Economic and healthcare resource utilization for hereditary transthyretin amyloidosis with polyneuropathy in Spain: real-world insights from early-stage patients and mutation carriers.
Fernando Martínez Valle, Lucía Galán Dávila, Juan Buades Reinés, Juan Gonzalez-Moreno, Inés Losada López, Teresa Sevilla, Francisco Muñoz Beamud, José Eulalio Bárcena Llona, Manuel Romero Acebal, Patricia Tarilonte, Francesca Setaro, Javier Rejas-Gutierrez, Alejandra López-Ibáñez de Aldecoa, Carmen Peral
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引用次数: 0
Abstract
Objective: Variant transthyretin (ATTRv) amyloidosis is a rare genetic multisystem disease inducing progressive morbidity. Evidence about its economic burden is scarce. This study estimated annual use and costs of healthcare-resource-utilization (HRU) and indirect costs for ATTRv mutation carriers and patients with polyneuropathy from a societal perspective in Spain.
Methods: A cross-sectional, retrospective non-interventional multicenter study was conducted in 2018-2020 to characterize asymptomatic carriers of mutations in the TTR gen (AC) and Coutinho stage 1 transthyretin amyloidosis polyneuropathy patients (PA). HRU, associated costs, and indirect wages were assessed during 12 months prior to enrollment.
Results: One-hundred-five participants were analyzed: 86 AC and 19 PA. Average healthcare cost-per-person-per-year was significantly higher in PA: €2,323.6 vs. €953.3 (p < 0.001), due to higher utilization of medical specialty visits, hospitalizations, and nerve conduction studies. Indirect costs were statistically not different (€380.1 for AC and €781.3 for PA, p = 0.581). Days worked with fatigue/pain were significantly higher in PA: 53.9 vs 5.5 (p = 0.034). Total cost was significantly higher in PA than AC (€3,105.0 vs €1,333.4; p < 0.001).
Conclusions: Both AC and early-stage ATTRv-PN patients showed a meaningful economic burden to the National Health System and society. We observed an annual incremental economic burden of €1,771.5 in PA versus AC.
期刊介绍:
Expert Review of Pharmacoeconomics & Outcomes Research (ISSN 1473-7167) provides expert reviews on cost-benefit and pharmacoeconomic issues relating to the clinical use of drugs and therapeutic approaches. Coverage includes pharmacoeconomics and quality-of-life research, therapeutic outcomes, evidence-based medicine and cost-benefit research. All articles are subject to rigorous peer-review.
The journal adopts the unique Expert Review article format, offering a complete overview of current thinking in a key technology area, research or clinical practice, augmented by the following sections:
Expert Opinion – a personal view of the data presented in the article, a discussion on the developments that are likely to be important in the future, and the avenues of research likely to become exciting as further studies yield more detailed results
Article Highlights – an executive summary of the author’s most critical points.