(D2) IRON-ing Out the Details: Beta Thalassemia Landscape, Emerging Treatments, and Payer Considerations

This activity is supported by an educational grant from Vertex Pharmaceuticals Inc.

Beta thalassemia is a rare inherited blood disorder characterized by significantly reduced or absent production of hemoglobin, an iron-containing protein in red blood cells. Disease burden spans from patient and caregiver quality of life and health system utilization to the overall cost of care. The approval of betibeglogene autotemcel at $2.8 million for a one-time treatment changed the treatment paradigm in beta thalassemia as the first cell-based gene therapy that provides a potential cure to patients. In addition, there is another potentially curative gene therapy, exagamglogene autotemcel, in late-stage development with approval anticipated in 2023. These novel treatments, combined with luspatercept-aamt, represent a shift in treatment options away from chronic transfusions. If these high-cost therapies get approved, with clinical benefits only shown in small clinical trials, payers must consider innovative management strategies.

This session will provide an in-depth review of the current treatment paradigm for beta thalassemia, approved and pipeline gene therapies, and new perspectives on payer management for these therapies.