The effectiveness and value of emicizumab and valoctocogene roxaparvovec for the management of hemophilia A without inhibitors

J Manag Care Spec Pharm. 2021 May;27(5):667-673. doi: 10.18553/jmcp.2021.27.5.667.


DISCLOSURES: Funding for this summary was contributed by Arnold Ventures, California Health Care Foundation, The Donaghue Foundation, Harvard Pilgrim Health Care, and Kaiser Foundation Health Plan to the Institute for Clinical and Economic Review (ICER), an independent organization that evaluates the evidence on the value of health care interventions. ICER's annual policy summit is supported by dues from AbbVie, Aetna, America's Health Insurance Plans, Anthem, Alnylam, AstraZeneca, Biogen, Blue Shield of CA, Boehringer-Ingelheim, Cambia Health Services, CVS, Editas, Evolve Pharmacy, Express Scripts, Genentech/Roche, GlaxoSmithKline, Harvard Pilgrim, Health Care Service Corporation, HealthFirst, Health Partners, Humana, Johnson & Johnson (Janssen), Kaiser Permanente, LEO Pharma, Mallinckrodt, Merck, Novartis, National Pharmaceutical Council, Pfizer, Premera, Prime Therapeutics, Regeneron, Sanofi, Spark Therapeutics, uniQure, and United Healthcare. Agboola, Rind, Herron-Smith, and Pearson are employed by ICER. Walton and Quach, through the University of Illinois at Chicago, received funding from ICER for development of the economic model described in this report.

MeSH terms

  • Antibodies, Bispecific / economics*
  • Antibodies, Bispecific / pharmacology
  • Antibodies, Monoclonal, Humanized / economics*
  • Antibodies, Monoclonal, Humanized / pharmacology
  • Blood Coagulation / drug effects*
  • Cost-Benefit Analysis
  • Drug Costs
  • Factor VIII
  • Genetic Therapy
  • Hemophilia A / drug therapy*
  • Humans
  • Models, Economic
  • Treatment Outcome


  • Antibodies, Bispecific
  • Antibodies, Monoclonal, Humanized
  • emicizumab
  • F8 protein, human
  • Factor VIII