Commonwealth Fund November 24, 2025
Abstract
- Issue: The Orphan Drug Act of 1983 has helped spur the development of drugs targeting rare diseases for which no treatment exists. However, lack of investment in research and development, high levels of financial risk, extremely high costs for these new drugs, and manufacturing and regulatory hurdles remain major problems.
- Goal: To provide an overview of the law and its impact, outline concerns and challenges, and discuss proposals to address shortcomings.
- Methods: Review of federal government reports, documents, and data; academic research; and media stories.
- Key Findings and Conclusions: Some of the problems associated with the orphan drug market result from the actions of drugmakers exploiting loopholes in the Orphan Drug Act to take advantage of the law’s...
Today's Sponsors
Today's Sponsor