The Institute for Clinical and Economic Review (ICER) conducted a systematic literature review and cost-effectiveness analysis to evaluate the health and economic outcomes of etranacogene dezaparvovec and valoctocogene roxaparvovec gene therapies for hemophilia B and hemophilia A, respectively. Complete details of ICER’s systematic literature search and protocol, as well as the methodology and model structure for the economic evaluation, are available on ICER’s website. In this paper, we present the summary of our findings and highlights of the policy discussion with key stakeholders held at a public meeting of the California Technology Assessment Forum (CTAF) on November 18, 2022. The full report is available on the ICER website at h t t p s : // i c e r. o r g /w p – c o n t e n t/uploads/2022/05/ICER_Hemophilia_Final_Report_12222022.pdf.
