Wolfgang Miesbach։ Gene Therapy for Haemophilia B Is Cheaper Than Lifelong Factor IX Prophylaxis in Sweden
Wolfgang Miesbach, Head of Hemostasis/Hemophilia Center at Medical Clinic 2 at University Hospital Frankfurt, shared a post on LinkedIn about a recent article by Songkai Yan et al., published in Journal of Medical Economics, adding:
”Gene therapy for haemophilia B is cheaper than lifelong factor IX prophylaxis in Sweden.
A Swedish model (Yan et al., J Med Econ 2026, funded by CSL Behring) compared one infusion of etranacogene dezaparvovec with lifelong eftrenonacog alfa prophylaxis, based on five-year HOPE-B data.
- Per patient, over a lifetime
- Fewer bleeds: 0.33 vs 2.99 per year
- 1.75 more quality-adjusted life years
- SEK 21.3 million saved, although gene therapy itself costs SEK 29.3 million
About 90 percent predicted to keep FIX activity above 5 percent for 60 years
Treating at 18 instead of 41.5 saves even more: SEK 29.2 million
Keep in mind:
Most of the QALY gain comes from an assumed quality-of-life boost after gene therapy Factor IX was costed at list price. At the ceiling price set by the Swedish agency TLV, the saving falls to SEK 6.9 million
Gene therapy only loses its cost advantage if the effect fades after about 5 years without a manufacturer guarantee. In HOPE-B, mean FIX activity was still 36 IU/dL at year 5, and only 1 of 54 patients restarted prophylaxis.
The result depends on two things: the real price of factor IX and how long the effect lasts.”
Title: Cost-effectiveness of etranacogene dezaparvovec compared to eftrenonacog alfa for the treatment of adult patients with severe and moderately severe haemophilia B in Sweden
Authors: Songkai Yan, Olof Mogard, Anna Tjärnlund-Wolf, Fredrik Sjöö, André Eid, Jack Ettinger, Douglass Drelich, Radovan Tomic, Stina Johansson
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