Cost and quality of life; Cost-effectiveness; Medico economic analysis; Newborn screening; Real-world data; Spinal muscular atrophy
Abstract :
[en] The objective of the study was to assess the cost-effectiveness of real-world spinal muscular atrophy newborn screening followed by treatment. We modeled the lifetime cost-effectiveness of the spinal muscular atrophy newborn screening followed by treatment (screening) compared to treatment without screening (no screening) from the Belgian healthcare perspective. Real-world data, including quality of life, costs, and motor development data, were collected on 12 patients identified by screening and 43 patients identified by their symptoms. "Screening" was associated with slightly higher healthcare costs (€ 6,858,061 vs. € 6,738,120) but more quality-adjusted life years (QALY) (40.95 vs. 20.34) compared to "no screening", leading to an incremental cost-effectiveness ratio of € 5,820 per QALY gained. "Screening" was dominant from a societal perspective (negative incremental costs: € -14,457; incremental QALY = 20.61), when incorporating the burden on caregivers (negative incremental costs = € -74,353; incremental QALY = 27.51), and when the treatment was chosen by the parents (negative incremental costs = € -2,596,748; incremental QALY = 20.61). Spinal muscular atrophy newborn screening coupled with early treatment is thus cost-effective compared with late treatment following clinical diagnosis and is dominant when societal perspective, caregiver burden, and treatment based on parental preference were considered.
Disciplines :
Neurology Pediatrics
Author, co-author :
Dangouloff, Tamara ; Université de Liège - ULiège > Département des sciences cliniques
Thokala, Praveen; Health Economics and Decision Science, School of Health and Related Research, University of Sheffield, Sheffield, UK.
Stevenson, Matthew D; Health Economics and Decision Science, School of Health and Related Research, University of Sheffield, Sheffield, UK.
Deconinck, Nicolas; Neuromuscular Reference Center and Paediatric Neurology Department, Hôpital des Enfants Reine Fabiola (HUDERF), Université Libre de Bruxelles, Brussels, Belgium.
D'Amico, Adèle; Unit of Neuromuscular and Neurodegenerative Disorders, IRCCS Bambino Gesù Children's Hospital, Rome, Italy.
Daron, Aurore ; Centre Hospitalier Universitaire de Liège - CHU > > Service de pédiatrie
DELSTANCHE, Stéphanie ; Centre Hospitalier Universitaire de Liège - CHU > > Service de neurologie (CHR)
Hiligsmann, Mickael; Department of Health Services Research, CAPHRI Care and Public Health Research Institute, Maastricht University, Maastricht, The Netherlands.
Language :
English
Title :
Cost-effectiveness of spinal muscular atrophy newborn screening based on real-world data in Belgium.
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