Basics
Breaking Barriers for Rare Diseases – Tax Series Part 5 of 7
Health Care Tax
Published on March 19, 2025
Explore This BasicWith the Orphan Drug Act (ODA) entering its forty-second year in 2025, advocates and lawmakers are taking stock of the United States’ progress in treating patients with rare diseases. Today, patients with rare diseases have shorter diagnostic journeys and more viable treatments than ever before. Despite this progress, hurdles continue as the gap between conditions and treatments remains significant, and some stakeholders believe the power of the ODA has decreased over time. Lawmakers and experts are searching for solutions to align what some say are misaligned incentives, improve the supply chain, and foster rare disease innovation which can provide hope to the 95% of rare disease patients without an FDA approved treatment while simultaneously ensuring those options are affordable when available.
- Congress.gov: The Orphan Drug Act – Legal Overview and Policy Considerations
- Government Accountability Office: Rare Disease Drugs
- Health and Human Services Department: High-expenditure Medicare drugs often qualified for Orphan Drug Act incentives
- Johnson & Johnson – What is a Rare Disease
- NORD – Rare Disease Policy in Action
- NORD – The Orphan Drug Act Turns 40
- NORD – Understanding Rare Diseases
- National Library of Medicine – A Comprehensive Study of the Rare Diseases and Conditions Targeted by Orphan Drug Designations and Approvals Over the Forty Years of the Orphan Drug Act
- Summit Health – The Challenges of Treating Rare Diseases
- University of Chicago – The Orphan Drug Act at 35
- University of Southern California – Medicare’s “Coverage with Evidence Development”