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. 2024 Jan:34:61-67.
doi: 10.1016/j.nmd.2023.11.013. Epub 2023 Dec 2.

Cost-effectiveness of spinal muscular atrophy newborn screening based on real-world data in Belgium

Tamara Dangouloff  1 , Praveen Thokala  2 , Matthew D Stevenson  2 , Nicolas Deconinck  3 , Adèle D'Amico  4 , Aurore Daron  5 , Stephanie Delstanche  5 , Laurent Servais  6 , Mickael Hiligsmann  7
Affiliations
Free article

Cost-effectiveness of spinal muscular atrophy newborn screening based on real-world data in Belgium

Tamara Dangouloff et al. Neuromuscul Disord. 2024 Jan.
Free article

Abstract

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.

Keywords: Cost and quality of life; Cost-effectiveness; Medico economic analysis; Newborn screening; Real-world data; Spinal muscular atrophy.

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Conflict of interest statement

Declaration of Competing Interest TD has given lectures sponsored by Biogen, Novartis, and Roche. TD's doctoral thesis was financed by Biogen, Novartis and Roche funding the implementation of the newborn screening pilot programme in southern Belgium for three years (2018–2020). PT has performed paid consultancy for Roche and Novartis and served on an advisory board for Roche on SMA. ND is investigator on SMA studies for Roche, Novartis Gene Therapies, and Biogen and has received honoraria for service on scientific advisory boards of Roche and Novartis Gene Therapies. AD'A has received honoraria as a consultant and scientific advisory board member and has given sponsored lectures for Biogen, Avexis, and Roche. AD is investigator on SMA studies for Roche and Novartis Gene Therapies and has received honoraria as a scientific advisory board member of AveXis Belgium. SD has consulted for Biogen and Roche. LS is a coordinating investigator of SMA newborn screening programs in Belgium and in the UK funded by Roche, Novartis, and Biogen. He has consulted for Zentech, Biogen, Novartis, Roche, Scholar Rock, and BioHaven and has received research grants from Zentech and Perkin Elmer. MDS and MH have no conflict of interest relevant to this paper.