Cost-effectiveness of prophylactic emicizumab versus prophylactic recombinant factor VIII in patients with moderate or mild hemophilia A without inhibitors in the United States

Kunal C Potnis; Giri Viswanathan; Robert D Bona; Satoko Ito; Christine L Kempton; Ankur Pandya; Harlan M Krumholz; George Goshua

doi:10.1002/ajh.27014

Cost-effectiveness of prophylactic emicizumab versus prophylactic recombinant factor VIII in patients with moderate or mild hemophilia A without inhibitors in the United States

Am J Hematol. 2023 Sep;98(9):E247-E250. doi: 10.1002/ajh.27014. Epub 2023 Jul 4.

Authors

Kunal C Potnis¹, Giri Viswanathan², Robert D Bona³, Satoko Ito³, Christine L Kempton⁴, Ankur Pandya⁵, Harlan M Krumholz^{6

7}, George Goshua³

Affiliations

¹ Department of Internal Medicine, Yale School of Medicine, New Haven, Connecticut, USA.
² Yale College, New Haven, Connecticut, USA.
³ Section of Hematology, Department of Internal Medicine, Yale School of Medicine and Yale Cancer Center, New Haven, Connecticut, USA.
⁴ Department of Hematology and Medical Oncology and Hemophilia of Georgia Center for Bleeding and Clotting Disorders of Emory, Emory University School of Medicine, Atlanta, Georgia, USA.
⁵ Department of Health Policy and Management, Harvard T.H. Chan School of Public Health, Boston, Massachusetts, USA.
⁶ Section of Cardiovascular Medicine, Department of Internal Medicine, Yale School of Medicine, New Haven, Connecticut, USA.
⁷ Center for Outcomes Research and Evaluation, Yale New Haven Hospital, New Haven, Connecticut, USA.

PMID: 37401660
DOI: 10.1002/ajh.27014

Abstract

Prophylactic emicizumab is cost-ineffective in adults with moderate or mild hemophilia A without inhibitors at current pricing. The price of prophylactic emicizumab would need to decrease by >35% to become cost-effective in this patient population.

Publication types

Letter
Research Support, Non-U.S. Gov't

MeSH terms

Adult
Antibodies, Bispecific* / therapeutic use
Cost-Benefit Analysis
Factor VIII / therapeutic use
Hemophilia A* / drug therapy
Hemorrhage / prevention & control
Humans
United States

Substances

Factor VIII
emicizumab
Antibodies, Bispecific