Findings This cross-sectional study identified 25 “sole orphan” drugs qualifying for exemption from Medicare price negotiation. Meaning The results of this study suggest that exempting sole orphan drugs from Medicare price negotiation will cost taxpayers billions of dollars per year; such savings could be used to control Medicare premium increases or provide other benefits for patients. Outcomes The primary outcomes were the number of sole orphan drugs, estimated Medicare spending on those drugs from 2012 to 2021, and global revenue since launch. Results Among 282 drugs, 95 (34%) were approved to treat at least 1 rare disease, including 25 sole orphan drugs (26%), 20 multiorphan drugs (21%), 13 orphan first drugs (14%), and 37 non–orphan first drugs (39%). From 2012 to 2021, Medicare spending on sole orphan drugs increased from $3.4 billion to $10.0 billion.

Source: New York Times November 27, 2023 16:05 UTC