Interventions of Interest: Fair pricing benchmarks suggest upper bounds for price of Roctavian at approximately $1.9 M and for Hemgenix at approximately $2.9 M. Payers should work with manufacturers to develop and implement outcomes-based agreements to address the uncertainty and the high cost of gene therapies for hemophilia. Finally, clinical experts affirm that it would […]
Interventions of Interest: Factor VIII: Factor VIII concentrate is given intravenously, whether administered on-demand or prophylactically. Prophylaxis is administered multiple times per week, which is burdensome. Emicizumab (Hemlibra®, Genentech): a monoclonal antibody with dual targets that allow it to bridge activated factor IX and factor X, the role normally played by activated factor VIII in […]
Interventions of Interest: Emicizumab-kxwh (Hemlibra®, Genentech) Hemophilia A is an inherited condition that results in a deficiency in the factor VIII blood clotting protein. Individuals with hemophilia A are at risk for life-threatening bleeding; bleeding into a joint or muscle is common and can lead to substantial disability. To reduce the risk of bleeding, patients […]