US Spending on High-Revenue Rare Disease Drugs in 2022

Nearly one in five of the top 100 highest-revenue prescription drugs in the US were approved only for rare diseases, with spending on these 19 rare disease drugs totaling $45.1 billion, or 7.5% of US prescription drug spending. Excluding rare disease drugs from federal and state drug price policies limits potential savings and patient impact of efforts improve prescription drug affordability.