Interventions of Interest:
- etranacogene dezaparvovec (Hemgenix, CSL Behring)
- valoctocogene roxaparvovec (Roctavian ™; BioMarin)
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 be inappropriate to require step therapy with emicizumab prior to coverage for Roctavian.
ICER’s Chief Medical Officer David Rind, MD:
“Hemophilia is a serious, lifelong disease, that leads to pain and disability as a result of repeated bleeds into joints and muscles, and can limit options for education, employment, and recreation. Prophylaxis with factor replacement is burdensome and does not achieve normal clotting although, for patients with hemophilia A, emicizumab has reduced burdens and improved outcomes. The new gene therapies can result in successfully treated patients appearing ‘cured’ for at least a period of time. During this period, these gene therapies will eliminate the need for expensive prophylactic treatment. However, the duration of this ‘cure’ and the safety of therapies remain important uncertainties. At least with valoctocogene roxaparvovec, patients will eventually return to needing prophylaxis.”