US spending on high-revenue rare disease drugs in 2022

To estimate the use and spending for high-revenue rare disease drugs in 2022 in the US.

Cross-sectional study.

Among the 100 prescription drugs with the highest 2022 US net sales revenue, we selected drugs exclusively approved to treat rare diseases as designated under the Orphan Drug Act. We estimated commercial, Medicare, and Medicaid spending using manufacturer-reported net sales and public Medicare and Medicaid spending data. We also estimated the number of individuals using each drug based on Medicare spending per beneficiary data.

Nineteen of the 100 highest-revenue prescription drugs in 2022 were exclusively approved to treat Orphan Drug Act-designated conditions; 9 had a single indication, and 10 had multiple indications. Median annual net sales for the 19 drugs were $1.97 billion (range, $988 million-$8.36 billion). Total spending on these 19 drugs was $45.1 billion, which represented approximately 7.5% of the $603 billion in prescription drug spending in 2022. The median estimated number of individuals using each drug was 17,152 (range, 3735-71,171). Of the total spending, $23.6 billion (52.3%) was spent for individuals with Medicare, $3.5 billion (7.8%) for those with Medicaid, and $18.0 billion (39.9%) for those with commercial insurance.

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.