Specialty drugs, used to treat complex conditions such as cancer, autoimmune disorders, and rare diseases, including both biologics and high-cost small molecule therapies, represent less than 5% of prescriptions yet account for 54% of pharmaceutical spending (approximately $263 billion in 2024), up from 47% in 2019. The median annual list prices of medicines launched in 2024 exceeded $350,000, with oncology and rare disease therapies surpassing $400 000 per patient.
Rising specialty drug spending raises concerns about the sustainability of health care budgets, yet prior studies have largely focused on costs and utilization trends, offering limited insight into the extent to which growth in specialty spending reflects a structural shift in the composition of newly approved therapies.
To address this gap, we examined US Food and Drug Administration (FDA) approvals of specialty drugs from 2000 to 2024 to estimate the share of therapies classified as specialty medicines and to characterize their key attributes, including orphan status and therapeutic area.