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Open Access 23-03-2024 | Hemophilia | Original Research Paper

Cost-Effectiveness Analysis of Etranacogene Dezaparvovec Versus Extended Half-Life Prophylaxis for Moderate-to-Severe Haemophilia B in Germany

Authors: Niklaus Meier, Hendrik Fuchs, Katya Galactionova, Cedric Hermans, Mark Pletscher, Matthias Schwenkglenks

Published in: PharmacoEconomics - Open | Issue 3/2024

Abstract

Background and Objective

Haemophilia B is a rare genetic disease that is caused by a deficiency of coagulation factor IX (FIX) in the blood and leads to internal and external bleeding. Under the current standard of care, haemophilia is treated either prophylactically or on-demand via intravenous infusions of FIX. These treatment strategies impose a high burden on patients and health care systems as haemophilia B requires lifelong treatment, and FIX is costly. Etranacogene dezaparvovec (ED) is a gene therapy for haemophilia B that has been recently approved by the United States Food and Drug Administration and has received a recommendation for conditional marketing authorization by the European Medicines Agency. We aimed to examine the cost-effectiveness of ED versus extended half-life FIX (EHL-FIX) prophylaxis for moderate-to-severe haemophilia B from a German health care payer perspective.

Methods

A microsimulation model was implemented in R. The model used data from the ED phase 3 clinical trial publication and further secondary data sources to simulate and compare patients receiving ED or EHL-FIX prophylaxis over a lifetime horizon, with the potential for ED patients to switch treatment to EHL-FIX prophylaxis when the effectiveness of ED waned. Primary outcomes of this analysis included discounted total costs, discounted quality-adjusted life years (QALYs), incremental cost-effectiveness, and the incremental net monetary benefit. The annual discount rate for costs and effects was 3%. Uncertainty was examined via probabilistic analysis and additional univariate sensitivity analyses.

Results

Probabilistic analysis indicated that patients treated with ED instead of EHL-FIX prophylaxis gained 0.50 QALYs and experienced cost savings of EUR 1,179,829 at a price of EUR 1,500,000 per ED treatment. ED was the dominant treatment strategy. At a willingness to pay of EUR 50,000/QALY, the incremental net monetary benefit amounted to EUR 1,204,840.

Discussion

Depending on the price, ED can save costs and improve health outcomes of haemophilia patients compared with EHL-FIX prophylaxis, making it a potentially cost-effective alternative. These results are uncertain due to a lack of evidence regarding the long-term effectiveness of ED.

Available only for authorised users

Based on the publication for the HOPE-B clinical trial, it was not possible to filter out the treatment failures from the mean ABR after treatment with ED. This means that the mean ABR of 1.51 may be an overestimation for ABR under treatment success, but due to the lack of further data, it was not possible to correct for this.

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Title: Cost-Effectiveness Analysis of Etranacogene Dezaparvovec Versus Extended Half-Life Prophylaxis for Moderate-to-Severe Haemophilia B in Germany
Authors: Niklaus Meier
Hendrik Fuchs
Katya Galactionova
Cedric Hermans
Mark Pletscher
Matthias Schwenkglenks
Publication date: 23-03-2024
Publisher: Springer International Publishing
Keywords: Hemophilia
Gene Therapy in Oncology
Published in: PharmacoEconomics - Open / Issue 3/2024
Print ISSN: 2509-4262
Electronic ISSN: 2509-4254
DOI: https://doi.org/10.1007/s41669-024-00480-z

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Cost-Effectiveness Analysis of Etranacogene Dezaparvovec Versus Extended Half-Life Prophylaxis for Moderate-to-Severe Haemophilia B in Germany

Abstract

Background and Objective

Methods

Results

Discussion

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Abstract

Background and Objective

Methods

Results

Discussion

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