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Cost-Effectiveness of Technologies for the Treatment of Spinal Muscular Atrophy: A Systematic Review of Economic Studies.
Motta-Santos, André; Noronha, Kenya; Reis, Carla; Freitas, Daniela; Carvalho, Lélia; Andrade, Mônica.
Afiliación
  • Motta-Santos A; 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.
  • Noronha K; Department of Economics, Universidade Federal de Minas Gerais, Belo Horizonte, Minas Gerais, Brazil.
  • Reis C; 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.
  • Freitas D; 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.
  • Carvalho L; Center for Health Technology Assessment of the UFMG Teaching Hospital/Coordinator, Universidade Federal de Minas Gerais, Belo Horizonte, Minas Gerais, Brazil.
  • Andrade M; Department of Economics/Professor, Universidade Federal de Minas Gerais, Belo Horizonte, Minas Gerais, Brazil.
Value Health Reg Issues ; 42: 100985, 2024 Jul.
Article en En | MEDLINE | ID: mdl-38669792
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.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Oligonucleótidos / Atrofia Muscular Espinal / Análisis Costo-Beneficio Límite: Humans Idioma: En Revista: Value Health Reg Issues Año: 2024 Tipo del documento: Article Pais de publicación: Estados Unidos
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Oligonucleótidos / Atrofia Muscular Espinal / Análisis Costo-Beneficio Límite: Humans Idioma: En Revista: Value Health Reg Issues Año: 2024 Tipo del documento: Article Pais de publicación: Estados Unidos