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.
- Describe the disease burden associated with beta thalassemia including disease complications and common treatment risks.
- Identify the current treatments for beta thalassemia and their costs.
- Summarize the safety, efficacy, and clinical use of new and emerging treatments for beta thalassemia.
- Discuss managed care trends and strategies for the management of beta thalassemia.
Jennifer Billings, PharmD, BCGP
Director, Clinical Pharmacy
IPD Analytics, LLC
Julia Mahler, PhamD
Curtis Lucas, RPh, MBA
Director -Medical Drug Strategy
The Academy of Managed Care Pharmacy (AMCP) is accredited by the Accreditation Council for Pharmacy Education as a provider of continuing pharmacy education.
ACPE UAN:0233-0000-23-002-H01-P • Activity Type: Knowledge-Based
Contact Hours: 1.25
Release Date: March 22, 2023 • Expiration Date: December 31, 2024
Activity Fee: Free for Members and for Non-Members
Participants should claim continuing pharmacy education (CPE) credit for this home study activity only if they have not claimed credit for the live activity presented at AMCP 2023. CPE credit will be processed through the AMCP Learn portal and reported directly to CPE Monitor. Please allow 72 hours for processing and posting in your NABP account.
To receive credit for an online activity you must:
- Review the full content of the activity.
- Successfully complete the post-test (required to pass with a score of 70% or higher, it may be taken a maximum of three times).
- Complete a specific activity evaluation.
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|Activity Number||Credit Amount||Accreditation Period|
|ACPE UAN #0233-0000-23-002-H01-P||1.25 Contact Hours||March 22, 2023 - December 31, 2024|