Open AccessModeling Benefits, Costs, and Affordability of a Novel Gene Therapy in Hemophilia A
Author(s) -
Ham Renske M.T.,
Walker Simon M.,
Soares Marta O.,
Frederix Geert W.J.,
Leebeek Frank W.G.,
Fischer Kathelijn,
Coppens Michiel,
Palmer Stephen J.
Publication year - 2022
Publication title -
hemasphere
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.677
H-Index - 11
ISSN - 2572-9241
DOI - 10.1097/hs9.0000000000000679
Subject(s) - medicine , quality adjusted life year , payment , cost effectiveness , economics , finance , risk analysis (engineering)
The objective was to undertake an early cost‐effectiveness assessment of valoctocogene roxaparvovec (valrox; Roctavian) compared to factor (F)VIII prophylaxis or emicizumab (Hemlibra; Roche HQ, Bazel, Switzerland) in patients with severe Hemophilia A (HA) without FVIII‐antibodies. We also aimed to incorporate and quantify novel measures of value such as treatment durability, maximum value‐based price (MVBP) and break‐even time (ie, time until benefits begin to offset upfront payment). We constructed a Markov model to model bleeds over time which were linked to costs and quality‐of‐life decrements. In the valrox arm, FVIII over time was estimated combining initial effect and treatment waning and then linked to bleeds. In FVIII and emicizumab arms, bleeds were based on trial evidence. Evidence and assumptions were validated using expert elicitation. Model robustness was tested via sensitivity analyses. A Dutch societal perspective was applied with a 10‐year time horizon. Valrox in comparison to FVIII, and emicizumab showed small increases in quality‐adjusted life years at lower costs, and were therefore dominant. Valrox’ base case MVBP was estimated at €2.65 million/treatment compared to FVIII and €3.5 million/treatment versus emicizumab. Mean break‐even time was 8.03 years compared to FVIII and 5.68 years to emicizumab. Early modeling of patients with HA in The Netherlands treated with valrox resulted in estimated improved health and lower cost compared to prophylactic FVIII and emicizumab. We also demonstrated feasibility of incorporation of treatment durability and novel outcomes such as value‐based pricing scenarios and break‐even time. Future work should aim to better characterize uncertainties and increase translation of early modeling to direct research efforts.