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Matthias Fritz Uhrmann, Birgit Lorenz, Christian Gissel; Cost Effectiveness of Voretigene Neparvovec for RPE65-Mediated Inherited Retinal Degeneration in Germany. Trans. Vis. Sci. Tech. 2020;9(9):17. doi: https://doi.org/10.1167/tvst.9.9.17.
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Voretigene Neparvovec-rzyl (VN) is the first available treatment for biallelic RPE65 mutation-associated inherited retinal degeneration, which is usually associated with infancy-onset severe visual impairment and complete blindness during the third life decade. We aim to estimate the cost effectiveness of VN in Germany considering medication costs of €410,550 per eye and potential indirect cost offsets by higher labor force participation.
We developed an individual patient sampling model to simulate patients over their lifetime. In a Monte Carlo analysis, 1000 simulations are performed. Cycle length of the two-state Markov model is 1 year. For each cycle, visual field and best-corrected visual acuity are tracked, compared with natural progression and converted to quality of life. Direct and indirect costs are recorded and the incremental cost-utility ratio is calculated.
In the base case scenario, VN provides 4.82 additional quality-adjusted life-years over a patient's lifetime at an incremental cost-utility ratio of €156,853 per additional quality-adjusted life-year gained. Sensitivity analyses show the robustness of the results when altering treatment effect duration, discounting of quality-adjusted life-years and costs, direct costs, and natural progression.
Under a lifetime perspective, VN proves to be cost effective for the German statutory health insurance system despite high initial treatment costs. Because VN has important implications for future gene therapies, cost-utility analyses have high economic relevance from a societal perspective.
Our research analyzes the value of a gene augmentation therapy in clinical care in terms of quality of life gains for patients with blindness from retinal degeneration.
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