PAREXEL International (United States)

Cost-effectiveness of etranacogene dezaparvovec compared to eftrenonacog alfa for the treatment of adult patients with severe and moderately severe haemophilia B in Sweden

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.Aim To evaluate cost-effectiveness of EDZ vs. prophyl...