A recent analysis examining U.S. trends in approvals and costs of classical hematology drugs shows that an estimated 2024 U.S. gross sales in this category was more than $70 billion. Other highlights include that approvals have doubled each decade over the past three decades, and biologics and biosimilars now represent two-thirds of approvals.
“In 2022, Medicare alone spent $42.1 billion on prescription drugs, with antineoplastic and classical hematology drugs accounting for 44% and 8% of the total, respectively,” wrote the authors, led by researchers at the Mayo Clinic in Rochester, Minnesota. “While antineoplastic drugs have been extensively studied, classical hematology drugs have received comparatively little attention.”
To shed more light on the specialty, the researchers collected data on all approvals of classical hematology drugs by the U.S. Food and Drug Administration (FDA) in the past 30 years. They analyzed per-patient costs and annual total U.S. sales. They then estimated costs and collected relevant sales data.
From 1995 to 2024, the FDA approved 98 classical hematology drugs; 11% in the first decade, 26% in the second, and 63% in the third. The products categories were pharmaceuticals (36%), biologics (54%), and biosimilars (10%). The most common indications for approval were bleeding (31%), anemia (30%), neutropenia (9%), and thrombocytopenia (9%). The most common associated conditions were hemophilia (25%), chemotherapy-induced neutropenia (9%), thromboembolism (8%), paroxysmal nocturnal hemoglobinuria (7%), anemia of chronic kidney disease (6%), iron-deficiency anemia (6%), and immune thrombocytopenia (5%). Six classical hematology drugs have been discontinued in that time.
Among all treatments that are not gene therapies, median costs were $3,000 per course for fixed-duration treatments (range = $64 to $378,000) and $467,000 annually for continuous-duration treatments (range = $2,400 to $1.8 million). Among the six gene therapies (three for hemophilia and one each for sickle disease, beta thalassemia, and sickle cell disease/beta thalassemia), the median cost per treatment was $2.9 million (range = $2.2 million to $3.5 million).
In total, the analysis estimates that U.S. gross sales across all drugs exceeded $70 billion in 2024. The top-selling indications were thromboembolism ($43.5 billion), hemophilia ($6.9 billion), immune thrombocytopenia ($4.9 billion), paroxysmal nocturnal hemoglobinuria ($4.7 billion), chemotherapy-induced neutropenia ($2.8 billion), anemia of chronic kidney disease ($2 billion), and iron-deficiency anemia ($1.8 billion). Total sales for all gene therapies combined were $143 million.
The authors noted that medications for thromboembolism accounted for more than half of spending and that continuous-duration treatments, particularly for rare hereditary diseases such as hemophilia, carry significant financial burden
“In these conditions, if gene therapies demonstrate durable efficacy, they may prove to be cost-effective alternatives.” They wrote. “As more gene therapies are developed, evaluating their long-term value relative to chronic high-cost treatments will be essential for shaping future reimbursement and access strategies.”
Reference
Go L, Go L, Elliott M, et al. 30 years of classical hematology drugs in the US: Approvals, costs, and sales. Abstract 2604. Presented at: the 67th American Society of Hematology Annual Meeting and Exposition, Dec. 6-9, 2025, Orlando, FL.
