Posted by Michael Wonder on 04 Jan 2020
ICER to assess gene therapy for haemophilia A
3 January 2020 - Report will be subject of New England CEPAC meeting in August 2020; open Input now being accepted until 22 January 2020.
The Institute for Clinical and Economic Review (ICER) announced today that it plans to assess the comparative clinical effectiveness and value of valoctocogene roxaparvovec (BioMarin Pharmaceutical) for the treatment of haemophilia. Valoctocogene roxaparvovec is an investigational gene therapy, and a decision regarding FDA approval is expected by mid-2020. ICER’s analysis will likely compare valoctocogene roxaparvovec to factor VIII replacement therapy and emicizumab (Hemlibra, Genentech).
ICER’s Evidence Report on haemophilia A will be reviewed during a public meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC) in August 2020.
