Nusinersena (Spinraza) na intervenção da atrofia muscular espinhal tipo I versus ausência de tratamento medicamentoso sob a ótica do custo-efetividade: uma revisão sistemática.

Abstract

Spinal Muscular Atrophy (SMA) is a rare and devastating disease for which new disease-modifying drugs have recently been approved by the world's leading regulatory agencies. Given the growing importance of economic considerations in evaluating the incorporation of health technologies into Brazilian’s public health system, this review summarizes the studies that assess the cost-effectiveness of type I SMA and the economic evaluations of treatments via Nusinersen versus conventional treatment. A systematic literature review in Medline via PubMed, Lilacs via BVS and SciELO up to December 31, 2020 was conducted in accordance with PRISMA’s guidelines. The methodology allowed to clearly identify the cost-effective relationship of treatment based only on supportive therapy versus drug treatment via Nusinersen. It wa concluded that drug treatment is not cost-effective in relation to supportive therapy, but it brings favorable clinical, financial, and psychological benefits to the patient and their families. Studies focusing on health technology assessments are still quite scarce, therefore, they lack more in-depth research in this area, where resources are insufficient and, therefore, absorb a large part of the government budget.