Disruptive technology and hemophilia care: The multiple impacts of emicizumab

Abstract

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Antithrombotic Treatment in Patients With Hemophilia: an EHA-ISTH-EAHAD-ESO Clinical Practice Guidance

“…Although emicizumab prophylaxis has demonstrated hemostatic efficacy, patients may require concomitant administration of a bypassing agent or FVIII clotting factor replacement to prevent bleeding in the perioperative period. 129 The occurrence of thrombotic events and thrombotic microangiopathy (TMA) following aPCC administration in adult PWHA and inhibitors on emicizumab prompted several recommendations for managing patients with inhibitors on emicizumab prophylaxis in favor of rFVIIa. 130,131 In surgical and emergency settings, low doses of aPCC may be considered to manage cases of patients who do not respond to first-line treatment with rFVIIa.…”

“…Although emicizumab prophylaxis has demonstrated hemostatic efficacy, patients may require concomitant administration of a bypassing agent or FVIII clotting factor replacement to prevent bleeding in the perioperative period 129 …”

Modeling Benefits, Costs, and Affordability of a Novel Gene Therapy in Hemophilia A

“…Reassessment when new the evidence becomes available may decrease some uncertainty, but mostly will add to learnings of interpretation of early clinical data 65 . To add, full pipelines of novel therapies are observed for HA, including extended half‐life FVIII, nonreplacement therapies, and other gene therapies 66 . By adding arms to this model, (early) economic evaluations will allow more rapid assessment of their relative‐effectiveness and cost‐effectiveness.…”

“…65 To add, full pipelines of novel therapies are observed for HA, including extended half-life FVIII, nonreplacement therapies, and other gene therapies. 66 By adding arms to this model, (early) economic evaluations will allow more rapid assessment of their relative-effectiveness and cost-effectiveness.…”

Impact of novel hemophilia therapies around the world

“…Few agents have been incorporated into clinical practice at such a fast pace. According to Hermans and Makris, 75 25% to 35% of people with hemophilia A without inhibitors are under prophylaxis with emicizumab in Israel, the United Kingdom, and Belgium. In a real‐world cost estimate in the United States, emicizumab seems to be economically favorable compared to other agents for prophylaxis in people with or without inhibitors 76 …”