US Spending on High-Revenue Rare Disease Drugs in 2022
Approximately 1 in 5 of the highest-revenue prescription drugs in the US in 2022 was approved to treat only rare conditions; these drugs were used by a small number of patients but accounted for a sizeable share of spending. Rare disease drugs should be included in federal and state policies aimed at improving the affordability of prescription drugs for patients and the health care system.
Authors
- Helen Mooney
- Aaron Seth Kesselheim (ORCID: https://orcid.org/0000-0002-8867-2666)
- Benjamin N. Rome (ORCID: https://orcid.org/0000-0001-5776-3293)
Institutions
- Brigham and Women's Hospital (US)
Publication Details
- Journal
- The American Journal of Managed Care
- Published
- 2025-11-14
- DOI
- https://doi.org/10.37765/ajmc.2025.89823
- Primary Topic
- Health Systems, Economic Evaluations, Quality of Life
- Type
- article
- Field-Weighted Citation Impact
- 0.00