On August 26, 2020, ICER released its Draft Evidence Report entitled, “Valoctocogene Roxaparvovec and Emicizumab for Hemophilia A.”  Patients Rising Now submitted comments on the draft report raising a number of concerns about both the substance of the report and the methodology.  For example, the FDA has declined to advance its own review of valoctocogene roxaparvovec, delaying the earliest possible approval date to 2021 or 2022, but ICER is proceeding with its review anyway.  It is premature to make any clinical or policy decisions about this treatment, making the ICER report’s “findings” inherently unactionable.  To read Patients Rising Now’s full comments, click here.

Terry Wilcox, Co-Founder and Executive Director
Patients Rising Now