This budget-impact study modelled 5-, 10- and 15-year payer costs of etranacogene dezaparvovec (Hemgenix) in Swedish adults with haemophilia B. A cohort Markov model compared full upfront, 30% reduced upfront and outcome-based annuity payment against factor IX prophylaxis. Scenarios covered short-term trial outcomes, full response, and partial response with a 10% annual failure rate. Hemgenix cost more than prophylaxis for the first 10 years under every scenario and payment model. At 15 years, reduced upfront payment cost least with sustained full response; annuities cost least with partial response. The cohort was hypothetical, and the annuity advantage under partial response disappeared after 3% annual discounting.
Why it is interesting: Single-dose gene therapies put their cost up front, while their benefit depends on uncertain long-term durability. This study shows how far the payment model and the discount rate change which option costs payers least.
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