Multinational cost-utility analysis of panel-based pharmacogenetics-guided treatment of patients enrolled in the U-PGx PREPARE study

作者：Vasileios Fragoulakis, Jesse J. Swen, Margarita-Ioanna Koufaki, Kathrin Blagec, Tanja Blagus, Stefan Böhringer, Anne Cambon‐Thomsen, Erika Cecchin, Ka‐Chun Cheung, Vera H.M. Deneer, Mathilde Dupui, Siv Jönsson, Candace Joefield‐Roka, Katja S. Just, Mats O. Karlsson, Lidija Konta, Rudolf Koopmann, Marjolein Kriek, Thorsten Lehr, Lisanne E. N. Manson, Emmanuelle Rial‐Sebbag, Victoria Rollinson, Rossana Roncato, Matthias Samwald, Elke Schaeffeler, Μαρία Σκώκου, Matthias Schwab, Daniela Steinberger, Julia Stingl, Roman Tremmel, Richard M. Turner, Mandy H. van Rhenen, Cathelijne H. van der Wouden, Cristina Lucía Dávila-Fajardo, Vita Dolžan, Munir Pirmohamed, Gere Sunder–Plassmann, Giuseppe Toffoli, Henk‐Jan Guchelaar, George P. Patrinos, Christina Mitropoulou · 发表于：EClinicalMedicine · 年份：2025 · DOI：10.1016/j.eclinm.2025.103686 · 被引用次数：6 · 研究领域：Pharmacogenetics and Drug Metabolism、Health Systems, Economic Evaluations, Quality of Life、Acute Lymphoblastic Leukemia research

Background: Pharmacogenetics (PGx) aims to revolutionize healthcare by individualizing drug doses and medication choices. However, clinical uptake will require positive evaluation evidence of both clinical utility and cost-effectiveness. We have recently demonstrated the clinical utility of this approach, using a panel-based PGx-guided treatment of patients from various indications recruited in seven countries (PREPARE study). Methods: Here, we provide economic evidence from a multinational cost-utility analysis of PGx-guided treatment in 6930 patients participating in the PREPARE study. The study was conducted from March 2017 to June 2020. We used the national healthcare system's perspective in each participating country, including only direct medical costs that budget holders cover. A Visual Analog Scale was used to measure utility and the quality of life was estimated by averaging the Visual Analog Scale scores of participants over four specific time points in the study, namely baseline visit (day 1), week 4, week 12, and 18 months from the baseline visit. Findings: Our analysis showed that the PGx-guided treatment is marginally cost-effective at the threshold of €11,000 QALYs. Cost drivers were hospitalization and ADRs costs, accounting for most of the resources used in both groups (46% and 37.5% in the PGx-guided group versus 49% and 48% in the control group, respectively), as a result of the average duration of hospitalization [1.51 days (95% CI: 1.23-1.82) for the PGx-...