and care as anyone else.
Since its passage, the ODA has transformed the treatment landscape. What began with just a few dozen approved therapies has grown into hundreds of orphan drugs addressing more than 1,000 rare conditions. Beyond expanding treatment options, the ODA elevated rare diseases within medical research, regulatory policy, and public awareness—fostering collaboration among researchers, clinicians, regulators, and patient advocacy organizations.
For our community, the Orphan Drug Act has expanded care options for people living with lymphatic malformations, including isolated LMs and complex lymphatic anomalies. While many treatments are not FDA-approved specifically for these conditions, therapies such as sirolimus, trametinib, propranolol, interferon-alpha, VEGF inhibitors, and alpelisib were developed within a system shaped by the ODA—highlighting both progress and the ongoing need for research.
Despite this progress, approximately 95% of rare diseases still lack an FDA-approved treatment. Policy changes that weaken development incentives risk slowing innovation at a time when scientific discovery is accelerating. Advocacy efforts continue to call for strengthening the ODA through restored incentives, improved data sharing, and greater patient involvement in drug development.
More than four decades later, the Orphan Drug Act remains a cornerstone of rare disease progress. Continued collaboration and advocacy are essential to ensure that its promise—hope, innovation, and access—extends to every rare disease community.
