Evaluating Long-Term Cost Benefits of Etranacogene Dezaparvovec in Hemophilia B Management

A study published in Journal of Managed Care and Specialty Pharmacy evaluated the budgetary implications of introducing etranacogene dezaparvovec, a novel gene therapy for hemophilia B, into a US commercial health plan.

Hemophilia B, a rare genetic disorder affecting blood clotting due to a deficiency in factor IX (FIX), is currently treated with routine FIX prophylaxis to prevent bleeding episodes. This approach is associated with high costs over $600 000 annually per patient with hemophilia B (PwHB), along with treatment burdens. Etranacogene dezaparvovec was approved by the US Food and Drug Administration (FDA) in 2022 as a one-time treatment designed to sustainably increase FIX levels, potentially eliminating the need for ongoing prophylaxis in PwHB.

The study modeled the impact of adding etranacogene dezaparvovec to a 1-million-member health plan over 5- and 10-year horizons, focusing on costs related to drug acquisition, bleed management, monitoring, and adverse events. The primary analysis compared scenarios with and without the gene therapy, incorporating gradual and immediate uptake models.

Over a 5-year horizon, gradual uptake of etranacogene dezaparvovec resulted in a modest budget impact, increasing plan costs cumulatively by $848 509, or an average of $0.007 to $0.023 per member per month (PMPM). In contrast, immediate adoption of the therapy for all eligible patients led to up-front costs but achieved cost savings by year 2, with cumulative 5-year savings of $754 844. Over a 10-year horizon, both adoption scenarios demonstrated cost savings, with gradual uptake saving $1.28 million and immediate adoption saving $7.98 million.

The study highlights that while introducing etranacogene dezaparvovec may initially strain health plan budgets, its potential to replace routine prophylaxis and significantly reduce associated costs makes it an economically favorable option over time. By addressing the high up-front cost with durable clinical benefits, the therapy is a groundbreaking treatment strategy for hemophilia B.

“Initiating PwHB on etranacogene dezaparvovec sooner may produce greater overall savings and earlier annual savings. Etranacogene dezaparvovec is a treatment option that may provide overall cost savings for US commercial health plans, which would increase as the plan size increases,” the researchers concluded.

Reference

Wilson M, McDade C, Thiruvillakkat K, Rouse R, Sivamurt K, Yan S. Assessing health plan payer's budget impact of etranacogene dezaparvovec for the treatment of hemophilia B in the United States. J Manag Care Spec Pharm. 2024;30(8):805-816. doi:10.18553/jmcp.2024.23214.