(D5) Rare Disease Spotlight: Hemophilia Treatment Advances

This activity is supported by an educational grant from CSL Behring.

Currently, about 20,000 individuals are living with hemophilia in the United States, and about 60% of individuals with hemophilia A experience a severe form of the blood disorder. Those with hemophilia B, however, might not begin exhibiting symptoms until later in life. Development in the rare disease drug landscape is expected to increase, where one-third of the drug pipeline extending to 2024 is through the orphan drug approval pathway. The first hemophilia B gene therapy received FDA approval in November 2022 and additional novel hemophilia treatment options are expected within the next year—what managed care strategies reduce treatment gaps and address unmet needs? Faculty for this session will discuss hemophilia treatment advances, identify considerations for developing payment models, and discuss innovative methods to address access to care.