OBJECTIVES: This systematic review aims to systematically collect data on cost-effectiveness analyses that assess technologies for spinal muscular atrophy (SMA) and evaluate their recommendations.

METHODS: A structured electronic search was conducted in four 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 to 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 eight countries. Most studies adopted 5, 6, or 10-state Markov models. Some authors took part in multiple studies. OA, risdiplam, and nusinersen were considered inefficient compared to the BST. Risdiplam and OA were generally regarded as cost-effective when compared to nusinersen. Since nusinersen is an inefficient drug, no recommendation can be derived from this result. Risdiplam and OA were compared in two studies that presented opposite results.

CONCLUSIONS: Nusinersen, risdiplam, and OA are being adopted worldwide as a treatment for SMA. Despite that, the cost-effectiveness analysis shows that the technologies are inefficient compared to the BST. The lack of controlled studies for risdiplam and OA hamper conclusions about their face-to-face safety and efficiency.