Gene therapy for hemophilia B and an update on gene therapy for hemophilia A: effectiveness and value : final evidence report

Gene therapy for hemophilia B and an update on gene therapy for hemophilia A: effectiveness and value : final evidence report

Collection:
Health Policy and Services Research
Author(s):
Tice, Jeffrey A., author
Chu, Janet N., author
Moradi, Ashton, author
Herce-Hagiwara, Belen, author
Fahim, Shahariar Mohammed, author
Agboola, Foluso, author
Pearson, Steven D., author
Rind, David M., author
Walton, Surrey M., author
Sarker, Jyotirmoy, author
Contributor(s):
Institute for Clinical and Economic Review, issuing body.
California Technology Assessment Forum (Organization), issuing body.
Publication:
[Boston, Massachusetts] : Institute for Clinical and Economic Review, December 22, 2022
Language(s):
English
Format:
Text
Subject(s):
Antibodies, Bispecific
Antibodies, Monoclonal, Humanized
Cost-Effectiveness Analysis
Genetic Therapy
Hemophilia A -- therapy
Hemophilia B -- therapy
United States
Genre(s):
Technical Report
Abstract:
We conducted an economic evaluation of etranacogene dezaparvovec for the treatment of hemophilia B patients without inhibitors compared with prophylactic treatment. We also updated our economic evaluation of valoctocogene roxaparvovec for the treatment of hemophilia A patients without inhibitors compared with emicizumab. We reviewed the clinical effectiveness of etranacogene dezaparvovec compared with prophylaxis using factor IX preparations in adults eligible for factor prophylaxis. We updated our prior review of the clinical effectiveness of valoctocogene roxaparvovec in adults eligible for factor prophylaxis compared with both factor VIII prophylaxis and emicizumab. In ICER’s 2020 review, the evidence on the success rate, initial levels of factor achieved, and duration of benefit were limited because the valoctocogene roxaparvovec Phase 3 trial (GENEr8-1) data had only short follow-up data available for review. For both patient populations, we searched for evidence on patient-important outcomes including patient-reported quality of life, bleeding rates, treated bleeding events, pain, mental health status, and adverse events as well as factor activity levels which are important intermediate outcomes for gene therapy.
Copyright:
Reproduced with permission of the copyright holder. Further use of the material is subject to CC BY license. (More information)
Extent:
1 online resource (1 PDF file (various pagings))
Illustrations:
Illustrations
NLM Unique ID:
9918697386506676 (See catalog record)
Permanent Link:
http://resource.nlm.nih.gov/9918697386506676