Roundtable on Optimizing the Management of Inherited Blood Disorders

Findings from an AMCP Market Insights Program

Hemophilia, sickle cell disease (SCD), and beta-thalassemia (β thal) are inherited blood disorders likely to see multiple significant novel therapies approved in the near future, including the possibility for partial or complete cures with gene therapies. Although these are rare blood disorders, the high cost of treatment is an important consideration for payers and employers in ensuring that potentially curative therapies are affordable and accessible to the patients who need them. Patients with inherited blood disorders, particularly those with severe disease, are at risk for life-threatening complications and report significant impact on family life, recreational activities, school activities, and work.

To understand the appropriate and cost-effective use of treatments for inherited blood disorders, AMCP convened an expert panel of managed care stakeholders in October 2022. Panelists included representatives from national and regional health plans, integrated delivery systems, hospitals, pharmacy benefit managers, and patient advocacy organizations. The double-blinded program included testimony from patients with hemophilia, SCD, and β thal.