Cost-Effectiveness of Technologies for the Treatment of Spinal Muscular Atrophy: A Systematic Review of Economic Studies

André Motta-Santos; Kenya Noronha; Carla Reis; Daniela Freitas; Lélia Carvalho; Mônica Andrade

doi:10.1016/j.vhri.2024.02.002

Cost-Effectiveness of Technologies for the Treatment of Spinal Muscular Atrophy: A Systematic Review of Economic Studies

Value Health Reg Issues. 2024 Apr 25:42:100985. doi: 10.1016/j.vhri.2024.02.002. Online ahead of print.

Authors

André Motta-Santos¹, Kenya Noronha², Carla Reis³, Daniela Freitas⁴, Lélia Carvalho⁵, Mônica Andrade⁶

Affiliations

¹ Department of Economics, Universidade Federal de Minas Gerais, Belo Horizonte, Minas Gerais, Brazil; School of Business, University of Southern Queensland, Toowoomba, Queensland, Australia. Electronic address: andresantos111@ufmg.br.
² Department of Economics, Universidade Federal de Minas Gerais, Belo Horizonte, Minas Gerais, Brazil.
³ Department of Economics, Universidade Federal de Minas Gerais, Belo Horizonte, Minas Gerais, Brazil; Escola Nacional de Saúde Pública Sergio Arouca, Fundação Oswaldo Cruz, Rio de Janeiro, Rio de Janeiro, Brazil.
⁴ School of Medicine/Professor, Universidade José do Rosário Vellano, Belo Horizonte, Minas Gerais, Brazil; Center for Health Technology Assessment of the UFMG Teaching Hospital/Researcher, Universidade Federal de Minas Gerais, Belo Horizonte, Minas Gerais, Brazil.
⁵ Center for Health Technology Assessment of the UFMG Teaching Hospital/Coordinator, Universidade Federal de Minas Gerais, Belo Horizonte, Minas Gerais, Brazil.
⁶ Department of Economics/Professor, Universidade Federal de Minas Gerais, Belo Horizonte, Minas Gerais, Brazil.

PMID: 38669792
DOI: 10.1016/j.vhri.2024.02.002

Abstract

Objectives: This study aims to systematically collect data on cost-effectiveness analyses that assess technologies to treat type I and II spinal muscular atrophy and evaluate their recommendations.

Methods: A structured electronic search was conducted in 4 databases. Additionally, a complementary manual search was conducted. Complete economic studies that evaluated nusinersen, risdiplam, onasemnogene abeparvovec (OA), and the best support therapy (BST) from the health system's perspective were selected. The incremental cost-effectiveness ratios were compared with various thresholds for the analysis. The review was registered a priori in PROSPERO (CRD42022365391).

Results: Twenty studies were included in the analyses. They were all published between 2017 and 2022 and represent the recommendations in 8 countries. Most studies adopted 5, 6, or 10-state Markov models. Some authors took part in multiple studies. Four technologies were evaluated: BST (N = 14), nusinersen (N = 19), risdiplam (N = 5), and OA (N = 9). OA, risdiplam, and nusinersen were considered inefficient compared with the BST. Risdiplam and OA were generally regarded as cost-effective when compared with nusinersen. Because nusinersen is not a cost-effective drug, no recommendation can be derived from this result. Risdiplam and OA were compared in 2 studies that presented opposite results.

Conclusions: Nusinersen, risdiplam, and OA are being adopted worldwide as a treatment for spinal muscular atrophy. Despite that, the pharmacoeconomic analyses show that the technologies are not cost-effective compared with the BST. The lack of controlled studies for risdiplam and OA hamper any conclusions about their face-to-face comparison.

Keywords: Análise de custo-benefício; Atrofia Muscula Espinhal; Nusinersen; Onasemnogene Abeparvovec; Risdiplam; cost-benefit analysis; muscular atrophy; nusinersen; onasemnogene abeparvovec; risdiplam; spinal.