Aim: The aim of the project is to investigate the cost-effectiveness of treating Danish patients with spinal muscular atrophy with Spinraza compared to the current treatment which is Best Standard of Care (BSC)

Methods: There is used a Markov model to estimate both costs and QALY for both treatment strategies: ‘Spinraza-alternative’ and ‘BSC-alternative’. The models have a lifelong time horizon from a limited social perspective. The model structure, costs and effects are based on a systematic literature search. Because type 3 is an orphan disease and Spinraza is an orphan drugs the available evidence is limited and assumptions had to be made.
Results: The analysis show that the costs for the ‘Spinraza-alternative’ is very high coming in at 45.699.163,02 DKK. The costs per incremental QALY produced with treatment with Spinraza is 3.952.419,64. However, due to the assumptions made there are a large uncertainty surrounding the result.

Discussion: This project presents the first health economic evaluation of Spinraza compared to BSC in Denmark. Furthermore, this is also the first economic description of BSC in Denmark. The methods applied is used in other studies, and some have been used as basis for decision making for implementing Spinraza in other countries. Lastly future research is discussed along with the limitations in collecting data about orphan drugs diseases.

Conclusion: In Denmark, an ICER cannot be judged as cost-efficient since there is no threshold. However, is the result compared to the applied to known thresholds in England, Spinraza is not cost-efficient.