COST-EFFECTIVENESS ANALYSIS OF VILLAGE IN A DISH AS A PHARMACOGENOMIC TEST TO PREDICT TREATMENT OUTCOMES OF RILUZOLE VERSUS EDARAVONE FOR AMYOTROPHIC LATERAL SCLEROSIS PATIENTS
Author(s)
Jacqui Simpkin, BSc1, Talha Qureshi, MSc2, Pieter Vandekerckhove, PhD3, Carin Uyl-De Groot, Sr., PhD4, Ken Redekop, MPH, PhD5.
1Erasmus University Rotterdam, Amsterdam, Netherlands, 2Erasmus School of Health Policy & Management, Rotterdam, Netherlands, 3TU Delft, P.b.m.vandekerckhove@tudelft.nl, Belgium, 4ESHPM/iMTA Erasmus University Rotterdam, Rotterdam, Netherlands, 5IMTA (Erasmus University), Rotterdam, Netherlands.
1Erasmus University Rotterdam, Amsterdam, Netherlands, 2Erasmus School of Health Policy & Management, Rotterdam, Netherlands, 3TU Delft, P.b.m.vandekerckhove@tudelft.nl, Belgium, 4ESHPM/iMTA Erasmus University Rotterdam, Rotterdam, Netherlands, 5IMTA (Erasmus University), Rotterdam, Netherlands.
OBJECTIVES: Amyotrophic Lateral Sclerosis (ALS) is a rare, progressive, and fatal neurodegenerative disease currently treated with Riluzole. Village in a Dish (ViaD) is a novel cell culture technology that leverages genetic diversity to improve personalized medicine. ViaD can be applied as a pharmacogenomic tool to guide treatment selection for ALS between edaravone and riluzole. As edaravone is not approved in the Netherlands and ViaD is not yet developed for clinical use, this study evaluated cost-effectiveness of ViaD under the hypothetical scenario in which edaravone was available in the Netherlands and ViaD was functional in clinical care.
METHODS: A combined decision tree and Markov model was developed to compare ViaD-based treatment with standard care. The analysis was conducted from a Dutch societal perspective over a 40-year lifetime horizon with a starting cohort of 1,000 patients and monthly transition cycles. Costs and health outcomes were discounted at annual rates of 3% and 1.5%, respectively. All parameters were derived from the literature, the Dutch costing manual, and informed assumptions where data were unavailable. Uncertainty was assessed using probabilistic sensitivity analysis (1,000 simulations), diagnostic accuracy analysis, and scenario analyses.
RESULTS: At base case, ViaD generated an ICER of €1,358,227/QALY. In comparison with standard care, ViaD produced more QALYs (1.84 vs 1.82) but at higher costs (€622,827 vs €587,289). Disease progression and edaravone efficacy were the main drivers of high ICER. Results were robust across all sensitivity analyses and scenarios. The probabilistic ICER was consistent with the base case.
CONCLUSIONS: ViaD-guided treatment selection for ALS would not be cost-effective in the Netherlands if edaravone was available alongside riluzole, as the ICER is approximately seventeen times the Dutch WTP threshold of €80,000. Therefore, stakeholders should prioritize the development of other applications of ViaD that may yield greater health benefits at lower costs.
METHODS: A combined decision tree and Markov model was developed to compare ViaD-based treatment with standard care. The analysis was conducted from a Dutch societal perspective over a 40-year lifetime horizon with a starting cohort of 1,000 patients and monthly transition cycles. Costs and health outcomes were discounted at annual rates of 3% and 1.5%, respectively. All parameters were derived from the literature, the Dutch costing manual, and informed assumptions where data were unavailable. Uncertainty was assessed using probabilistic sensitivity analysis (1,000 simulations), diagnostic accuracy analysis, and scenario analyses.
RESULTS: At base case, ViaD generated an ICER of €1,358,227/QALY. In comparison with standard care, ViaD produced more QALYs (1.84 vs 1.82) but at higher costs (€622,827 vs €587,289). Disease progression and edaravone efficacy were the main drivers of high ICER. Results were robust across all sensitivity analyses and scenarios. The probabilistic ICER was consistent with the base case.
CONCLUSIONS: ViaD-guided treatment selection for ALS would not be cost-effective in the Netherlands if edaravone was available alongside riluzole, as the ICER is approximately seventeen times the Dutch WTP threshold of €80,000. Therefore, stakeholders should prioritize the development of other applications of ViaD that may yield greater health benefits at lower costs.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE64
Topic
Economic Evaluation, Medical Technologies
Disease
Neurological Disorders