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 with severe hemophilia A administer factor VIII concentrate intravenously several times per week. About 25% of these patients develop “inhibitors” that make the factor ineffective.
The report highlights need for future pricing of hemophilia treatments that allows for “shared savings” between innovators and society.
Interventions of Interest:
- Emicizumab-kxwh (Hemlibra®, Genentech)
Below you will find the final documents from the assessment review process:
“Emicizumab is an exciting innovation. It addresses unmet clinical needs in a group of patients with a very high burden of illness, while producing overall cost savings in the health system. However, emicizumab can be cost-saving at a very high price only because it is being compared to the huge costs currently associated with treating hemophilia. High drug prices, paired with an insurance structure that often requires significant patient cost sharing, results in overwhelming financial burdens for families year after year – so extreme that even a cost-saving treatment like emicizumab likely won’t prevent these families from reaching their out-of-pocket maximum payment. Payers, manufacturers, and policy makers must seek new approaches to address financial toxicity across the hemophilia landscape.”