Keeping up with the Cartashians - Classical Hematology Drug Approvals over Past 5 Years

Abstract

Classical hematological disorders encompass a broad array of congenital and acquired blood conditions, ranging from thrombotic and hemorrhagic disorders, hemoglobinopathies such as sickle cell disease/thalassemia, abnormalities in blood counts, disorders of iron metabolism, obstetric hematologic conditions, and rare genetic hematologic diseases. The past decade has seen a revolution in research and therapeutic development for these disorders. It has led to FDA approval of novel therapies such as gene therapies for hemophilia and hemoglobinopathies, targeted therapies for coagulation and complement disorders, factor VIII mimetics, new immunotherapeutic targets for cytopenia and genomic-directed diagnosis and therapy for a wide range of blood disorders. Between January 2018 and July 2023, 34 new drugs have been approved for different classical hematological disorders, and several more have received approval for new or expanded indications. This is changing how we treat patients with classical hematological conditions. Most classical hematological disorders have an impact on patients' quality of life as well as life span, and for decades, many of these disorders were managed with limited therapy options with variable efficacy. Rare hemolytic disorders such as cold agglutinin disease, paroxysmal nocturnal hemoglobinuria, thrombotic thrombocytopenic purpura, atypical hemolytic uremic syndrome, sickle cell disease, thalassemia, porphyria all have expanded options. Newer agents for the management of anemia, thrombocytopenia and leukopenia in different clinical settings are available. Lastly, management of hemophilia is being revolutionized by extended half-life products, non-factor options and gene therapy. Hemophilia care has always been deemed to be costly, but gene therapy is getting additional attention due to the high price tag. We have compiled the list of all new drugs that got FDA approval for classical hematological disorders over past 5 years and plan to present this in the form of an infographic providing details on the drug along with its indication, trial leading to approval and cost implications of each therapy. Our intention is to summarize the drug development for classical hematology and highlight “not so benign” nature of these disorders and related therapy particularly in terms of financial toxicity for the patients.