Cost-effectiveness of etranacogene dezaparvovec compared to eftrenonacog alfa for the treatment of adult patients with severe and moderately severe haemophilia B in Sweden.
One-time gene therapy for hemophilia B may eliminate lifelong infusions and costs, offering potential freedom from frequent treatments.
INTRODUCTION: Etranacogene dezaparvovec (EDZ), a novel one-time gene therapy for severe/moderately severe haemophilia B, provides sustained factor IX (FIX) activity, usually eliminating the need for lifelong replacement therapy. Etranacogene dezaparvovec was modeled as more effective and cost-saving than eftrenonacog alfa for adults with severe or moderately severe hemophilia B in Sweden.
What the study was
- Study design
- Comparative study
- Population
- Adults with severe or moderately severe hemophilia B
- Category
- Treatment Innovation
- Maturity
- Validated
- Journal
- Journal of medical economics
Why it surfaced
Matched Rare diseases with high unmet need; Comparative study; human evidence; conservative score 8/10.
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