ICER Publishes Revised Hemophilia Gene Therapy Evidence Report

A revised evidence report has been published by the Institute for Clinical and Economic Review (ICER) detailing the effectiveness and value of two gene therapies for hemophilia A and B. The gene therapies offer nearly complete remission of bleeds for a period of time without the need for prophylactic factor replacement.

According to ICER, “Patients treated with etranacogene dezaparvovec had an 80% reduction in treated joint bleeds and similar reductions in other bleeds when compared with their bleeding rates on factor prophylaxis prior to gene therapy. However, there is still considerable uncertainty about the duration of benefit and the risk of uncommon harms with etranacogene dezaparvovec. We conclude that there is moderate certainty of a small or substantial health benefit with high certainty of at least a small net health benefit (B+) for etranacogene dezaparvovec compared with factor IX prophylaxis.”

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(Source: ICER, November 2^nd, 2022)