Journal article

Publication year: 2020

Journal: Translational Vision Science & Technology

Volume number: 9

Issue number: 9

ISSN: 2164-2591

Open access status: Gold

DOI Link: https://doi.org/10.1167/tvst.9.9.17

Publisher: Association for Research in Vision and Ophthalmology

Abstract: 

Purpose: 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 (sic)410,550 per eye and potential indirect cost offsets by higher labor force participation.

Methods: We developed an individual patient samplingmodel 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, comparedwith natural progression and converted to quality of life. Direct and indirect costs are recorded and the incremental cost-utility ratio is calculated.

Results: In the base case scenario, VN provides 4.82 additional quality-adjusted lifeyears over a patient's lifetime at an incremental cost-utility ratio of (sic)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 lifeyears and costs, direct costs, and natural progression.

Conclusions: Under a lifetime perspective, VN proves to be cost effective for theGerman 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.

Translational Relevance: 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.

Harvard Citation style: Uhrmann, M., Lorenz, B. and Gissel, C. (2020) Cost Effectiveness of Voretigene Neparvovec for RPE65-Mediated Inherited Retinal Degeneration in Germany, Translational Vision Science & Technology, 9(9), Article 17. https://doi.org/10.1167/tvst.9.9.17

APA Citation style: Uhrmann, M., Lorenz, B., & Gissel, C. (2020). Cost Effectiveness of Voretigene Neparvovec for RPE65-Mediated Inherited Retinal Degeneration in Germany. Translational Vision Science & Technology. 9(9), Article 17. https://doi.org/10.1167/tvst.9.9.17